Trial · NCT03848832
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
NCT03848832, Phase 3, Terminated, Jazz Pharmaceuticals; results first posted 2021-12-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome. Trials, record NCT03848832. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03848832
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
- NCT number
- NCT03848832
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 29 participants, actual
- Lead sponsor
- Jazz Pharmaceuticals
- Conditions
- Rett Syndrome, RTT
- Interventions
- GWP42003-P, Placebo
- First posted
- 2019-02-21
- Last updated
- 2022-09-02
- Other registries' IDs
- eudract:2018-003370-27
- Results first posted
- 2021-12-29
- Limitations and caveats
This study was terminated early due to enrollment challenges and the Coronavirus disease-2019 (COVID-19) pandemic. Numbers of participants in each treatment groups were small, which precluded the planned formal statistical inferences and limited data interpretation.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Baseline; Week 24 | RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). The total summed score ranges from 0 to 90, with higher scores representing greater severity. |
| Secondary | Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Baseline; Week 24 | RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). The total summed score ranges from 0 to 90, with higher scores represent greater severity. |
| Secondary | Mean Clinical Global Impressions - Improvement (CGI-I) Score at Week 24 | Baseline; Week 24 | CGI-I is a 7-point scale that requires the clinician to assess how much a participant's illness has improved or worsened relative to a Baseline state at the beginning of the intervention. This is rated as: 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; or 7 = very much worse. |
| Secondary | Change From Baseline in Mean Clinician Global Impressions - Severity (CGI-S) Score at Week 24 | Baseline; Week 24 | CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment relative to the clinician's experience with participants who had the same diagnosis. This is rated as: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; or 7 = extremely ill. |
| Secondary | Change From Baseline in Mean RSBQ Subscale Scores at Week 24 | Baseline; Week 24 | RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics in individuals with Rett Syndrome. The 45-item RSBQ is comprised of 8 subscales: 1) general mood (score range 0-16), 2) breathing problems (range 0-10), 3) hand behaviors (range 0-12), 4) face movements (range 0-8), 5) body rocking (BR)/expressionless face (range 0-12), 6) night-time behaviors (range 0-6), 7) anxiety/fear (range 0-8), 8) walking/standing (range 0-4). Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). Higher scores representing greater severity. CFB = Change from Baseline. |
| Secondary | Change From Baseline in Mean 9-items Motor Behavioral Assessment (MBA-9) Total Score and Subscale Scores at Week 24 | Baseline; Week 24 | MBA-9 is derived from the full MBA scale (37 Rett syndrome symptoms) by selecting the items deemed amenable to change and which reflected areas of meaningful clinical change. the MBA-9 includes 9 items (1-Regression of motor skills; 2-Poor eye/social contact; 3-Lack of sustained interest; 4-Does not reach for objects or people; 5-Chewing difficulties; 6-Speech disturbance; 7-Hand clumsiness; 8-Dystonia and 9-Hypertonia/rigidity); for each item, the severity of current symptoms is rated by the investigator on a 5-point numerical scale ranging from 0 to 4 with higher scores representing greater severity (0 = normal or never; 1 = mild or rare; 2 = moderate or occasional; 3 = marked or frequent; 4 = very severe or constant). Total MBA-9 score was calculated by summing the scores of 9 different subscale items. The total summed score ranges from 0 to 36, with higher scores representing greater severity. CFB = Change from Baseline. |
| Secondary | Change From Baseline in Mean Children's Sleep Habits Questionnaire (CSHQ) Total Score and Subscale Scores at Week 24 | Baseline; Week 24 | CSHQ is a caregiver-completed sleep screening instrument designed for school-aged children; which includes 33 items within 8 subscales: 1) bedtime resistance (score range 6-18), 2) sleep onset delay (range 1-3), 3) sleep duration (range 3-9), 4) sleep anxiety (range 4-12), 5) night wakings (range 3-9), 6) parasomnias (range 7-21), 7) sleep-disordered breathing (range 3-9), 8) daytime sleepiness (range 8-24). Item scores range from 1 to 3, where 3="usually" (≥5 times/week), 2="sometimes" (2-4 times/week), and 1="rarely" (≤1 time/week); for items 31 and 32; 3=fall asleep, 2=very sleepy, 1=not sleepy. In general, a score of 3 indicates greater severity, however, 6 items (1-Goes to bed at same time; 2-Falls asleep in 20 minutes; 3-Falls asleep in own bed; 10-Sleeps the right amount; 11-Sleeps same amount each day; 26-Wakes by himself) are reverse scored. Total summed score ranges from 33 to 99, with higher scores representing more disturbed sleep behavior. CFB = Change from Baseline. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Baseline; Week 24 | Intent-to-Treat (ITT) Set: All randomized participants who received at least 1 dose of drug in the trial, and had Baseline efficacy data. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Baseline; Week 24 | ITT Set. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
| SECONDARY | Mean Clinical Global Impressions - Improvement (CGI-I) Score at Week 24 | Baseline; Week 24 | ITT Set. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean Clinician Global Impressions - Severity (CGI-S) Score at Week 24 | Baseline; Week 24 | ITT Set. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean RSBQ Subscale Scores at Week 24 | Baseline; Week 24 | ITT Set. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean 9-items Motor Behavioral Assessment (MBA-9) Total Score and Subscale Scores at Week 24 | Baseline; Week 24 | ITT Set. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Mean Children's Sleep Habits Questionnaire (CSHQ) Total Score and Subscale Scores at Week 24 | Baseline; Week 24 | ITT Set. Participants with non-missing data were included for analysis. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group179
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | 15 mg/kg/Day GWP42003-P | Baseline | 45.9 | Standard Deviation 16.77 | units on a scale |
| Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Placebo | Baseline | 50.0 | Standard Deviation 7.56 | units on a scale |
| Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | 15 mg/kg/Day GWP42003-P | Change from Baseline at Week 24 | -12.1 | Standard Deviation 13.63 | units on a scale |
| Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Placebo | Change from Baseline at Week 24 | -6.1 | Standard Deviation 7.22 | units on a scale |
| Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | 5 mg/kg/Day GWP42003-P | Baseline | 39.8 | Standard Deviation 12.80 | units on a scale |
| Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Placebo | Baseline | 50.0 | Standard Deviation 7.56 | units on a scale |
| Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | 5 mg/kg/Day GWP42003-P | Change from Baseline at Week 24 | 0.4 | Standard Deviation 12.51 | units on a scale |
| Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo | Placebo | Change from Baseline at Week 24 | -6.1 | Standard Deviation 7.22 | units on a scale |
| Mean Clinical Global Impressions - Improvement (CGI-I) Score at Week 24 | 5 mg/kg/Day GWP42003-P | 3.2 | Standard Deviation 1.20 | units on a scale | |
| Mean Clinical Global Impressions - Improvement (CGI-I) Score at Week 24 | 15 mg/kg/Day GWP42003-P | 2.9 | Standard Deviation 1.07 | units on a scale |
1 to 10 of 179Source ClinicalTrials.gov
See all 179 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 5 mg/kg/Day GWP42003-P | 10 | |
| Overall Study | STARTED | 15 mg/kg/Day GWP42003-P | 9 | |
| Overall Study | STARTED | Placebo | 10 | |
| Overall Study | COMPLETED | 5 mg/kg/Day GWP42003-P | 7 | |
| Overall Study | COMPLETED | 15 mg/kg/Day GWP42003-P | 6 | |
| Overall Study | COMPLETED | Placebo | 4 | |
| Overall Study | NOT COMPLETED | 5 mg/kg/Day GWP42003-P | 3 | |
| Overall Study | NOT COMPLETED | 15 mg/kg/Day GWP42003-P | 3 | |
| Overall Study | NOT COMPLETED | Placebo | 6 |
Source ClinicalTrials.gov
Adverse events189
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 | Events |
|---|---|---|---|---|---|---|
| other | Coagulopathy | Blood and lymphatic system disorders | 5 mg/kg/Day GWP42003-P | 1 | 11 | 1 |
| other | Coagulopathy | Blood and lymphatic system disorders | 15 mg/kg/Day GWP42003-P | 0 | 9 | 0 |
| other | Coagulopathy | Blood and lymphatic system disorders | Placebo | 0 | 9 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | 5 mg/kg/Day GWP42003-P | 0 | 11 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | 15 mg/kg/Day GWP42003-P | 0 | 9 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | Placebo | 1 | 9 | 1 |
| other | Macrocytosis | Blood and lymphatic system disorders | 5 mg/kg/Day GWP42003-P | 1 | 11 | 1 |
| other | Macrocytosis | Blood and lymphatic system disorders | 15 mg/kg/Day GWP42003-P | 0 | 9 | 0 |
| other | Macrocytosis | Blood and lymphatic system disorders | Placebo | 1 | 9 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | 5 mg/kg/Day GWP42003-P | 1 | 11 | 1 |
1 to 10 of 189Source ClinicalTrials.gov
See all 189 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 5 mg/kg/Day GWP42003-P | 0 | 11 |
| Total, all-cause mortality | 15 mg/kg/Day GWP42003-P | 0 | 9 |
| Total, all-cause mortality | Placebo | 0 | 9 |
| Total, other adverse events | 5 mg/kg/Day GWP42003-P | 9 | 11 |
| Total, other adverse events | 15 mg/kg/Day GWP42003-P | 7 | 9 |
| Total, other adverse events | Placebo | 7 | 9 |
| Total, serious adverse events | 5 mg/kg/Day GWP42003-P | 2 | 11 |
| Total, serious adverse events | 15 mg/kg/Day GWP42003-P | 2 | 9 |
| Total, serious adverse events | Placebo | 1 | 9 |
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.
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03848832
GET https://conductscience.org/api/trials/v1/NCT03848832/results?page=2