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TerminatedNCT04485104Updated Oct 31, 2025Results posted

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

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

Why this study was terminated
The study was terminated by the Sponsor due to low patient enrollment.
Phase
Phase 3
Study type
Interventional
Enrollment
3
Allocation
Not applicable
Ages
1 Month to 23 Months
Sex
All
01

Study summary

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).

Read the detailed description

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).

02

Conditions studied

  • Seizure in Participants With Tuberous Sclerosis Complex
  • Seizure in Participants With Dravet Syndrome
  • Seizure in Participants With Lennox-Gastaut Syndrome

Keywords

  • Epilepsy
  • Seizures
  • Infantile Spasms
  • Pediatric
  • Children
  • Infants
  • Cannabidiol oral solution
  • GWP42003-P
  • Tuberous Sclerosis Complex
  • TSC
  • Tuberous Sclerosis
  • Cannabidiol
  • Epidiolex
  • CBD
  • Seizure
  • Child
  • TSC1
  • TSC2
  • Tuberous Sclerosis 1
  • Tuberous Sclerosis 2
  • Lennox-Gastaut Syndrome
  • Dravet Syndrome
03

Who can participate

Ages eligible
1 Month to 23 Months
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • Participants with TSC (1 month to \< 2 years of age), or DS (1 year to \< 2 years of age), or LGS (1 year to \< 2 years of age) within the specified age range at the time of initial informed consent.
  • Parent(s)/legal representative is/are willing and able to give informed consent for participation in the study.
  • Parent(s)/legal representative is/are willing and able (in the investigator's opinion) to comply with all study requirements (including accurate electronic participant-reported outcome [ePRO] diary completion).
  • Participants with TSC must have a diagnosis per the 2012 International Tuberous Sclerosis Complex Consensus Conference. Participants with LGS or DS must have a diagnosis that is consistent with International League Against Epilepsy (ILAE) guidelines and confirmed by the Epilepsy Study Consortium (ESCI).
  • Participants who have uncontrolled seizures, and who are currently receiving 1 or more antiseizure medication (ASMs).
  • A suitable VEEG, as available in the medical record, within 1 year of Visit 1. When a historical VEEG is not available, and if clinically indicated and appropriate (due to uncertainties or new seizures), a VEEG will be completed and read to confirm diagnosis prior to Visit 3. All VEEGs are to be read at baseline by the investigator and by an independent reviewer.
  • Has seizures which are not adequately controlled through their current ASMs, defined as ≥ 1 seizure reported on the seizure diary during the screening/baseline period

Key Exclusion Criteria:

  • Has tumor growth which, in the opinion of the investigator, could affect participant safety.
  • Has clinically significant abnormal laboratory values, in the investigator's opinion, at screening/baseline.
  • Has clinically significant abnormalities in the electrocardiogram (ECG) measured at screening/baseline.
  • Has any concurrent cardiovascular conditions, that will, in the investigator's opinion, interfere with the ability to assess their ECGs.
  • Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention such as sesame seed oil.

  • Has significantly impaired hepatic function prior to Visit 3, defined as:

    • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) and (total bilirubin [TBL] > 2 × ULN or international normalized ratio [INR] > 1.5).
    • Serum ALT or AST > 5 × ULN.
    • Serum ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (> 5%).
    • Elevated ALT or AST should be discussed with the medical monitor prior to Visit 3; the medical monitor may allow for a confirmatory re-draw prior to Visit 3.
  • Has received another study intervention within 4 weeks prior to Visit 1 or plans to take another study intervention during the study.
  • Has any other clinically significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the study, may influence the result of the study, or may affect the participant's ability to take part in the study.
  • Any clinically significant abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the study.
  • Has previously been enrolled into this study.
  • Has plans to travel outside their country of residence during the study, unless the participant has confirmation that the study intervention is permitted in the destination country.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
3 participants (actual)

Study arms

  • Experimental
    GWP42003-P

    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

Interventions

  • DrugGWP42003-P

    Oral Solution

    Also known as: Cannabidiol, Epidiolex, Epidyolex

05

What researchers measure

Primary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. 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

  11. 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

  12. 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

  13. 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

  14. 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

  15. 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

  16. 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)

  17. 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

  18. 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)

  19. 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)

Secondary outcomes

  1. 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

  2. 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

  3. 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

Other outcomes

  1. 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)

  2. 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

06

Results

Posted Oct 31, 2025
Limitations and caveats
Three participants were enrolled. There was not enough information to answer the research questions. The limited data collected were not enough to provide meaningful conclusions regarding the efficacy, safety, and PK of CBD-OS in this clinical trial.

Participant flow

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.

Participant flow — Overall Study
MilestoneTuberous Sclerosis ComplexDravet Syndrome
Started21
Completed00
Not completed21
Withdrew: Physician decision20
Withdrew: Decision by sponsor01

Outcome measures

PrimaryNumber 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
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
participantsTuberous Sclerosis ComplexDravet Syndrome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)21
PrimaryMean 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
Reported as:
Mean · mmHg
Mean Change From Baseline in Blood Pressure
mmHgTuberous Sclerosis ComplexDravet Syndrome
Systolic blood pressure-3.0 ± 9.9011.0 ± NA
Diastolic blood pressure13.5 ± 19.09-2.0 ± NA
PrimaryMean 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
Reported as:
Mean · beats per minute
Mean Change From Baseline in Pulse Rate
beats per minuteTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in Pulse Rate-11.0 ± 1.4111.0 ± NA
PrimaryMean 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
Reported as:
Mean · breaths/minute
Mean Change From Baseline in Respiratory Rate
breaths/minuteTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in Respiratory Rate-10.0 ± 16.970 ± NA
PrimaryMean 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
Reported as:
Mean · degrees Celsius
Mean Change From Baseline in Body Temperature
degrees CelsiusTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in Body Temperature-0.05 ± 0.354-0.10 ± NA
PrimaryMean Change From Baseline in Height
Time frame:
From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks
Reported as:
Mean · centimeters
Mean Change From Baseline in Height
centimetersTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in Height1.95 ± 1.3444.20 ± NA
PrimaryMean 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
Reported as:
Mean · kilogram
Mean Change From Baseline in Body Weight
kilogramTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in Body Weight0.15 ± 0.3540.60 ± NA
PrimaryMean 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
Reported as:
Mean · beats/minute
Mean Change From Baseline in Heart Rate
beats/minuteTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in Heart Rate-10.0 ± NA8.0 ± NA
PrimaryMean 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
Reported as:
Mean · millisecond
Mean Change From Baseline in RR Interval
millisecondTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in RR Interval48.0 ± NA-45.0 ± NA
PrimaryMean 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
Reported as:
Mean · millisecond
Mean Change From Baseline in PR Interval
millisecondTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in PR Interval11.0 ± 12.73-2.0 ± NA
PrimaryMean 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
Reported as:
Mean · millisecond
Mean Change From Baseline in QRS Duration
millisecondTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in QRS Duration3.0 ± 1.416.0 ± NA
PrimaryMean 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
Reported as:
Mean · millisecond
Mean Change From Baseline in QT Interval
millisecondTuberous Sclerosis ComplexDravet Syndrome
Mean Change From Baseline in QT Interval0 ± 8.490 ± NA
PrimaryMean 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
Reported as:
Mean · millisecond
Mean Change From Baseline in QTcB and QTcF
millisecondTuberous Sclerosis ComplexDravet Syndrome
QTcB-10.0 ± NA16.0 ± NA
QTcF-4.0 ± NA9.0 ± NA
PrimaryNumber 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
Reported as:
Number · participants
Number of Participants With a Clinically Significant Change in Laboratory Parameters
participantsTuberous Sclerosis ComplexDravet Syndrome
Alanine aminotransferase01
Aspartate aminotransferase01
Gamma Glutamyltransferase01
PrimaryNumber 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
Reported as:
Number · participants
Number of Participants With Emergence of New Types of Seizures
participantsTuberous Sclerosis ComplexDravet Syndrome
Number of Participants With Emergence of New Types of Seizures10
PrimaryPlasma Concentrations of GWP42003-P and Its Major Metabolites
Time frame:
Predose, 3 hours and 6 hours post dose at End of Treatment (Week 52)
Reported as:
Mean · ng/mL
Plasma Concentrations of GWP42003-P and Its Major Metabolites
ng/mLTuberous Sclerosis ComplexDravet Syndrome
CBD (pre-dose)55.3 ± 46.399.8 ± NA
CBD (3-hour post-dose)456 ± NA427 ± NA
CBD (6-hour post-dose)—149 ± NA
7-OH-CBD (pre-dose)37.8 ± 29.7123 ± NA
7-OH-CBD (3-hour post-dose)161 ± NA580 ± NA
7-OH-CBD (6-hour post-dose)—292 ± NA
7-COOH-CBD (pre-dose)2790 ± 8847460 ± NA
7-COOH-CBD (3-hour post-dose)4870 ± NA10700 ± NA
7-COOH-CBD (6-hour post-dose)—9680 ± NA
PrimaryNumber 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
Reported as:
Number · participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers
participantsTuberous Sclerosis ComplexDravet Syndrome
Day 1-29: > 25% (increase)20
Day 1-29: >= 0% to <=25% (increase)00
Day 1-29: > -25% to 0% (reduction)00
Day 1-29: <= -25% to > -50% change (reduction)01
Day 1-29: <= -50% to > -75% change (reduction)00
Day 1-29: <= -75% (reduction)00
Day 30-57: > 25% (increase)00
Day 30-57: >= 0% to <=25% (increase)10
Day 30-57: > -25% to 0% (reduction)00
Day 30-57: <= -25% to > -50% change (reduction)00
Day 30-57: <= -50% to > -75% change (reduction)01
Day 30-57: <= -75% (reduction)00
Day 58-85: > 25% (increase)00
Day 58-85: >= 0% to <=25% (increase)10
Day 58-85: > -25% to 0% (reduction)00
Day 58-85: <= -25% to > -50% change (reduction)00
Day 58-85: <= -50% to > -75% change (reduction)00
Day 58-85: <= -75% (reduction)01
Day 86-113: > 25% (increase)00
Day 86-113: >= 0% to <=25% (increase)00
Day 86-113: > -25% to 0% (reduction)00
Day 86-113: <= -25% to > -50% change (reduction)00
Day 86-113: <= -50% to > -75% change (reduction)01
Day 86-113: <= -75% (reduction)10
Day 114-141: > 25% (increase)00
Day 114-141: >= 0% to <=25% (increase)00
Day 114-141: > -25% to 0% (reduction)00
Day 114-141: <= -25% to > -50% change (reduction)00
Day 114-141: <= -50% to > -75% change (reduction)01
Day 114-141: <= -75% (reduction)10
Day 142-169: > 25% (increase)00
Day 142-169: >= 0% to <=25% (increase)00
Day 142-169: > -25% to 0% (reduction)00
Day 142-169: <= -25% to > -50% change (reduction)00
Day 142-169: <= -50% to > -75% change (reduction)00
Day 142-169: <= -75% (reduction)10
Day 170-197: > 25% (increase)00
Day 170-197: >= 0% to <=25% (increase)00
Day 170-197: > -25% to 0% (reduction)00
Day 170-197: <= -25% to > -50% change (reduction)00
Day 170-197: <= -50% to > -75% change (reduction)00
Day 170-197: <= -75% (reduction)10
Taper Period: > 25% (increase)10
Taper Period: >= 0% to <=25% (increase)00
Taper Period: > -25% to 0% (reduction)00
Taper Period: <= -25% to > -50% change (reduction)00
Taper Period: <= -50% to > -75% change (reduction)00
Taper Period: <= -75% (reduction)00
PrimaryClinician 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)
Reported as:
Mean · score on a scale
Clinician Global Impression of Severity (CGI/S) Score
score on a scaleTuberous Sclerosis ComplexDravet 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
PrimaryClinician 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)
Reported as:
Mean · score on a scale
Clinician Global Impression of Change (CGI/C) Score
score on a scaleTuberous Sclerosis ComplexDravet 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
Other pre-specifiedInfant 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)
Reported as:
Mean · score on a scale
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score
score on a scaleTuberous Sclerosis ComplexDravet Syndrome
Bodily Pain/Discomfort Zone0 ± NA12.50 ± NA
Combined Behavior Scale17.90 ± NA0 ± NA
Change in Health Score0 ± NA1.0 ± NA
Family Cohesion Score-55.0 ± NA15.0 ± NA
Growth and Development Score5.0 ± NA-15.0 ± NA
General Health Perceptions Score-20.0 ± NA-20.0 ± NA
Overall Health Score25.0 ± NA0 ± NA
Physical Abilities Score11.10 ± NA-5.60 ± NA
Parental Impact-Emotional Score-25.00 ± NA12.50 ± NA
Parental Impact-Time Score0 ± NA8.30 ± NA
Temperament and Moods Score-8.30 ± NA4.20 ± NA
SecondaryNumber 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
Reported as:
Number · participants
Number of Treatment Responders
participantsTuberous Sclerosis ComplexDravet Syndrome
Day 1-2900
Day 30-5701
Day 58-8501
Day 86-11311
Day 114-14111
Day 142-1691—
Day 170-1971—
Taper Period0—
Other pre-specifiedPercentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers
Time frame:
Day 1 up to Taper Period, up to Week 52
Reported as:
Mean · seizure frequency
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers
seizure frequencyTuberous Sclerosis ComplexDravet Syndrome
Day 1-2956.05 ± 26.23-67.80 ± NA
Day 30-5724.80 ± NA-89.50 ± NA
Day 58-852.70 ± 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 Period71.90 ± NA—
SecondaryNumber 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
Reported as:
Number · participants
Number of Participants Who Achieved Seizure-Free Status
participantsTuberous Sclerosis ComplexDravet Syndrome
Week 1200
Week 1600
Week 2000
Week 240—
SecondaryPercentage 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
Reported as:
Number · percentage of participants
Percentage of Participants Still Receiving GWP42003-P
percentage of participantsTuberous Sclerosis ComplexDravet Syndrome
Week 1250100
Week 1650100
Week 2050100
Week 24500

Adverse events

Collected over Adverse events were collected from baseline up to 62 weeks.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Tuberous Sclerosis Complex0/2 (0%)2/2 (100%)1/2 (50%)
Dravet Syndrome0/1 (0%)0/1 (0%)1/1 (100%)
Most frequent serious events
Most frequent serious events
EventTuberous Sclerosis ComplexDravet Syndrome
Change in seizure presentationNervous system disorders2/20/1
Respiratory syncytial virus bronchiolitisInfections and infestations1/20/1
Most frequent other events
Showing 10 of 16
Most frequent other events
EventTuberous Sclerosis ComplexDravet Syndrome
FatigueGeneral disorders0/21/1
Rhinovirus infectionInfections and infestations0/21/1
ContusionInjury, poisoning and procedural complications0/21/1
Alanine aminotransferase increasedInvestigations0/21/1
Aspartate aminotransferase increasedInvestigations0/21/1
Gamma-glutamyltransferase increasedInvestigations0/21/1
IrritabilityPsychiatric disorders0/21/1
ProteinuriaRenal and urinary disorders0/21/1
CoughRespiratory, thoracic and mediastinal disorders0/21/1
RhinorrhoeaRespiratory, thoracic and mediastinal disorders0/21/1

Baseline characteristics

Demographic characteristics were assessed in the Safety Analysis Set.

Age, Categorical
Age, Categorical(Participants)Tuberous Sclerosis ComplexDravet SyndromeTotal
<=18 years213
Between 18 and 65 years000
>=65 years000
Sex: Female, Male
Sex: Female, Male(Participants)Tuberous Sclerosis ComplexDravet SyndromeTotal
Female101
Male112
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Tuberous Sclerosis ComplexDravet SyndromeTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American000
White213
More than one race000
Unknown or Not Reported000
07

Study locations

11 sites
  • Clinical Trial Site
    Little Rock, Arkansas 72202, United States
  • Clinical Trial Site
    Los Angeles, California 90095, United States
  • Clinical Trial Site
    Chicago, Illinois 60611, United States
  • Clinical Trial Site
    Boston, Massachusetts 02114, United States
  • Clinical Trial Site
    Cincinnati, Ohio 45229, United States
  • Clinical Trial Site
    Houston, Texas 77030, United States
  • Clinical Trial Site
    Florence, 50139, Italy
  • Clinical Trial Site
    Genova, 16147, Italy
  • Clinical Trial Site
    Rome, 00165, Italy
  • Clinical Trial Site
    Barcelona, 08950, Spain
  • Clinical Trial Site
    Madrid, 28034, Spain
08

References and documents

Study documents

  • Study protocol · Jan 24, 2024
  • Statistical analysis plan · Mar 4, 2025

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

Individual participant data

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.

09

Registry details

Key details

Study ID
NCT04485104
Lead sponsor
Jazz Pharmaceuticals
Collaborators
Jazz Pharmaceuticals Research UK Limited
Responsible party
Sponsor
First posted
Jul 24, 2020
Start date
May 19, 2021
Primary completion
Jan 28, 2025
Completion
Jan 28, 2025
Results posted
Oct 31, 2025
Last update
Oct 31, 2025

Oversight

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

Not currently enrolling

This study is terminated, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.

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