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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04252586
Registration number
NCT04252586
Ethics application status
Date submitted
30/01/2020
Date registered
5/02/2020
Date last updated
8/08/2022
Titles & IDs
Public title
A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
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Scientific title
An Open-label Extension Trial to Investigate the Long-term Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
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Secondary ID [1]
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2019-001605-24
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Secondary ID [2]
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GWND19002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome
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RTT
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - GWP42003-P
Experimental: GWP42003-P - 100 milligrams per milliliter (mg/mL) GWP42003-P oral solution, taken twice daily (morning and evening).
Treatment: Drugs: GWP42003-P
GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil with anhydrous ethanol, sweetener (sucralose), and strawberry flavoring
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 16 (Day 767) in the OLE
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Primary outcome [2]
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Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Primary outcome [3]
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Number of Participants With Potentially Clinically Significant Changes in Vital Sign Values of Blood Pressure From the Baseline at Any Time Post-dose
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE
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Primary outcome [4]
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Number of Participants With Clinically Significant Changes in the Vital Sign Values of Pulse Rate From the Baseline at Any Time Post-dose
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Assessment method [4]
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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.
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Timepoint [4]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE
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Primary outcome [5]
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Number of Participants With Clinically Significant Percent Change in Body Weight From the Baseline at Any Time Post-dose
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Assessment method [5]
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Clinically significant body weight changes were defined as the percent change in body weight (=7% change or =7% change).
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Timepoint [5]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Primary outcome [6]
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Number of Participants With At Least One Post Open Label Extension Baseline Flagged ECG Result
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Assessment method [6]
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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.
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Timepoint [6]
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Visit 4 (Day 29) up to Visit 15 (Day 739) in the OLE
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Primary outcome [7]
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Number of Participants With Any Changes In Menstruation Cycle at Any Time Post-dose
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Assessment method [7]
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Participants were evaluated for any changes to typical menstrual cycles, duration of menstrual cycles, and typical strength of the menstrual cycles during the study.
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Timepoint [7]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Primary outcome [8]
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Number of Participants With Suicidal Ideation or Behavior at the Baseline and at Any Time Post-dose
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Assessment method [8]
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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.
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Timepoint [8]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Primary outcome [9]
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Mean Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Levels at End of Treatment
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Assessment method [9]
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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.
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Timepoint [9]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Primary outcome [10]
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Number of Participants With Changes in Tanner Staging at the Baseline and at End of Treatment
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Assessment method [10]
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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.
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Timepoint [10]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Secondary outcome [1]
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Mean Change From Baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) Overall Score at End of Treatment
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Assessment method [1]
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RSBQ is a caregiver-completed questionnaire that assesses the overall condition (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. A negative mean change from baseline indicates an improvement in overall condition.
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Timepoint [1]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Secondary outcome [2]
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Mean Clinical Global Impressions-Improvement (CGI-I) Continuous Score at End of Treatment
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Assessment method [2]
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CGI-I is a 7-point scale that requires the clinician to assess whether a patient's condition has improved or worsened relative to a baseline state at the beginning of the intervention. This was 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. The mean continuous CGI-I score (averaged from each treatment enrolled under protocol and under annex) at Visit 14 (Day 729) is being reported. Higher mean scores indicate worse condition.
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Timepoint [2]
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Visit 14 (Day 729) in the OLE
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Secondary outcome [3]
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Mean Clinician Global Impressions - Severity Scale (CGI-S) Continuous Score at End of Treatment
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Assessment method [3]
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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 was 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. The mean continuous CGI-S score (averaged from each treatment enrolled under protocol and under annex) at Visit 14 (Day 729) is being reported. Higher scores indicate more severe disease.
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Timepoint [3]
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Visit 14 (Day 729) in the OLE
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Secondary outcome [4]
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Mean Change From Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Score at End of Treatment
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Assessment method [4]
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CSHQ is a caregiver-completed sleep screening instrument designed for school-aged children; which includes 33 items within 8 subscales:bedtime resistance (score range 6-18), sleep onset delay (range 1-3), sleep duration (range 3-9), sleep anxiety (range 4-12), night wakings (range 3-9), parasomnias (range 7-21), sleep-disordered breathing (range 3-9), daytime sleepiness (range 8-24). The 33 items 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. A score of 3 indicates greater severity; however, 6 items (1-Goes to bed at same time; 3-Falls asleep in own bed; 26-Wakes by himself; 2-Falls asleep in 20 minutes; 10-Sleeps the right amount; 11-Sleeps same amount each day) are reverse scored. The total summed score ranges from 33 to 99, with higher scores indicating disturbed sleep behavior. A negative mean change from baseline indicates improvement in sleep.
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Timepoint [4]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Secondary outcome [5]
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Mean Change From Baseline in 9-item Motor Behavioral Assessment (MBA-9) Total Score at End of Treatment
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Assessment method [5]
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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 (Regression of motor skills; Poor eye/social contact; Lack of sustained interest; Does not reach for objects or people; Chewing difficulties; Speech disturbance; Hand clumsiness; Dystonia; and 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. A negative mean change from baseline indicates improvement in behavior.
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Timepoint [5]
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Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
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Eligibility
Key inclusion criteria
- Participant has completed all scheduled visits of the treatment phase of the
randomized controlled trial (RCT), GWND18064 (NCT03848832), and has transitioned to
open-label extension (OLE) by the point of RCT follow-up
- Participant (if possessing adequate understanding, in the investigator's opinion)
and/or the participant(s)/legal representative is willing and able to give informed
consent/assent for participation in the trial.
- Participant and the participant's caregiver are willing and able (in the
investigator's opinion) to comply with all trial requirements (including the
completion of all caregiver assessments by the same caregiver throughout the trial).
- Ability to swallow the investigational medicinal product (IMP) provided as a liquid
solution, or the ability for the IMP to be delivered via gastrostomy (G) or
nasogastric (NG) feeding tube (only G- or NG-tubes made from polyurethane or silicon
are allowed).
- Participant and/or parent(s)/legal representative is willing to allow the responsible
authorities to be notified of participation in the trial, if mandated by local law.
- Participant and/or parent(s)/legal representative is willing to allow the
participant's primary care practitioner (if the participant has one) and consultant
(if the participant has one) to be notified of participation in the trial, if the
primary care practitioner/consultant is different from the investigator.
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Minimum age
2
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Maximum age
18
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Participant meets the withdrawal criteria (including clinically significant abnormal
laboratory values), in the investigator's opinion.
- Participant met during the RCT the criteria for permanent IMP discontinuation (unless
in the case of an adverse event [AE], if the AE was not considered related with the
IMP; participants that met alanine aminotransferase (ALT)/aspartate aminotransferase
(AST) elevations discontinuation criteria must be excluded).
- Females of childbearing potential, unless willing to ensure that they or their partner
use a highly effective method of birth control (e.g., combined [estrogen and
progestogen containing] hormonal contraception associated with inhibition of ovulation
[oral, intravaginal, or transdermal], progestogen-only hormonal contraception
associated with inhibition of ovulation [oral, injectable, or implantable],
intrauterine devices/hormone-releasing systems, bilateral tubal occlusion,
vasectomized partner, sexual abstinence during the trial and for 3 months after the
last dose
- Participant has been previously enrolled and dosed in this trial.
- Participant is unwilling to abstain from donation of blood during the trial.
- Male participants who are fertile (i.e., after puberty unless permanently sterile by
bilateral orchidectomy) and with a partner of childbearing potential unless agree to
ensure that they use male contraception (e.g., condom) or remain sexually abstinent
during the trial and for 3 months after the last dose
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/02/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
9/06/2021
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Sample size
Target
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Accrual to date
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Final
21
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Trial Site - Perth
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Recruitment hospital [2]
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Clinical Trial Site - South Brisbane
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Recruitment postcode(s) [1]
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- Perth
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Recruitment postcode(s) [2]
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- South Brisbane
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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United States of America
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California
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United States of America
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Colorado
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Illinois
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Maryland
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Massachusetts
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Minnesota
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United States of America
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State/province [8]
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Missouri
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New York
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United States of America
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Ohio
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Pennsylvania
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United States of America
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South Carolina
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Tennessee
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State/province [14]
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Texas
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Canada
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State/province [15]
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Toronto
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Canada
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State/province [16]
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Vancouver
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Italy
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State/province [17]
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Genova
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Country [18]
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Italy
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State/province [18]
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Messina
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Country [19]
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Italy
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State/province [19]
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Milano
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Country [20]
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Italy
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State/province [20]
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Rome
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Italy
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State/province [21]
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Siena
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Valencia
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United Kingdom
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Edinburgh
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United Kingdom
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Liverpool
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United Kingdom
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State/province [27]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Jazz Pharmaceuticals
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Address
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Country
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Other collaborator category [1]
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Other
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Name [1]
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GW Pharmaceuticals
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Address [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
This study will be conducted to evaluate the long-term safety of cannabidiol oral solution
(GWP42003-P, CBD-OS) in participants with Rett syndrome.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04252586
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Contact person for public queries
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04252586
Download to PDF