Clinical trial NCT04252586: A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Official 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Rett Syndrome
RTT
Keywords: Cannabidiol
CBD
Epidiolex
GWP42003-P
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: GW Pharmaceuticals Ltd
First posted: 2020-02-05
Enrollment: 21
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

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

Primary outcomes:
measure: Number of Participants With Any Treatment-emergent Adverse Events (TEAEs), Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 16 (Day 767) in the OLE
measure: Number of Participants With Treatment-emergent Clinical Laboratory Parameters From the Baseline at Any Time Post-dose
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Number of Participants With Potentially Clinically Significant Changes in Vital Sign Values of Blood Pressure From the Baseline at Any Time Post-dose
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE
measure: Number of Participants With Clinically Significant Changes in the Vital Sign Values of Pulse Rate From the Baseline at Any Time Post-dose
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 15 (Day 739) in the OLE
measure: Number of Participants With Clinically Significant Percent Change in Body Weight From the Baseline at Any Time Post-dose
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Number of Participants With At Least One Post Open Label Extension Baseline Flagged ECG Result
timeFrame: Visit 4 (Day 29) up to Visit 15 (Day 739) in the OLE
measure: Number of Participants With Any Changes In Menstruation Cycle at Any Time Post-dose
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Number of Participants With Suicidal Ideation or Behavior at the Baseline and at Any Time Post-dose
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Mean Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Levels at End of Treatment
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Number of Participants With Changes in Tanner Staging at the Baseline and at End of Treatment
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
Secondary outcomes:
measure: Mean Change From Baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) Overall Score at End of Treatment
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Mean Clinical Global Impressions-Improvement (CGI-I) Continuous Score at End of Treatment
timeFrame: Visit 14 (Day 729) in the OLE
measure: Mean Clinician Global Impressions - Severity Scale (CGI-S) Continuous Score at End of Treatment
timeFrame: Visit 14 (Day 729) in the OLE
measure: Mean Change From Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Score at End of Treatment
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
measure: Mean Change From Baseline in 9-item Motor Behavioral Assessment (MBA-9) Total Score at End of Treatment
timeFrame: Baseline of randomized controlled trial (RCT) Visit 1 (Day 1) up to Visit 14 (Day 729) in the OLE
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Clinical Trial Site
state: Alabama
city: La Jolla
country: United States
facility: Clinical Trial Site
state: California
city: Aurora
country: United States
facility: Clinical Trial Site
state: Colorado
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Baltimore
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Saint Paul
country: United States
facility: Clinical Trial Site
state: Minnesota
city: Saint Louis
country: United States
facility: Clinical Trial Site
state: Missouri
city: Bronx
country: United States
facility: Clinical Trial Site
state: New York
city: Cincinnati
country: United States
facility: Clinical Trial Site
state: Ohio
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Greenwood
country: United States
facility: Clinical Trial Site
state: South Carolina
city: Nashville
country: United States
facility: Clinical Trial Site
state: Tennessee
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Perth
country: Australia
facility: Clinical Trial Site
city: South Brisbane
country: Australia
facility: Clinical Trial Site
city: Toronto
country: Canada
facility: Clinical Trial Site
city: Vancouver
country: Canada
facility: Clinical Trial Site
city: Genova
country: Italy
facility: Clinical Trial Site
city: Messina
country: Italy
facility: Clinical Trial Site
city: Milano
country: Italy
facility: Clinical Trial Site
city: Rome
country: Italy
facility: Clinical Trial Site
city: Siena
country: Italy
facility: Clinical Trial Site
city: Barcelona
country: Spain
facility: Clinical Trial Site #1
city: Barcelona
country: Spain
facility: Clinical Trial Site #2
city: Madrid
country: Spain
facility: Clinical Trial Site #1
city: Madrid
country: Spain
facility: Clinical Trial Site #2
city: Valencia
country: Spain
facility: Clinical Trial Site
city: Edinburgh
country: United Kingdom
facility: Clinical Trial Site
city: Liverpool
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
Study officials:
References: