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

NCT ID: NCT04252586

Last Updated: 2022-08-08

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-02-28

Study Completion Date

2021-06-09

Brief Summary

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This study will be conducted to evaluate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in participants with Rett syndrome.

Detailed Description

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Conditions

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Rett Syndrome RTT

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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GWP42003-P

100 milligrams per milliliter (mg/mL) GWP42003-P oral solution, taken twice daily (morning and evening).

Group Type EXPERIMENTAL

GWP42003-P

Intervention Type DRUG

GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil with anhydrous ethanol, sweetener (sucralose), and strawberry flavoring

Interventions

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GWP42003-P

GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil with anhydrous ethanol, sweetener (sucralose), and strawberry flavoring

Intervention Type DRUG

Other Intervention Names

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Cannabidiol CBD Epidiolex CBD-OS

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GW Pharmaceuticals Ltd

INDUSTRY

Sponsor Role collaborator

Jazz Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Trial Site

Birmingham, Alabama, United States

Site Status

Clinical Trial Site

La Jolla, California, United States

Site Status

Clinical Trial Site

Aurora, Colorado, United States

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Clinical Trial Site

Chicago, Illinois, United States

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Clinical Trial Site

Baltimore, Maryland, United States

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Clinical Trial Site

Boston, Massachusetts, United States

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Clinical Trial Site

Saint Paul, Minnesota, United States

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Clinical Trial Site

St Louis, Missouri, United States

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Clinical Trial Site

The Bronx, New York, United States

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Clinical Trial Site

Cincinnati, Ohio, United States

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Clinical Trial Site

Philadelphia, Pennsylvania, United States

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Clinical Trial Site

Greenwood, South Carolina, United States

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Clinical Trial Site

Nashville, Tennessee, United States

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Clinical Trial Site

Houston, Texas, United States

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Clinical Trial Site

Perth, , Australia

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Clinical Trial Site

South Brisbane, , Australia

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Clinical Trial Site

Toronto, , Canada

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Clinical Trial Site

Vancouver, , Canada

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Clinical Trial Site

Genova, , Italy

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Clinical Trial Site

Messina, , Italy

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Clinical Trial Site

Milan, , Italy

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Clinical Trial Site

Rome, , Italy

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Clinical Trial Site

Siena, , Italy

Site Status

Clinical Trial Site #1

Barcelona, , Spain

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Clinical Trial Site #2

Barcelona, , Spain

Site Status

Clinical Trial Site #1

Madrid, , Spain

Site Status

Clinical Trial Site #2

Madrid, , Spain

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Clinical Trial Site

Valencia, , Spain

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Clinical Trial Site

Edinburgh, , United Kingdom

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Clinical Trial Site

Liverpool, , United Kingdom

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Clinical Trial Site

London, , United Kingdom

Site Status

Countries

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United States Australia Canada Italy Spain United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2019-001605-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GWND19002

Identifier Type: -

Identifier Source: org_study_id

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