Trial · NCT04252586

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

NCT04252586, Phase 3, Terminated, Jazz Pharmaceuticals; results first posted 2022-08-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome. Trials, record NCT04252586. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04252586

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
NCT number: NCT04252586
Status: Terminated
Phases: Phase 3
Enrollment: 21 participants, actual
Lead sponsor: Jazz Pharmaceuticals
Conditions: Rett Syndrome, RTT
Interventions: GWP42003-P
First posted: 2020-02-05
Last updated: 2022-08-08
Other registries' IDs: eudract:2019-001605-24
Results first posted: 2022-08-08
Limitations and caveats: This trial was terminated due to enrollment challenges and the COVID-19 pandemic. Due to early termination and participants withdrawal, the number of participants was small and the length of treatment was reduced.

Source ClinicalTrials.gov

Registered outcomes15

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 16 (Day 767) in the OLE	Adverse events (AEs) were defined as any new unfavorable/unintended signs/symptoms (including abnormal laboratory findings when relevant) or diagnosis or worsening of a pre-existing condition that occurs during the study. TEAEs were defined as the AEs that started or worsened in severity or seriousness following the first dose of GWP42003-P. A serious AE was defined as any AE that results in any of the following outcomes: death, life-threatening adverse experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or cancer, any other experience that suggests a significant hazard, contraindication, side effect or precaution that may require medical or surgical intervention to prevent one of the outcomes listed above, or an event that changes the risk/benefit ratio of the study.
Primary	Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Treatment-emergent clinical parameters were defined as the following: Treatment-emergent alanine transferase (ALT) \> 3×upper limit of normal (ULN), \> 5×ULN and \> 8×ULN; Treatment-emergent aspartate aminotransferase (AST) \> 3×ULN, \> 5×ULN and \> 8×ULN; Treatment-emergent ALT or AST \> 3×ULN, \> 5×ULN and \> 8×ULN; Treatment-emergent ALT or AST \> 3×ULN and either bilirubin \> 2×ULN or international normalized ratio (INR) \> 1.5.
Primary	Number of Participants With Potentially Clinically Significant Changes in Vital Sign Values of Blood Pressure From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE	Potentially clinically significant vital sign values of blood pressure (BP) were defined as sitting systolic BP (mmHg) change: \< -20 or \> 20 mmHg and sitting diastolic BP change: \< -10 or \> 10 mmHg. Vital sign measurements were taken in a sitting position at rest for 5 minutes. Blood pressure readings were recorded using the same arm throughout the trial, when possible.
Primary	Number of Participants With Clinically Significant Changes in the Vital Sign Values of Pulse Rate From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE	Potentially clinically significant vital sign values of pulse rate were defined as pulse rate change: \< -10 or \> 10 beats per minute. Vital sign measurements were taken in a sitting position at rest for 5 minutes.
Primary	Number of Participants With Clinically Significant Percent Change in Body Weight From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Clinically significant body weight changes were defined as the percent change in body weight (≤7% change or ≥7% change).
Primary	Number of Participants With At Least One Post Open Label Extension Baseline Flagged ECG Result	Visit 4 (Day 29) up to Visit 15 (Day 739) in the OLE	Electrocardiogram assessments were performed for QTcB and QTcF \>450 msec, \>480 msec, and \>500 msec. QTcB = corrected QT interval with Bazette correction. QTcF = QTc corrected by Fridericia.
Primary	Number of Participants With Any Changes In Menstruation Cycle at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Participants were evaluated for any changes to typical menstrual cycles, duration of menstrual cycles, and typical strength of the menstrual cycles during the study.
Primary	Number of Participants With Suicidal Ideation or Behavior at the Baseline and at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Suicidal ideation and behavior was assessed by the investigator via a clinical interview with the caregiver. The questionnaire included following questions: Has the child expressed any wish to be dead?, Has the child made any suicide attempts?, Has the child shown any non-suicidal self-injurious behavior? The responses were recorded as Yes/No.
Primary	Mean Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Levels at End of Treatment	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Blood samples were collected to assess changes in serum IGF-1 levels. A negative mean change from baseline indicates a reduction in IGF-1 levels.
Primary	Number of Participants With Changes in Tanner Staging at the Baseline and at End of Treatment	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Pubic hair growth and breast development of all adolescents were assessed by the investigator or caregiver using Tanner Staging categorization from 1 to 5. Stage 1- No glandular tissue; areola follows skin contours of chest; No pubic hair Stage 2- Breast bud forms; areola begins to widen; a small amount of long, downy hair with slight pigmentation on labia majora Stage 3- Breast begins to become more elevated and extends beyond borders of the areola, which continues to widen but remains in contour with surrounding breast; Hair becomes more coarse and curly and begins to extend laterally Stage 4- Increased breast size and elevation; areola and papilla form a secondary mound projecting from the contour of the surrounding breast; Adult-like hair quality, extending across pubis but sparing medial thighs Stage 5- Breast reaches final adult size; areola returns to the contour of the surrounding breast, with a projecting central papilla; Hair extends to medial surface of the thigh.

1 to 10 of 15Source ClinicalTrials.gov

See all 15 registered outcomes

Outcomes15

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 16 (Day 767) in the OLE	Adverse events were assessed in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Clinical laboratory parameters were assessed in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Changes in Vital Sign Values of Blood Pressure From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE	Vital signs were assessed in participants with available data in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Clinically Significant Changes in the Vital Sign Values of Pulse Rate From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE	Pulse rate was assessed in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Clinically Significant Percent Change in Body Weight From the Baseline at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Body weight was assessed in participants with available data in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With At Least One Post Open Label Extension Baseline Flagged ECG Result	Visit 4 (Day 29) up to Visit 15 (Day 739) in the OLE	Electrocardiogram assessments were performed in participants with available data in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Any Changes In Menstruation Cycle at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Changes in menstruation cycle was assessed in participants with available data in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Suicidal Ideation or Behavior at the Baseline and at Any Time Post-dose	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Suicidal ideation or behavior was assessed in participants with available data in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Mean Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Levels at End of Treatment	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	IGF-1 levels were assessed in participants with available data in the Safety Analysis Set.	units on a scale	MEAN
PRIMARY	Number of Participants With Changes in Tanner Staging at the Baseline and at End of Treatment	Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE	Changes in Tanner staging was assessed in the Safety Analysis Set.	Participants	COUNT_OF_PARTICIPANTS

1 to 10 of 15Source ClinicalTrials.gov

See all 15 outcomes

Results by group189

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	GWP42003-P	Any TEAE	19	Participants
Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	GWP42003-P	Treatment-related TEAEs	7	Participants
Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	GWP42003-P	TEAEs leading to discontinuation	3	Participants
Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	GWP42003-P	Treatment-related TEAE leading to discontinuation	1	Participants
Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	GWP42003-P	Serious TEAEs	5	Participants
Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs	GWP42003-P	Treatment-related serious TEAEs	0	Participants
Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose	GWP42003-P	Treatment-emergent ALT >3xULN	1	Participants
Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose	GWP42003-P	Treatment-emergent ALT >5xULN	0	Participants
Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose	GWP42003-P	Treatment-emergent ALT >8xULN	0	Participants
Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose	GWP42003-P	Treatment-emergent AST >3xULN	0	Participants

1 to 10 of 189Source ClinicalTrials.gov

See all 189 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	GWP42003-P	21
Overall Study	COMPLETED	GWP42003-P	0
Overall Study	NOT COMPLETED	GWP42003-P	21

Source ClinicalTrials.gov

Adverse events27

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Neutropenia	Blood and lymphatic system disorders	GWP42003-P	2	21
other	Diarrhoea	Gastrointestinal disorders	GWP42003-P	4	21
other	Vomiting	Gastrointestinal disorders	GWP42003-P	7	21
other	Pyrexia	General disorders	GWP42003-P	4	21
other	Otitis media	Infections and infestations	GWP42003-P	2	21
other	Upper respiratory tract infection	Infections and infestations	GWP42003-P	3	21
other	Fall	Injury, poisoning and procedural complications	GWP42003-P	2	21
other	Mean cell volume increased	Investigations	GWP42003-P	2	21
other	Monocyte count decreased	Investigations	GWP42003-P	3	21
other	Decreased appetite	Metabolism and nutrition disorders	GWP42003-P	3	21

1 to 10 of 27Source ClinicalTrials.gov

See all 27 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	GWP42003-P	2	21
Total, other adverse events	GWP42003-P	19	21
Total, serious adverse events	GWP42003-P	5	21

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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