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CompletedNCT03406702T-WAVEUpdated Sep 8, 2022Results posted

CX-8998 for Absence Seizures

A Phase 2 interventional study of CX-8998 in Epilepsy, sponsored by Jazz Pharmaceuticals. Completed at 7 sites in United States. Open to participants aged 16 Years to 55 Years. Per ClinicalTrials.gov, last updated 2022-09-08.

Sponsored by Jazz Pharmaceuticals · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
7
Allocation
Not applicable
Ages
16 Years to 55 Years
Sex
All
01

Study summary

This is a Phase 2a, open-label study consisting of a screening period of up to 4 weeks and a 4-dose-titration treatment period to a dose of up to 10 mg twice daily (BID) of CX-8998, followed by a 1-week safety follow-up period after the last dose of study medication.

Read the detailed description

This is a Phase 2a, open-label study consisting of a screening period of up to 4 weeks and a 4-dose-titration treatment period to a dose of up to 10 mg twice daily (BID) of CX-8998, followed by a 1-week safety follow-up period after the last dose of study medication.

Subjects will participate for a total of up to 9 weeks, including screening, the 4-week treatment period and follow-up.

02

Conditions studied

  • Epilepsy

Keywords

  • Epilepsy, Idiopathic Generalized Epilepsy, Absence Seizures
03

In context

Epilepsy

1,842 studies on the registry are indexed under Epilepsy; 423 are open to participants now.

This study's enrollment of 7 is below the median of 50 across 1,228 interventional studies indexed under Epilepsy.

Browse Epilepsy studies →

Lead sponsor

Jazz Pharmaceuticals is the lead sponsor of 173 studies on the registry; 19 are open to participants now.

Of its 42 completed or terminated interventional studies of FDA-regulated products, 31 (74%) have results posted.

Browse Jazz Pharmaceuticals studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
16 Years to 55 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Signed informed consent form (ICF) indicating that the subject has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts.
  2. Men or non-pregnant, non-breastfeeding women 16 to 55 years-of-age who are able to read and understand written and spoken local language.
  3. Clinical diagnosis of IGE (including, but not limited to, CAE, JAE, juvenile myoclonic epilepsy, or Jeavons syndrome) with absence seizures consistent with the International League against Epilepsy Revised Classification of Seizures (2017).
  4. Absence seizures persisting despite standard of care (SOC) treatment, defined as treatment with at least 2 AEDs appropriate for the patient's epilepsy syndrome. SOC failure, per investigator discretion, will be defined as insufficient clinical response or intolerable side effects, which precludes use of the appropriate AED.
  5. Observation of at least 3 instances of generalized discharges of approximately 2.5 - 4 Hz lasting ≥2 seconds via 24-hour ambulatory EEG (centrally reviewed), with approximately 75% normal background based on age and medication use per the central EEG reader's discretion. Intermittent focal spikes are allowed.
  6. On stable doses of one or more antiepileptic medication(s) for at least 30 days. If a subject is not on medication, adequate documentation justifying lack of therapy may be acceptable for the subject after sponsor review. Ketogenic, modified Atkins diet (MAD), or low glycemic diet with stable carbohydrate ratio for at least 30 days before screening is an acceptable antiepileptic therapy. Vagal nerve stimulation at stable settings (for at least 30 days before screening), without use of the magnet, is also acceptable.
  7. Body weight ≥ 45 kg at screening.
  8. Subjects with reproductive capability including all males and women of child-bearing potential (WOCBP) must agree to practice continuous abstinence or adequate contraception methods (appropriate double barrier method or oral, patch, implant, or injectable contraception) from as soon as feasible during screening period until at least 30 days after the last dose (i.e., intermittent abstinence based on "rhythm", temperature monitoring, or other means of timing is not acceptable). WOCBP include any woman who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, and/or bilateral oophorectomy) or is not post-menopausal. Post-menopausal is defined as amenorrhea ≥ 12 consecutive months without another cause, and a documented serum follicle stimulating hormone (FSH) level ≥ 35 mIU/mL.
  9. Male subjects with a partner of child-bearing potential must be surgically sterilized or be willing to use condoms with spermicide from as soon as feasible during screening period until at least 30 days after the last dose.
  10. Able and willing to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
  11. Approval by the sponsor medical personnel or delegate as to final eligibility for the study.

Exclusion criteria

Exclusion Criteria:

  1. History of surgical intervention for treatment of epilepsy.
  2. Additional seizure (clinical and electrographic) types, including, but not limited to, epileptic spasms, generalized tonic seizures, atonic seizures, or focal seizures. Subjects with GTCS or myoclonic seizures are eligible for the study.
  3. Inadequately treated psychotic or mood disorder (e.g., schizophrenia, major depression, bipolar disorder).
  4. Presence of severe intellectual disability, severe autism spectrum disorder, or severe developmental disorder such that the subject cannot sign the ICF or cannot cooperate with the study procedures.
  5. Presence of positive urine drug screen for drugs of abuse, except if this is explained by use of an allowed prescription medicine.
  6. Regular use of more than 2 standard drinks of alcohol per day (28 grams of pure alcohol).
  7. Hypersensitivity/allergic reaction to other T-type calcium agents, such as (but not limited to) ethosuximide and zonisamide.
  8. Use of strong CYP3A4 inhibitors, including prescription or non-prescription drugs or other products (i.e. grapefruit juice), which cannot be discontinued at least 2 weeks prior to Day 1 of dosing and throughout the study (Appendix C).

  9. Concurrent illnesses that would be a contraindication to trial participation, including, but not limited to:

    1. Severe arterial thromboembolic events (myocardial infarction, unstable angina pectoris, stroke) less than 6 months before screening
    2. NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled hypertension
    3. Clinically significant ECG abnormality per the Investigator assessment or any of the following: i) QTcF ≥450 msec (males) or ≥470 msec (females) ii) PR interval ≥250 msec iii) Atrioventricular block of second degree or higher, including Mobitz I iv) Persistent sinus bradycardia ≤ 50 beats per minute; persistent means the bradycardia is present on the first ECG and on one repeat ECG performed on another day v) For other ECG findings (e.g., including, but not necessarily limited to, tachycardia, bundle branch block, frequent ectopic beats, etc.) the Investigator should send a scanned, identity-blinded copy of the ECG tracing to the Study Safety Representative for review.
  10. Positive result for HIV, Hepatitis B [indicating ongoing infection], or Hepatitis C at screening or otherwise known ongoing infection with HIV, hepatitis B, or hepatitis C, unless curative therapy completed; for hepatitis C curative therapy is defined as negative PCR for HCV RNA.
  11. Significant hepatic (AST/ALT or bilirubin ≥ 2X upper limit of normal) or renal disease (creatinine clearance ≤39 mL/min) at screening.
  12. History of alcohol or substance abuse within the last year.
  13. A current C-SSRS score of 4 or 5 at screening or history of suicide attempt.
  14. Psychological, social, familial, or geographical reasons that would hinder or prevent compliance with the requirements of the protocol or compromise the informed consent process.
  15. Any other condition and/or situation that causes the Investigator or Study Safety Representative to deem a subject unsuitable for the study (including, but not limited to, expected study medication non-compliance, inability to medically tolerate the study procedures, or a subject's unwillingness to comply with study-related procedures).
  16. Treatment with an investigational agent within 30 days prior to the first dose of CX-8998 or planning to receive an investigational agent during the study.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
7 participants (actual)

Study arms

  • Experimental
    CX-8998

    T-type calcium channel blocker

    Drug: CX-8998

Interventions

  • DrugCX-8998

    T-type calcium channel blocker

06

What researchers measure

Primary outcomes

  1. Change From Baseline to End of Treatment in QT Interval Corrected for Heart Rate Using Fridericia's Formula (QTcF)

    Fridericia's Correction Formula (QTCF) is a formula which takes into account the physiologic shortening of the QT interval which occurs as the heart rate increases, permitting comparison of the QT interval across a range of rates.

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  2. Change From Baseline to End of Treatment in Clinical Alanine Aminotransferase Serum Chemistry Concentration

    Clinical safety laboratory assessment in alanine aminotransferase serum chemistry concentration.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  3. Change From Baseline to End of Treatment in Clinical Albumin Serum Chemistry Concentration

    Clinical safety laboratory assessment in albumin serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  4. Change From Baseline to End of Treatment in Clinical Albumin/Globulin Serum Chemistry Concentration

    Clinical safety laboratory assessment in albumin/globulin serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  5. Change From Baseline to End of Treatment in Clinical Alkaline Phosphatase Serum Chemistry Concentration

    Clinical safety laboratory assessment in alkaline phosphatase serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  6. Change From Baseline to End of Treatment in Clinical Aspartate Aminotransferase Serum Chemistry Concentration

    Clinical safety laboratory assessment in aspartate aminotransferase serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  7. Baseline Clinical Blood Urea Nitrogen/Creatinine Serum Chemistry Concentration

    Clinical safety laboratory assessment in BUN/Creatinine serum chemistry.

    Time frame: Baseline (Day 1)

  8. Change From Baseline to End of Treatment in Clinical Bilirubin Serum Chemistry Concentration

    Clinical safety laboratory assessment in bilirubin serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  9. Change From Baseline to End of Treatment in Clinical Blood Urea Nitrogen Serum Chemistry Concentration

    Clinical safety laboratory assessment in blood urea nitrogen serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  10. Change From Baseline to End of Treatment in Clinical Carbon Dioxide Serum Chemistry Concentration

    Clinical safety laboratory assessment in carbon dioxide serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  11. Change From Baseline to End of Treatment in Clinical Chloride Serum Chemistry Concentration

    Clinical safety laboratory assessment in chloride serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  12. Change From Baseline to End of Treatment in Clinical Calcium Serum Chemistry Concentration

    Clinical safety laboratory assessment in calcium serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  13. Change From Baseline to End of Treatment in Clinical Cholesterol Serum Chemistry Concentration

    Clinical safety laboratory assessment in cholesterol serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  14. Baseline Clinical Cholesterol/HDL-Cholesterol Serum Chemistry Concentration

    Clinical safety laboratory assessment in cholesterol/HDL-cholesterol serum chemistry.

    Time frame: Baseline (Day 1)

  15. Change From Baseline to End of Treatment in Clinical Creatine Kinase Serum Chemistry Concentration

    Clinical safety laboratory assessment in creatine kinase serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  16. Change From Baseline to End of Treatment in Clinical Creatinine Serum Chemistry Concentration

    Clinical safety laboratory assessment in creatinine serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  17. Change From Baseline to End of Treatment in Clinical Globulin Serum Chemistry Concentration

    Clinical safety laboratory assessment in globulin serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  18. Baseline Clinical Glomerular Filtration Rate (GFR) Adjusted for Body Surface Area (BSA) Serum Chemistry Concentration

    Clinical safety laboratory assessment in glomerular filtration rate adjusted for BSA chemistry.

    Time frame: Baseline (Day 1)

  19. Change From Baseline to End of Treatment in Clinical Estimated Glomerular Filtration Rate (GFR) Serum Chemistry Concentration

    Clinical safety laboratory assessment in GFR serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  20. Change From Baseline to End of Treatment in Clinical Glucose Serum Chemistry Concentration

    Clinical safety laboratory assessment in glucose serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  21. Baseline Clinical HDL Cholesterol Serum Chemistry Concentration

    Clinical safety laboratory assessment in HDL cholesterol serum chemistry.

    Time frame: Baseline (Day 1)

  22. Baseline Clinical LDL Cholesterol Serum Chemistry Concentration

    Clinical safety laboratory assessment in LDL cholesterol serum chemistry.

    Time frame: Baseline (Day 1)

  23. Change From Baseline to End of Treatment in Clinical Lactate Dehydrogenase Serum Chemistry Concentration

    Clinical safety laboratory assessment in lactate dehydrogenase serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  24. Change From Baseline to End of Treatment in Clinical Magnesium Serum Chemistry Concentration

    Clinical safety laboratory assessment in magnesium serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  25. Baseline Clinical Non-HDL Cholesterol Serum Chemistry Concentration

    Clinical safety laboratory assessment in non-HDL cholesterol serum chemistry.

    Time frame: Baseline (Day 1)

  26. Change From Baseline to End of Treatment in Clinical Phosphate Serum Chemistry Concentration

    Clinical safety laboratory assessment in phosphate serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  27. Change From Baseline to End of Treatment in Clinical Potassium Serum Chemistry Concentration

    Clinical safety laboratory assessment in potassium serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  28. Change From Baseline to End of Treatment in Clinical Protein Serum Chemistry Concentration

    Clinical safety laboratory assessment in protein serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  29. Change From Baseline to End of Treatment in Clinical Sodium Serum Chemistry Concentration

    Clinical safety laboratory assessment in sodium serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  30. Change From Baseline to End of Treatment in Clinical Triglycerides Serum Chemistry Concentration

    Clinical safety laboratory assessment in triglycerides serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  31. Change From Baseline to End of Treatment in Clinical Urate Serum Chemistry Concentration

    Clinical safety laboratory assessment in urate serum chemistry.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  32. Change From Baseline to End of Treatment in Basophils Hematology Assessment

    Clinical safety laboratory basophils hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  33. Change From Baseline to End of Treatment in Basophils/Leukocytes Hematology Assessment

    Clinical safety laboratory basophils/leukocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  34. Change From Baseline to End of Treatment in Eosinophils Hematology Assessment

    Clinical safety laboratory eosinophils hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  35. Change From Baseline to End of Treatment in Eosinophils/Leukocytes Hematology Assessment

    Clinical safety laboratory eosinophils/leukocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  36. Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Concentration Hematology Assessment

    Clinical safety laboratory mean corpuscular HGB concentration hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  37. Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Hematology Assessment

    Clinical safety laboratory mean corpuscular HGB hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  38. Change From Baseline to End of Treatment in Mean Corpuscular Volume Hematology Assessment

    Clinical safety laboratory mean corpuscular volume hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  39. Change From Baseline to End of Treatment in Erythrocytes Hematology Assessment

    Clinical safety laboratory erythrocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  40. Change From Baseline to End of Treatment in Erythrocytes Distribution Width Hematology Assessment

    Clinical safety laboratory erythrocytes distribution width hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  41. Change From Baseline to End of Treatment in Hematocrit Hematology Assessment

    Clinical safety laboratory hematocrit hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  42. Change From Baseline to End of Treatment in Hemaglobin Hematology Assessment

    Clinical safety laboratory hemaglobin hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  43. Change From Baseline to End of Treatment in Leukocytes Hematology Assessment

    Clinical safety laboratory leukocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  44. Change From Baseline to End of Treatment in Lymphocytes Hematology Assessment

    Clinical safety laboratory lymphocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  45. Change From Baseline to End of Treatment in Lymphocytes/Leukocytes Hematology Assessment

    Clinical safety laboratory lymphocytes/leukocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  46. Change From Baseline to End of Treatment in Mean Platelet Volume Hematology Assessment

    Clinical safety laboratory mean platelet volume hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  47. Change From Baseline to End of Treatment in Monocytes Hematology Assessment

    Clinical safety laboratory monocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  48. Change From Baseline to End of Treatment in Monocytes/Leukocytes Hematology Assessment

    Clinical safety laboratory monocytes/leukocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  49. Change From Baseline to End of Treatment in Neutrophils Hematology Assessment

    Clinical safety laboratory neutrophils hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  50. Change From Baseline to End of Treatment in Neutrophils/Leukocytes Hematology Assessment

    Clinical safety laboratory neutrophils/leukocytes hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  51. Change From Baseline to End of Treatment in Platelets Hematology Assessment

    Clinical safety laboratory platelets hematology assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  52. Baseline to End of Treatment in Bacteria Urinalysis Assessment

    Clinical safety laboratory bacteria urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  53. Baseline to End of Treatment in Urine Bilirubin Urinalysis Assessment

    Clinical safety laboratory urine bilirubin urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  54. Baseline to End of Treatment in Epithelial Cells Urinalysis Assessment

    Clinical safety laboratory epithelial cells urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-10 epithelial cells/high power field (hpf). A worse outcome is \>10 epithelial cells.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  55. Baseline to End of Treatment in Urine Erythrocytes Urinalysis Assessment

    Clinical safety laboratory urine erythrocytes urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-2 erythrocytes/high power field (hpf). A better outcome is 0 or "none seen."

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  56. Baseline to End of Treatment in Urine Glucose Urinalysis Assessment

    Clinical safety laboratory urine glucose urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  57. Baseline to End of Treatment in Ketones Urinalysis Assessment

    Clinical safety laboratory ketones urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  58. Baseline to End of Treatment in Leukocyte Esterase Urinalysis Assessment

    Clinical safety laboratory leukocyte esterase urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is "negative." An abnormal assessment or worse outcome is a positive assessment (i.e., 2+).

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  59. Baseline to End of Treatment in Urine Leukocytes Urinalysis Assessment

    Clinical safety laboratory urine leukocytes urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-5 leukocytes/high power field (hpf). An abnormal assessment or worse outcome is \>5 leukocytes/hpf (i.e., 11-30).

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  60. Baseline to End of Treatment in Mucous Threads Urinalysis Assessment

    Clinical safety laboratory mucous threads urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  61. Baseline to End of Treatment in Nitrite Urinalysis Assessment

    Clinical safety laboratory nitrite urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  62. Baseline to End of Treatment in Occult Blood Urinalysis Assessment

    Clinical safety laboratory occult blood urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is "negative." An abnormal assessment or worse outcome is a positive assessment (i.e., 2+).

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  63. Baseline to End of Treatment in Protein Urinalysis Assessment

    Clinical safety laboratory protein urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  64. Baseline to End of Treatment in Specific Gravity Urinalysis Assessment

    Clinical safety laboratory specific gravity urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is 1.005-1.030. An abnormal assessment or worse outcome is a value outside of this range.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  65. Baseline to End of Treatment in Specimen Appearance Urinalysis Assessment

    Clinical safety laboratory specimen appearance urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  66. Change From Baseline to End of Treatment in Urobilinogen Urinalysis Assessment

    Clinical safety laboratory urobilinogen urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  67. Baseline to End of Treatment in pH Urinalysis Assessment

    Clinical safety laboratory pH urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  68. Baseline to End of Treatment in Urine Color Urinalysis Assessment

    Clinical safety laboratory urine color urinalysis assessment.

    Time frame: Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

  69. Number (%) of Participants Who Did Not Complete The Study Due to Treatment-Emergent Adverse Events

    Treatment-emergent adverse events are all adverse events occurring during the treatment period or a pretreatment event that worsens in intensity during the treatment period as assessed by CTCAE v4.0.

    Time frame: Baseline (Day 1) up to Day 26 post-dose, or up to 1 year 3 weeks.

  70. Number (%) of Participants With Adverse Events of Special Interest

    An adverse event of special interest is a serious adverse event as defined in Outcome 6. This includes, however is not limited to, increased seizure frequency, new seizure types, worsening of EEG parameters, systemic adverse events based on safety profile as assessed by CTCAE v4.0.

    Time frame: Baseline (Day 1) up to Day 26 post-dose, or up to 1 year 3 weeks.

  71. Change From Baseline to End of Treatment in Respiration Rate

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  72. Change From Baseline to End of Treatment in Temperature

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  73. Baseline Weight

    Time frame: Baseline (Day 1)

  74. Change From Baseline to End of Treatment in Pulse

    The change from baseline to end of treatment in participants' pulses was assessed. The changes in recumbent pulse, standing pulse, and the change from recumbent to standing pulse are reported. Change from recumbent to standing pulse was measured by the difference in recumbent pulse change and standing pulse change.

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  75. Change From Baseline to End of Treatment in Systolic Blood Pressure

    The change from baseline to end of treatment in participants' systolic blood pressure (sbp) was assessed. The changes in recumbent sbp, standing sbp, and the change from recumbent to standing sbp are reported. Change from recumbent to standing sbp was measured by the difference in recumbent sbp change and standing sbp change.

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  76. Change From Baseline to End of Treatment in Diastolic Blood Pressure

    The change from baseline to end of treatment in participants' diastolic blood pressure (dbp) was assessed. The changes in recumbent dbp, standing dbp, and the change from recumbent to standing dbp are reported. Change from recumbent to standing dbp was measured by the difference in recumbent dbp change and standing dbp change.

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  77. Change From Baseline to End of Treatment in QT Interval

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  78. Change From Baseline to End of Treatment in QRS Interval

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  79. Change From Baseline to End of Treatment in PR Interval

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

  80. Change From Baseline to End of Treatment in Heart Rate

    Time frame: Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

07

Results

Posted Sep 8, 2022

Participant flow

Participant flow — Overall Study
MilestoneCX-8998
Started7
Completed6
Not completed1
Withdrew: Missed day 27 dosing1

Outcome measures

PrimaryChange From Baseline to End of Treatment in QT Interval Corrected for Heart Rate Using Fridericia's Formula (QTcF)

Fridericia's Correction Formula (QTCF) is a formula which takes into account the physiologic shortening of the QT interval which occurs as the heart rate increases, permitting comparison of the QT interval across a range of rates.

Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · msec
Change From Baseline to End of Treatment in QT Interval Corrected for Heart Rate Using Fridericia's Formula (QTcF)
msecCX-8998
Change From Baseline to End of Treatment in QT Interval Corrected for Heart Rate Using Fridericia's Formula (QTcF)3.50 ± 10.213
PrimaryChange From Baseline to End of Treatment in Clinical Alanine Aminotransferase Serum Chemistry Concentration

Clinical safety laboratory assessment in alanine aminotransferase serum chemistry concentration.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · U/L
Change From Baseline to End of Treatment in Clinical Alanine Aminotransferase Serum Chemistry Concentration
U/LCX-8998
Change From Baseline to End of Treatment in Clinical Alanine Aminotransferase Serum Chemistry Concentration17.29 ± 33.604
PrimaryChange From Baseline to End of Treatment in Clinical Albumin Serum Chemistry Concentration

Clinical safety laboratory assessment in albumin serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · g/L
Change From Baseline to End of Treatment in Clinical Albumin Serum Chemistry Concentration
g/LCX-8998
Change From Baseline to End of Treatment in Clinical Albumin Serum Chemistry Concentration-0.29 ± 4.152
PrimaryChange From Baseline to End of Treatment in Clinical Albumin/Globulin Serum Chemistry Concentration

Clinical safety laboratory assessment in albumin/globulin serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Clinical Albumin/Globulin Serum Chemistry Concentration
ratioCX-8998
Change From Baseline to End of Treatment in Clinical Albumin/Globulin Serum Chemistry Concentration-0.15 ± 0.212
PrimaryChange From Baseline to End of Treatment in Clinical Alkaline Phosphatase Serum Chemistry Concentration

Clinical safety laboratory assessment in alkaline phosphatase serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · U/L
Change From Baseline to End of Treatment in Clinical Alkaline Phosphatase Serum Chemistry Concentration
U/LCX-8998
Change From Baseline to End of Treatment in Clinical Alkaline Phosphatase Serum Chemistry Concentration3.14 ± 10.399
PrimaryChange From Baseline to End of Treatment in Clinical Aspartate Aminotransferase Serum Chemistry Concentration

Clinical safety laboratory assessment in aspartate aminotransferase serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · U/L
Change From Baseline to End of Treatment in Clinical Aspartate Aminotransferase Serum Chemistry Concentration
U/LCX-8998
Change From Baseline to End of Treatment in Clinical Aspartate Aminotransferase Serum Chemistry Concentration27.71 ± 60.810
PrimaryBaseline Clinical Blood Urea Nitrogen/Creatinine Serum Chemistry Concentration

Clinical safety laboratory assessment in BUN/Creatinine serum chemistry.

Time frame:
Baseline (Day 1)
Reported as:
Mean · ratio
Baseline Clinical Blood Urea Nitrogen/Creatinine Serum Chemistry Concentration
ratioCX-8998
Baseline Clinical Blood Urea Nitrogen/Creatinine Serum Chemistry Concentration12.70 ± NA
PrimaryChange From Baseline to End of Treatment in Clinical Bilirubin Serum Chemistry Concentration

Clinical safety laboratory assessment in bilirubin serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · umol/L
Change From Baseline to End of Treatment in Clinical Bilirubin Serum Chemistry Concentration
umol/LCX-8998
Change From Baseline to End of Treatment in Clinical Bilirubin Serum Chemistry Concentration-0.55 ± 2.577
PrimaryChange From Baseline to End of Treatment in Clinical Blood Urea Nitrogen Serum Chemistry Concentration

Clinical safety laboratory assessment in blood urea nitrogen serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · umol/L
Change From Baseline to End of Treatment in Clinical Blood Urea Nitrogen Serum Chemistry Concentration
umol/LCX-8998
Change From Baseline to End of Treatment in Clinical Blood Urea Nitrogen Serum Chemistry Concentration0.23 ± 1.254
PrimaryChange From Baseline to End of Treatment in Clinical Carbon Dioxide Serum Chemistry Concentration

Clinical safety laboratory assessment in carbon dioxide serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Carbon Dioxide Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Carbon Dioxide Serum Chemistry Concentration0.43 ± 2.936
PrimaryChange From Baseline to End of Treatment in Clinical Chloride Serum Chemistry Concentration

Clinical safety laboratory assessment in chloride serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Chloride Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Chloride Serum Chemistry Concentration-0.86 ± 3.716
PrimaryChange From Baseline to End of Treatment in Clinical Calcium Serum Chemistry Concentration

Clinical safety laboratory assessment in calcium serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Calcium Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Calcium Serum Chemistry Concentration-0.01 ± 0.109
PrimaryChange From Baseline to End of Treatment in Clinical Cholesterol Serum Chemistry Concentration

Clinical safety laboratory assessment in cholesterol serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Cholesterol Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Cholesterol Serum Chemistry Concentration0.60 ± 0.895
PrimaryBaseline Clinical Cholesterol/HDL-Cholesterol Serum Chemistry Concentration

Clinical safety laboratory assessment in cholesterol/HDL-cholesterol serum chemistry.

Time frame:
Baseline (Day 1)
Reported as:
Mean · ratio
Baseline Clinical Cholesterol/HDL-Cholesterol Serum Chemistry Concentration
ratioCX-8998
Baseline Clinical Cholesterol/HDL-Cholesterol Serum Chemistry Concentration2.40 ± NA
PrimaryChange From Baseline to End of Treatment in Clinical Creatine Kinase Serum Chemistry Concentration

Clinical safety laboratory assessment in creatine kinase serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · U/L
Change From Baseline to End of Treatment in Clinical Creatine Kinase Serum Chemistry Concentration
U/LCX-8998
Change From Baseline to End of Treatment in Clinical Creatine Kinase Serum Chemistry Concentration1174.86 ± 3110.151
PrimaryChange From Baseline to End of Treatment in Clinical Creatinine Serum Chemistry Concentration

Clinical safety laboratory assessment in creatinine serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · umol/L
Change From Baseline to End of Treatment in Clinical Creatinine Serum Chemistry Concentration
umol/LCX-8998
Change From Baseline to End of Treatment in Clinical Creatinine Serum Chemistry Concentration-4.14 ± 9.737
PrimaryChange From Baseline to End of Treatment in Clinical Globulin Serum Chemistry Concentration

Clinical safety laboratory assessment in globulin serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · g/dL
Change From Baseline to End of Treatment in Clinical Globulin Serum Chemistry Concentration
g/dLCX-8998
Change From Baseline to End of Treatment in Clinical Globulin Serum Chemistry Concentration0.15 ± 0.071
PrimaryBaseline Clinical Glomerular Filtration Rate (GFR) Adjusted for Body Surface Area (BSA) Serum Chemistry Concentration

Clinical safety laboratory assessment in glomerular filtration rate adjusted for BSA chemistry.

Time frame:
Baseline (Day 1)
Reported as:
Mean · mL/sec/1.73m^2
Baseline Clinical Glomerular Filtration Rate (GFR) Adjusted for Body Surface Area (BSA) Serum Chemistry Concentration
mL/sec/1.73m^2CX-8998
Baseline Clinical Glomerular Filtration Rate (GFR) Adjusted for Body Surface Area (BSA) Serum Chemistry Concentration1.80 ± NA
PrimaryChange From Baseline to End of Treatment in Clinical Estimated Glomerular Filtration Rate (GFR) Serum Chemistry Concentration

Clinical safety laboratory assessment in GFR serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · mL/sec/1.73m^2
Change From Baseline to End of Treatment in Clinical Estimated Glomerular Filtration Rate (GFR) Serum Chemistry Concentration
mL/sec/1.73m^2CX-8998
Change From Baseline to End of Treatment in Clinical Estimated Glomerular Filtration Rate (GFR) Serum Chemistry Concentration-5.82 ± 9.691
PrimaryChange From Baseline to End of Treatment in Clinical Glucose Serum Chemistry Concentration

Clinical safety laboratory assessment in glucose serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Glucose Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Glucose Serum Chemistry Concentration-0.14 ± 0.526
PrimaryBaseline Clinical HDL Cholesterol Serum Chemistry Concentration

Clinical safety laboratory assessment in HDL cholesterol serum chemistry.

Time frame:
Baseline (Day 1)
Reported as:
Mean · nmol/L
Baseline Clinical HDL Cholesterol Serum Chemistry Concentration
nmol/LCX-8998
Baseline Clinical HDL Cholesterol Serum Chemistry Concentration1.35 ± NA
PrimaryBaseline Clinical LDL Cholesterol Serum Chemistry Concentration

Clinical safety laboratory assessment in LDL cholesterol serum chemistry.

Time frame:
Baseline (Day 1)
Reported as:
Mean · nmol/L
Baseline Clinical LDL Cholesterol Serum Chemistry Concentration
nmol/LCX-8998
Baseline Clinical LDL Cholesterol Serum Chemistry Concentration1.50 ± NA
PrimaryChange From Baseline to End of Treatment in Clinical Lactate Dehydrogenase Serum Chemistry Concentration

Clinical safety laboratory assessment in lactate dehydrogenase serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · U/L
Change From Baseline to End of Treatment in Clinical Lactate Dehydrogenase Serum Chemistry Concentration
U/LCX-8998
Change From Baseline to End of Treatment in Clinical Lactate Dehydrogenase Serum Chemistry Concentration53.86 ± 98.104
PrimaryChange From Baseline to End of Treatment in Clinical Magnesium Serum Chemistry Concentration

Clinical safety laboratory assessment in magnesium serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Magnesium Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Magnesium Serum Chemistry Concentration-0.02 ± 0.077
PrimaryBaseline Clinical Non-HDL Cholesterol Serum Chemistry Concentration

Clinical safety laboratory assessment in non-HDL cholesterol serum chemistry.

Time frame:
Baseline (Day 1)
Reported as:
Mean · nmol/L
Baseline Clinical Non-HDL Cholesterol Serum Chemistry Concentration
nmol/LCX-8998
Baseline Clinical Non-HDL Cholesterol Serum Chemistry Concentration1.84 ± NA
PrimaryChange From Baseline to End of Treatment in Clinical Phosphate Serum Chemistry Concentration

Clinical safety laboratory assessment in phosphate serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Phosphate Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Phosphate Serum Chemistry Concentration0.09 ± 0.163
PrimaryChange From Baseline to End of Treatment in Clinical Potassium Serum Chemistry Concentration

Clinical safety laboratory assessment in potassium serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Potassium Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Potassium Serum Chemistry Concentration0.34 ± 0.532
PrimaryChange From Baseline to End of Treatment in Clinical Protein Serum Chemistry Concentration

Clinical safety laboratory assessment in protein serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · g/L
Change From Baseline to End of Treatment in Clinical Protein Serum Chemistry Concentration
g/LCX-8998
Change From Baseline to End of Treatment in Clinical Protein Serum Chemistry Concentration0.29 ± 3.352
PrimaryChange From Baseline to End of Treatment in Clinical Sodium Serum Chemistry Concentration

Clinical safety laboratory assessment in sodium serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Sodium Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Sodium Serum Chemistry Concentration-0.57 ± 1.902
PrimaryChange From Baseline to End of Treatment in Clinical Triglycerides Serum Chemistry Concentration

Clinical safety laboratory assessment in triglycerides serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Clinical Triglycerides Serum Chemistry Concentration
nmol/LCX-8998
Change From Baseline to End of Treatment in Clinical Triglycerides Serum Chemistry Concentration0.17 ± 0.659
PrimaryChange From Baseline to End of Treatment in Clinical Urate Serum Chemistry Concentration

Clinical safety laboratory assessment in urate serum chemistry.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · umol/L
Change From Baseline to End of Treatment in Clinical Urate Serum Chemistry Concentration
umol/LCX-8998
Change From Baseline to End of Treatment in Clinical Urate Serum Chemistry Concentration8.71 ± 48.555
PrimaryChange From Baseline to End of Treatment in Basophils Hematology Assessment

Clinical safety laboratory basophils hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Basophils Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Basophils Hematology Assessment0.02 ± 0.029
PrimaryChange From Baseline to End of Treatment in Basophils/Leukocytes Hematology Assessment

Clinical safety laboratory basophils/leukocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Basophils/Leukocytes Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Basophils/Leukocytes Hematology Assessment0.00 ± 0.004
PrimaryChange From Baseline to End of Treatment in Eosinophils Hematology Assessment

Clinical safety laboratory eosinophils hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Eosinophils Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Eosinophils Hematology Assessment0.02 ± 0.042
PrimaryChange From Baseline to End of Treatment in Eosinophils/Leukocytes Hematology Assessment

Clinical safety laboratory eosinophils/leukocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Eosinophils/Leukocytes Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Eosinophils/Leukocytes Hematology Assessment0.00 ± 0.007
PrimaryChange From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Concentration Hematology Assessment

Clinical safety laboratory mean corpuscular HGB concentration hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Concentration Hematology Assessment
nmol/LCX-8998
Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Concentration Hematology Assessment0.00 ± 1.230
PrimaryChange From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Hematology Assessment

Clinical safety laboratory mean corpuscular HGB hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · fmol
Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Hematology Assessment
fmolCX-8998
Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Hematology Assessment-0.04 ± 0.049
PrimaryChange From Baseline to End of Treatment in Mean Corpuscular Volume Hematology Assessment

Clinical safety laboratory mean corpuscular volume hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · fL
Change From Baseline to End of Treatment in Mean Corpuscular Volume Hematology Assessment
fLCX-8998
Change From Baseline to End of Treatment in Mean Corpuscular Volume Hematology Assessment-1.00 ± 2.828
PrimaryChange From Baseline to End of Treatment in Erythrocytes Hematology Assessment

Clinical safety laboratory erythrocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E12/L
Change From Baseline to End of Treatment in Erythrocytes Hematology Assessment
cellsx10E12/LCX-8998
Change From Baseline to End of Treatment in Erythrocytes Hematology Assessment-0.02 ± 0.194
PrimaryChange From Baseline to End of Treatment in Erythrocytes Distribution Width Hematology Assessment

Clinical safety laboratory erythrocytes distribution width hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Erythrocytes Distribution Width Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Erythrocytes Distribution Width Hematology Assessment0.00 ± 0.000
PrimaryChange From Baseline to End of Treatment in Hematocrit Hematology Assessment

Clinical safety laboratory hematocrit hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Hematocrit Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Hematocrit Hematology Assessment0.00 ± 0.015
PrimaryChange From Baseline to End of Treatment in Hemaglobin Hematology Assessment

Clinical safety laboratory hemaglobin hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · g/L
Change From Baseline to End of Treatment in Hemaglobin Hematology Assessment
g/LCX-8998
Change From Baseline to End of Treatment in Hemaglobin Hematology Assessment1.17 ± 6.555
PrimaryChange From Baseline to End of Treatment in Leukocytes Hematology Assessment

Clinical safety laboratory leukocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Leukocytes Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Leukocytes Hematology Assessment0.25 ± 2.228
PrimaryChange From Baseline to End of Treatment in Lymphocytes Hematology Assessment

Clinical safety laboratory lymphocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Lymphocytes Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Lymphocytes Hematology Assessment-0.17 ± 0.452
PrimaryChange From Baseline to End of Treatment in Lymphocytes/Leukocytes Hematology Assessment

Clinical safety laboratory lymphocytes/leukocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Lymphocytes/Leukocytes Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Lymphocytes/Leukocytes Hematology Assessment-0.02 ± 0.045
PrimaryChange From Baseline to End of Treatment in Mean Platelet Volume Hematology Assessment

Clinical safety laboratory mean platelet volume hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · fL
Change From Baseline to End of Treatment in Mean Platelet Volume Hematology Assessment
fLCX-8998
Change From Baseline to End of Treatment in Mean Platelet Volume Hematology Assessment0.65 ± 1.202
PrimaryChange From Baseline to End of Treatment in Monocytes Hematology Assessment

Clinical safety laboratory monocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Monocytes Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Monocytes Hematology Assessment0.01 ± 0.067
PrimaryChange From Baseline to End of Treatment in Monocytes/Leukocytes Hematology Assessment

Clinical safety laboratory monocytes/leukocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Monocytes/Leukocytes Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Monocytes/Leukocytes Hematology Assessment0.00 ± 0.021
PrimaryChange From Baseline to End of Treatment in Neutrophils Hematology Assessment

Clinical safety laboratory neutrophils hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Neutrophils Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Neutrophils Hematology Assessment0.41 ± 1.911
PrimaryChange From Baseline to End of Treatment in Neutrophils/Leukocytes Hematology Assessment

Clinical safety laboratory neutrophils/leukocytes hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · ratio
Change From Baseline to End of Treatment in Neutrophils/Leukocytes Hematology Assessment
ratioCX-8998
Change From Baseline to End of Treatment in Neutrophils/Leukocytes Hematology Assessment0.02 ± 0.063
PrimaryChange From Baseline to End of Treatment in Platelets Hematology Assessment

Clinical safety laboratory platelets hematology assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · cellsx10E9/L
Change From Baseline to End of Treatment in Platelets Hematology Assessment
cellsx10E9/LCX-8998
Change From Baseline to End of Treatment in Platelets Hematology Assessment10.67 ± 38.645
PrimaryBaseline to End of Treatment in Bacteria Urinalysis Assessment

Clinical safety laboratory bacteria urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Bacteria Urinalysis Assessment
ParticipantsCX-8998
Baseline (Not seen)1
End of Treatment (Few)1
PrimaryBaseline to End of Treatment in Urine Bilirubin Urinalysis Assessment

Clinical safety laboratory urine bilirubin urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Urine Bilirubin Urinalysis Assessment
ParticipantsCX-8998
Baseline (Negative)4
End of Treatment (Negative)2
PrimaryBaseline to End of Treatment in Epithelial Cells Urinalysis Assessment

Clinical safety laboratory epithelial cells urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-10 epithelial cells/high power field (hpf). A worse outcome is \>10 epithelial cells.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Epithelial Cells Urinalysis Assessment
ParticipantsCX-8998
Baseline (0-10)1
End of Treatment (>10)1
PrimaryBaseline to End of Treatment in Urine Erythrocytes Urinalysis Assessment

Clinical safety laboratory urine erythrocytes urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-2 erythrocytes/high power field (hpf). A better outcome is 0 or "none seen."

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Urine Erythrocytes Urinalysis Assessment
ParticipantsCX-8998
Baseline (0-2)1
End of Treatment (None seen)1
PrimaryBaseline to End of Treatment in Urine Glucose Urinalysis Assessment

Clinical safety laboratory urine glucose urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Urine Glucose Urinalysis Assessment
ParticipantsCX-8998
Baseline (Negative)4
End of Treatment (Negative)2
PrimaryBaseline to End of Treatment in Ketones Urinalysis Assessment

Clinical safety laboratory ketones urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Ketones Urinalysis Assessment
ParticipantsCX-8998
Baseline (Negative)3
Baseline (Trace)1
End of Treatment (Negative)2
PrimaryBaseline to End of Treatment in Leukocyte Esterase Urinalysis Assessment

Clinical safety laboratory leukocyte esterase urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is "negative." An abnormal assessment or worse outcome is a positive assessment (i.e., 2+).

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Leukocyte Esterase Urinalysis Assessment
ParticipantsCX-8998
Baseline (Negative)4
End of Treatment (2+)1
End of Treatment (Negative)1
PrimaryBaseline to End of Treatment in Urine Leukocytes Urinalysis Assessment

Clinical safety laboratory urine leukocytes urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-5 leukocytes/high power field (hpf). An abnormal assessment or worse outcome is \>5 leukocytes/hpf (i.e., 11-30).

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Urine Leukocytes Urinalysis Assessment
ParticipantsCX-8998
Baseline (0-5)1
End of Treatment (11-30)1
PrimaryBaseline to End of Treatment in Mucous Threads Urinalysis Assessment

Clinical safety laboratory mucous threads urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Mucous Threads Urinalysis Assessment
ParticipantsCX-8998
Baseline (Present)1
End of Treatment (Present)1
PrimaryBaseline to End of Treatment in Nitrite Urinalysis Assessment

Clinical safety laboratory nitrite urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Nitrite Urinalysis Assessment
ParticipantsCX-8998
Baseline (Negative)4
End of Treatment (Negative)2
PrimaryBaseline to End of Treatment in Occult Blood Urinalysis Assessment

Clinical safety laboratory occult blood urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is "negative." An abnormal assessment or worse outcome is a positive assessment (i.e., 2+).

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Occult Blood Urinalysis Assessment
ParticipantsCX-8998
Baseline (2+)1
Baseline (Negative)3
End of Treatment (Negative)2
PrimaryBaseline to End of Treatment in Protein Urinalysis Assessment

Clinical safety laboratory protein urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Protein Urinalysis Assessment
ParticipantsCX-8998
Baseline (Negative)4
End of Treatment (Negative)2
PrimaryBaseline to End of Treatment in Specific Gravity Urinalysis Assessment

Clinical safety laboratory specific gravity urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is 1.005-1.030. An abnormal assessment or worse outcome is a value outside of this range.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Specific Gravity Urinalysis Assessment
ParticipantsCX-8998
Baseline (1.015)1
Baseline (1.019)1
Baseline (1.025)1
Baseline (1.029)1
End of Treatment (1.023)1
End of Treatment (1.024)1
End of Treatment (1.026)1
PrimaryBaseline to End of Treatment in Specimen Appearance Urinalysis Assessment

Clinical safety laboratory specimen appearance urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Specimen Appearance Urinalysis Assessment
ParticipantsCX-8998
Baseline (Clear)4
End of Treatment (Clear)1
End of Treatment (Cloudy)1
End of Treatment (Turbid)1
PrimaryChange From Baseline to End of Treatment in Urobilinogen Urinalysis Assessment

Clinical safety laboratory urobilinogen urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Mean · nmol/L
Change From Baseline to End of Treatment in Urobilinogen Urinalysis Assessment
nmol/LCX-8998
Change From Baseline to End of Treatment in Urobilinogen Urinalysis Assessment0.00 ± 0.000
PrimaryBaseline to End of Treatment in pH Urinalysis Assessment

Clinical safety laboratory pH urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in pH Urinalysis Assessment
ParticipantsCX-8998
Baseline (5)1
Baseline (6)1
Baseline (6.6)1
Baseline (7)1
End of Treatment (5)1
End of Treatment (5.5)2
PrimaryBaseline to End of Treatment in Urine Color Urinalysis Assessment

Clinical safety laboratory urine color urinalysis assessment.

Time frame:
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.
Reported as:
Count of participants · Participants
Baseline to End of Treatment in Urine Color Urinalysis Assessment
ParticipantsCX-8998
Baseline (Yellow)4
End of Treatment (Yellow)2
PrimaryNumber (%) of Participants Who Did Not Complete The Study Due to Treatment-Emergent Adverse Events

Treatment-emergent adverse events are all adverse events occurring during the treatment period or a pretreatment event that worsens in intensity during the treatment period as assessed by CTCAE v4.0.

Time frame:
Baseline (Day 1) up to Day 26 post-dose, or up to 1 year 3 weeks.
Reported as:
Count of participants · Participants
Number (%) of Participants Who Did Not Complete The Study Due to Treatment-Emergent Adverse Events
ParticipantsCX-8998 Days 1 - 2 (4 mg/d)CX-8998 Days 3 - 8 (8 mg/d)CX-8998 Days 9 - 14 (12 mg/d)CX-8998 Days 15 - 20 (16 mg/d)CX-8998 Days 21 - 27 (20 mg/d)
Number (%) of Participants Who Did Not Complete The Study Due to Treatment-Emergent Adverse Events00000
PrimaryNumber (%) of Participants With Adverse Events of Special Interest

An adverse event of special interest is a serious adverse event as defined in Outcome 6. This includes, however is not limited to, increased seizure frequency, new seizure types, worsening of EEG parameters, systemic adverse events based on safety profile as assessed by CTCAE v4.0.

Time frame:
Baseline (Day 1) up to Day 26 post-dose, or up to 1 year 3 weeks.
Reported as:
Count of participants · Participants
Number (%) of Participants With Adverse Events of Special Interest
ParticipantsCX-8998 Days 1 - 2 (4 mg/d)CX-8998 Days 3 - 8 (8 mg/d)CX-8998 Days 9 - 14 (12 mg/d)CX-8998 Days 15 - 20 (16 mg/d)CX-8998 Days 21 - 27 (20 mg/d)
Any Event10001
Seizure10001
PrimaryChange From Baseline to End of Treatment in Respiration Rate
Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · breaths/min
Change From Baseline to End of Treatment in Respiration Rate
breaths/minCX-8998
Change From Baseline to End of Treatment in Respiration Rate0.29 ± 1.380
PrimaryChange From Baseline to End of Treatment in Temperature
Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · celsius
Change From Baseline to End of Treatment in Temperature
celsiusCX-8998
Change From Baseline to End of Treatment in Temperature-0.01 ± 0.430
PrimaryBaseline Weight
Time frame:
Baseline (Day 1)
Reported as:
Mean · kg
Baseline Weight
kgCX-8998
Baseline Weight82.19 ± 15.963
PrimaryChange From Baseline to End of Treatment in Pulse

The change from baseline to end of treatment in participants' pulses was assessed. The changes in recumbent pulse, standing pulse, and the change from recumbent to standing pulse are reported. Change from recumbent to standing pulse was measured by the difference in recumbent pulse change and standing pulse change.

Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · bpm
Change From Baseline to End of Treatment in Pulse
bpmCX-8998
Recumbent-9.71 ± 8.036
Standing-5.57 ± 6.973
Change from Recumbent to Standing4.14 ± 5.956
PrimaryChange From Baseline to End of Treatment in Systolic Blood Pressure

The change from baseline to end of treatment in participants' systolic blood pressure (sbp) was assessed. The changes in recumbent sbp, standing sbp, and the change from recumbent to standing sbp are reported. Change from recumbent to standing sbp was measured by the difference in recumbent sbp change and standing sbp change.

Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · mmHg
Change From Baseline to End of Treatment in Systolic Blood Pressure
mmHgCX-8998
Recumbent-3.29 ± 14.338
Standing-2.86 ± 12.103
Change from Recumbent to Standing0.43 ± 4.962
PrimaryChange From Baseline to End of Treatment in Diastolic Blood Pressure

The change from baseline to end of treatment in participants' diastolic blood pressure (dbp) was assessed. The changes in recumbent dbp, standing dbp, and the change from recumbent to standing dbp are reported. Change from recumbent to standing dbp was measured by the difference in recumbent dbp change and standing dbp change.

Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · mmHg
Change From Baseline to End of Treatment in Diastolic Blood Pressure
mmHgCX-8998
Recumbent-8.14 ± 14.916
Standing1.86 ± 7.267
Change from Recumbent to Standing10.00 ± 8.737
PrimaryChange From Baseline to End of Treatment in QT Interval
Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · msec
Change From Baseline to End of Treatment in QT Interval
msecCX-8998
Change From Baseline to End of Treatment in QT Interval-2.67 ± 12.801
PrimaryChange From Baseline to End of Treatment in QRS Interval
Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · msec
Change From Baseline to End of Treatment in QRS Interval
msecCX-8998
Change From Baseline to End of Treatment in QRS Interval-1.50 ± 2.811
PrimaryChange From Baseline to End of Treatment in PR Interval
Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · msec
Change From Baseline to End of Treatment in PR Interval
msecCX-8998
Change From Baseline to End of Treatment in PR Interval-0.17 ± 7.026
PrimaryChange From Baseline to End of Treatment in Heart Rate
Time frame:
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Reported as:
Mean · beats/min
Change From Baseline to End of Treatment in Heart Rate
beats/minCX-8998
Change From Baseline to End of Treatment in Heart Rate3.67 ± 13.866

Adverse events

Collected over Adverse events (AEs) were collected from Day 1 up to Day 26 post-dose, or up to 1 year 3 weeks.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
CX-8998 Days 1 - 2 (4 mg/d)0/7 (0%)0/7 (0%)2/7 (28.6%)
CX-8998 Days 3 - 8 (8 mg/d)0/5 (0%)0/5 (0%)2/5 (40%)
CX-8998 Days 9 - 14 (12 mg/d)0/6 (0%)0/6 (0%)0/6 (0%)
CX-8998 Days 15 - 20 (16 mg/d)0/6 (0%)0/6 (0%)1/6 (16.7%)
CX-8998 Days 21 - 27 (20 mg/d)0/6 (0%)0/6 (0%)2/6 (33.3%)
Most frequent other events
Most frequent other events
EventCX-8998 Days 1 - 2 (4 mg/d)CX-8998 Days 3 - 8 (8 mg/d)CX-8998 Days 9 - 14 (12 mg/d)CX-8998 Days 15 - 20 (16 mg/d)CX-8998 Days 21 - 27 (20 mg/d)
Dry mouthGastrointestinal disorders0/71/50/60/60/6
NauseaGastrointestinal disorders0/71/50/60/60/6
Euphoric moodPsychiatric disorders0/71/50/60/60/6
PalpitationsCardiac disorders0/70/50/61/60/6
SluggishnessGeneral disorders0/70/50/60/61/6
SeizureNervous system disorders1/70/50/60/61/6
LethargyNervous system disorders0/70/50/60/61/6
Visual impairmentEye disorders1/70/50/60/60/6
HeadacheNervous system disorders1/70/50/60/60/6

Baseline characteristics

Baseline characteristics were assessed using the Intent-to-Treat Population.

Age, Continuous
Age, Continuous(years)CX-8998
Mean31.3 ± 8.67
Sex: Female, Male
Sex: Female, Male(Participants)CX-8998
Female4
Male3
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)CX-8998
Hispanic or Latino7
Not Hispanic or Latino0
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)CX-8998
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American1
White6
More than one race0
Unknown or Not Reported0
08

Study locations

7 sites
  • Arkansas Epilepsy Program
    Little Rock, Arkansas 72205, United States
  • University of Florida
    Tampa, Florida 33612, United States
  • Bluegrass Epilepsy Research, LLC
    Lexington, Kentucky 40504, United States
  • Tufts Medical Center
    Boston, Massachusetts 02111, United States
  • NYU Comprehensive Epilepsy Center
    New York, New York 10016, United States
  • Cincinnati Children's Hospital
    Cincinnati, Ohio 45229, United States
  • Thomas Jefferson University
    Philadelphia, Pennsylvania 19107, United States
09

References and documents

Study documents

  • Study protocol · May 30, 2019
  • Statistical analysis plan · Apr 21, 2020

Documents are hosted by the registry — open the source record to download them.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Feb 2018 to primary completion in Mar 2019, 13 months.

Results were first posted in Sep 2022, 3 years 5 months after primary completion. A results delay was requested in Mar 2020.

Of 43 industry-sponsored phase 2 interventional studies under Epilepsy, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 31. This study's 13 months is below that.

Counting every sponsor, half of 63 studies reached it within 23 months; a quarter within 14 and three quarters within 36.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 2, 2018
First posted
Jan 23, 2018
StartActual
Feb 25, 2018
Primary completionActual
Mar 29, 2019
CompletionActual
Mar 29, 2019
Results delay requested
Mar 29, 2020
Results first posted
Sep 8, 2022
Last update
Sep 8, 2022
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Sep 8, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03406702
Lead sponsor
Jazz Pharmaceuticals
Responsible party
Sponsor
First posted
Jan 23, 2018
Start date
Feb 25, 2018
Primary completion
Mar 29, 2019
Completion
Mar 29, 2019
Results posted
Sep 8, 2022
Last update
Sep 8, 2022

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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