A Phase 3 interventional study of GWP42003-P in Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome and Seizure in Participants With Lennox-Gastaut Syndrome, sponsored by Jazz Pharmaceuticals. Terminated at 11 sites in 3 countries. Open to participants aged 1 Month to 23 Months. Per ClinicalTrials.gov, last updated 2025-10-31.
Sponsored by Jazz Pharmaceuticals · Phase 3, Interventional, and Treatment
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants \< 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet syndrome (DS).
The study duration will be up to approximately 62 weeks, including a 4-week screening/baseline period, a 52-week dose optimization treatment period (which includes a fixed 2-week titration period followed by flexible dose optimization), a 10-day taper period, and a safety follow-up period (4 weeks after the end-of-taper visit).
1,848 studies on the registry are indexed under Epilepsy; 422 are open to participants now.
This study's enrollment of 3 is below the median of 50 across 1,239 interventional studies indexed under Epilepsy.
Browse Epilepsy studies →Jazz Pharmaceuticals is the lead sponsor of 173 studies on the registry; 18 are open to participants now.
Of its 42 completed or terminated interventional studies of FDA-regulated products, 32 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Has significantly impaired hepatic function prior to Visit 3, defined as:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The 52-week treatment period includes a fixed 2-week titration schedule followed by flexible dose optimization. Day 1: 5 mg/kg/day (2.5 mg/kg twice daily (b.i.d.)) Day 8: 10 mg/kg/day (5 mg/kg b.i.d.) Day 15 to Week 52: Flexible dosing based on the participant's observed efficacy, safety, and tolerability per the investigator's clinical judgement. Up to a maximum of 20 mg/kg/day (10 mg/kg b.i.d.) for LGS and DS or 25 mg/kg/day (12.5 mg/kg b.i.d.) for TSC, in maximum weekly increments of 5 mg/kg/day (≤ 2.5 mg/kg b.i.d.).
Drug: GWP42003-P
Oral Solution
Also known as: Cannabidiol, Epidiolex, Epidyolex
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From start of treatment to the post-treatment safety follow-up visit, up to 62 weeks
Mean Change From Baseline in Blood Pressure
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in Pulse Rate
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in Respiratory Rate
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in Body Temperature
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in Height
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in Body Weight
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in Heart Rate
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in RR Interval
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in PR Interval
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in QRS Duration
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in QT Interval
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Mean Change From Baseline in QTcB and QTcF
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Number of Participants With a Clinically Significant Change in Laboratory Parameters
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Number of Participants With Emergence of New Types of Seizures
Time frame: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Plasma Concentrations of GWP42003-P and Its Major Metabolites
Time frame: Predose, 3 hours and 6 hours post dose at End of Treatment (Week 52)
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers
Time frame: Day 1 up to Taper Period, up to Week 52
Clinician Global Impression of Severity (CGI/S) Score
The CGIC/S is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores are reported. The severity of impairment in each domain is rated by the clinician in a scale of 1 through 7 where 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill. Higher scores indicate poor clinical outcome.
Time frame: At Day 365 (EOT)
Clinician Global Impression of Change (CGI/C) Score
The CGI/C is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores are reported. The severity of impairment in each domain is rated by the clinician in a scale of 1 through 7 where 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill. Higher scores indicate poor clinical outcome.
Time frame: At Day 365 (EOT)
Number of Treatment Responders
Treatment Responders are defined as participants with ≥ 50% reduction from baseline in caregiver-reported total countable seizures
Time frame: Day 1 up to the taper period, up to Week 52
Number of Participants Who Achieved Seizure-Free Status
Time frame: Week 12, and every 4 weeks thereafter, up to date of withdrawal or Week 24, whichever occurs first
Percentage of Participants Still Receiving GWP42003-P
Time frame: Week 12, and every 4 weeks thereafter, up to date of withdrawal or Week 24, whichever occurs first
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score
The Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) was developed for use in infants and toddlers from 12-months-to-5 years of age and assesses levels of health and well-being. The caregiver will complete the assessment on an electronic device. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Higher scores indicate better clinical outcome.
Time frame: At Day 365 (EOT)
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers
Time frame: Day 1 up to Taper Period, up to Week 52
A total of 3 participants who met all inclusion criteria and no exclusion criteria were enrolled in the study at 3 sites in the United States.
| Milestone | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Started | 2 | 1 |
| Completed | 0 | 0 |
| Not completed | 2 | 1 |
| Withdrew: Physician decision | 2 | 0 |
| Withdrew: Decision by sponsor | 0 | 1 |
| participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 2 | 1 |
| mmHg | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Systolic blood pressure | -3.0 ± 9.90 | 11.0 ± NA |
| Diastolic blood pressure | 13.5 ± 19.09 | -2.0 ± NA |
| beats per minute | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in Pulse Rate | -11.0 ± 1.41 | 11.0 ± NA |
| breaths/minute | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in Respiratory Rate | -10.0 ± 16.97 | 0 ± NA |
| degrees Celsius | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in Body Temperature | -0.05 ± 0.354 | -0.10 ± NA |
| centimeters | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in Height | 1.95 ± 1.344 | 4.20 ± NA |
| kilogram | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in Body Weight | 0.15 ± 0.354 | 0.60 ± NA |
| beats/minute | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in Heart Rate | -10.0 ± NA | 8.0 ± NA |
| millisecond | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in RR Interval | 48.0 ± NA | -45.0 ± NA |
| millisecond | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in PR Interval | 11.0 ± 12.73 | -2.0 ± NA |
| millisecond | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in QRS Duration | 3.0 ± 1.41 | 6.0 ± NA |
| millisecond | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Mean Change From Baseline in QT Interval | 0 ± 8.49 | 0 ± NA |
| millisecond | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| QTcB | -10.0 ± NA | 16.0 ± NA |
| QTcF | -4.0 ± NA | 9.0 ± NA |
| participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Alanine aminotransferase | 0 | 1 |
| Aspartate aminotransferase | 0 | 1 |
| Gamma Glutamyltransferase | 0 | 1 |
| participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Number of Participants With Emergence of New Types of Seizures | 1 | 0 |
| ng/mL | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| CBD (pre-dose) | 55.3 ± 46.3 | 99.8 ± NA |
| CBD (3-hour post-dose) | 456 ± NA | 427 ± NA |
| CBD (6-hour post-dose) | — | 149 ± NA |
| 7-OH-CBD (pre-dose) | 37.8 ± 29.7 | 123 ± NA |
| 7-OH-CBD (3-hour post-dose) | 161 ± NA | 580 ± NA |
| 7-OH-CBD (6-hour post-dose) | — | 292 ± NA |
| 7-COOH-CBD (pre-dose) | 2790 ± 884 | 7460 ± NA |
| 7-COOH-CBD (3-hour post-dose) | 4870 ± NA | 10700 ± NA |
| 7-COOH-CBD (6-hour post-dose) | — | 9680 ± NA |
| participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Day 1-29: > 25% (increase) | 2 | 0 |
| Day 1-29: >= 0% to <=25% (increase) | 0 | 0 |
| Day 1-29: > -25% to 0% (reduction) | 0 | 0 |
| Day 1-29: <= -25% to > -50% change (reduction) | 0 | 1 |
| Day 1-29: <= -50% to > -75% change (reduction) | 0 | 0 |
| Day 1-29: <= -75% (reduction) | 0 | 0 |
| Day 30-57: > 25% (increase) | 0 | 0 |
| Day 30-57: >= 0% to <=25% (increase) | 1 | 0 |
| Day 30-57: > -25% to 0% (reduction) | 0 | 0 |
| Day 30-57: <= -25% to > -50% change (reduction) | 0 | 0 |
| Day 30-57: <= -50% to > -75% change (reduction) | 0 | 1 |
| Day 30-57: <= -75% (reduction) | 0 | 0 |
| Day 58-85: > 25% (increase) | 0 | 0 |
| Day 58-85: >= 0% to <=25% (increase) | 1 | 0 |
| Day 58-85: > -25% to 0% (reduction) | 0 | 0 |
| Day 58-85: <= -25% to > -50% change (reduction) | 0 | 0 |
| Day 58-85: <= -50% to > -75% change (reduction) | 0 | 0 |
| Day 58-85: <= -75% (reduction) | 0 | 1 |
| Day 86-113: > 25% (increase) | 0 | 0 |
| Day 86-113: >= 0% to <=25% (increase) | 0 | 0 |
| Day 86-113: > -25% to 0% (reduction) | 0 | 0 |
| Day 86-113: <= -25% to > -50% change (reduction) | 0 | 0 |
| Day 86-113: <= -50% to > -75% change (reduction) | 0 | 1 |
| Day 86-113: <= -75% (reduction) | 1 | 0 |
| Day 114-141: > 25% (increase) | 0 | 0 |
| Day 114-141: >= 0% to <=25% (increase) | 0 | 0 |
| Day 114-141: > -25% to 0% (reduction) | 0 | 0 |
| Day 114-141: <= -25% to > -50% change (reduction) | 0 | 0 |
| Day 114-141: <= -50% to > -75% change (reduction) | 0 | 1 |
| Day 114-141: <= -75% (reduction) | 1 | 0 |
| Day 142-169: > 25% (increase) | 0 | 0 |
| Day 142-169: >= 0% to <=25% (increase) | 0 | 0 |
| Day 142-169: > -25% to 0% (reduction) | 0 | 0 |
| Day 142-169: <= -25% to > -50% change (reduction) | 0 | 0 |
| Day 142-169: <= -50% to > -75% change (reduction) | 0 | 0 |
| Day 142-169: <= -75% (reduction) | 1 | 0 |
| Day 170-197: > 25% (increase) | 0 | 0 |
| Day 170-197: >= 0% to <=25% (increase) | 0 | 0 |
| Day 170-197: > -25% to 0% (reduction) | 0 | 0 |
| Day 170-197: <= -25% to > -50% change (reduction) | 0 | 0 |
| Day 170-197: <= -50% to > -75% change (reduction) | 0 | 0 |
| Day 170-197: <= -75% (reduction) | 1 | 0 |
| Taper Period: > 25% (increase) | 1 | 0 |
| Taper Period: >= 0% to <=25% (increase) | 0 | 0 |
| Taper Period: > -25% to 0% (reduction) | 0 | 0 |
| Taper Period: <= -25% to > -50% change (reduction) | 0 | 0 |
| Taper Period: <= -50% to > -75% change (reduction) | 0 | 0 |
| Taper Period: <= -75% (reduction) | 0 | 0 |
The CGIC/S is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores are reported. The severity of impairment in each domain is rated by the clinician in a scale of 1 through 7 where 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill. Higher scores indicate poor clinical outcome.
| score on a scale | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Sensory | — | 3.0 ± NA |
| Motor | — | 3.0 ± NA |
| Cognition | — | 4.0 ± NA |
| Emotional/Behavioral | — | 3.0 ± NA |
| Communication | — | 4.0 ± NA |
| Social | — | 1.0 ± NA |
| Adaptive Functioning | — | 1.0 ± NA |
The CGI/C is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores are reported. The severity of impairment in each domain is rated by the clinician in a scale of 1 through 7 where 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill. Higher scores indicate poor clinical outcome.
| score on a scale | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Sensory | — | 3.0 ± NA |
| Motor | — | 3.0 ± NA |
| Cognition | — | 3.0 ± NA |
| Emotional/Behavioral | — | 3.0 ± NA |
| Communication | — | 4.0 ± NA |
| Social | — | 3.0 ± NA |
| Adaptive Functioning | — | 4.0 ± NA |
The Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) was developed for use in infants and toddlers from 12-months-to-5 years of age and assesses levels of health and well-being. The caregiver will complete the assessment on an electronic device. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Higher scores indicate better clinical outcome.
| score on a scale | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Bodily Pain/Discomfort Zone | 0 ± NA | 12.50 ± NA |
| Combined Behavior Scale | 17.90 ± NA | 0 ± NA |
| Change in Health Score | 0 ± NA | 1.0 ± NA |
| Family Cohesion Score | -55.0 ± NA | 15.0 ± NA |
| Growth and Development Score | 5.0 ± NA | -15.0 ± NA |
| General Health Perceptions Score | -20.0 ± NA | -20.0 ± NA |
| Overall Health Score | 25.0 ± NA | 0 ± NA |
| Physical Abilities Score | 11.10 ± NA | -5.60 ± NA |
| Parental Impact-Emotional Score | -25.00 ± NA | 12.50 ± NA |
| Parental Impact-Time Score | 0 ± NA | 8.30 ± NA |
| Temperament and Moods Score | -8.30 ± NA | 4.20 ± NA |
Treatment Responders are defined as participants with ≥ 50% reduction from baseline in caregiver-reported total countable seizures
| participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Day 1-29 | 0 | 0 |
| Day 30-57 | 0 | 1 |
| Day 58-85 | 0 | 1 |
| Day 86-113 | 1 | 1 |
| Day 114-141 | 1 | 1 |
| Day 142-169 | 1 | — |
| Day 170-197 | 1 | — |
| Taper Period | 0 | — |
| seizure frequency | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Day 1-29 | 56.05 ± 26.23 | -67.80 ± NA |
| Day 30-57 | 24.80 ± NA | -89.50 ± NA |
| Day 58-85 | 2.70 ± NA | -94.70 ± NA |
| Day 86-113 | -95.20 ± NA | -80.20 ± NA |
| Day 114-141 | -97.60 ± NA | -85.80 ± NA |
| Day 142-169 | -97.80 ± NA | — |
| Day 170-197 | -88.10 ± NA | — |
| Taper Period | 71.90 ± NA | — |
| participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Week 12 | 0 | 0 |
| Week 16 | 0 | 0 |
| Week 20 | 0 | 0 |
| Week 24 | 0 | — |
| percentage of participants | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Week 12 | 50 | 100 |
| Week 16 | 50 | 100 |
| Week 20 | 50 | 100 |
| Week 24 | 50 | 0 |
Collected over Adverse events were collected from baseline up to 62 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Tuberous Sclerosis Complex | 0/2 (0%) | 2/2 (100%) | 1/2 (50%) |
| Dravet Syndrome | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Event | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| Change in seizure presentationNervous system disorders | 2/2 | 0/1 |
| Respiratory syncytial virus bronchiolitisInfections and infestations | 1/2 | 0/1 |
| Event | Tuberous Sclerosis Complex | Dravet Syndrome |
|---|---|---|
| FatigueGeneral disorders | 0/2 | 1/1 |
| Rhinovirus infectionInfections and infestations | 0/2 | 1/1 |
| ContusionInjury, poisoning and procedural complications | 0/2 | 1/1 |
| Alanine aminotransferase increasedInvestigations | 0/2 | 1/1 |
| Aspartate aminotransferase increasedInvestigations | 0/2 | 1/1 |
| Gamma-glutamyltransferase increasedInvestigations | 0/2 | 1/1 |
| IrritabilityPsychiatric disorders | 0/2 | 1/1 |
| ProteinuriaRenal and urinary disorders | 0/2 | 1/1 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/2 | 1/1 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 0/2 | 1/1 |
Demographic characteristics were assessed in the Safety Analysis Set.
| Age, Categorical(Participants) | Tuberous Sclerosis Complex | Dravet Syndrome | Total |
|---|---|---|---|
| <=18 years | 2 | 1 | 3 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Tuberous Sclerosis Complex | Dravet Syndrome | Total |
|---|---|---|---|
| Female | 1 | 0 | 1 |
| Male | 1 | 1 | 2 |
| Race (NIH/OMB)(Participants) | Tuberous Sclerosis Complex | Dravet Syndrome | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 2 | 1 | 3 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — In accordance with ICMJE requirements, Jazz Pharmaceuticals may provide qualified external researchers access to individual participant data (IPD) and clinical trial data that underlie the results of this trial upon request. Qualified researchers can submit a request on https://www.jazzpharma.com/science/clinical-trial-data-sharing/ as outlined. Jazz Pharmaceuticals reserves the right not to consider a request. For inquiries about Jazz's data sharing policy contact clinicaldatasharing@jazzpharma.com.
Data sharing details ↗ · www.jazzpharma.com
Ran from May 2021 to primary completion in Jan 2025, 44 months.
Results were first posted in Oct 2025, 9 months after primary completion.
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Studies of the drug tested here: Cannabidiol