An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome

Phase 1

Conditions: Rett syndrome (RTT) [typical or atypical]
MedDRA version: 20.0Level: PTClassification code 10077709Term: Rett syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2019-001605-24-GB

Lead Sponsor: GW Research Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 252

Inclusion Criteria

For inclusion in the trial, patients must fulfil all of the following criteria:
• Patient has completed all scheduled visits of the treatment phase of the RCT, GWND18064, and has transitioned to OLE by the point of RCT follow-up (Visit 11).
• Patient (if possessing adequate understanding, in the investigator’s opinion) and/or the patient’s parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
• Patient and the patient’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).
• Patient must have the 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).
• Patient 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.
• Patient and/or parent(s)/legal representative is willing to allow the patient’s primary care practitioner (if the patient has one) and consultant (if the patient has one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.

Exclusion Criteria

The patient may not enter the trial if ANY of the following apply:
• Patient meets the withdrawal criteria (including clinically significant abnormal laboratory values), in the investigator’s opinion.
• Patient met during the RCT the criteria for permanent IMP discontinuation (unless in case of an AE, if AE was not considered related with the IMP; patients 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.
• Patient has been previously enrolled and dosed in this trial.
• Patient 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.

Per Protocol Annex 1, with the exception of the inclusion criterion that requires patients must have completed all scheduled visits of the treatment phase of the RCT, GWND18064, and transitioned to OLE by the point of RCT follow-up (Visit 11), all inclusion and exclusion criteria defined in the GWND19002 protocol must be met. In addition, the following exclusion criteria apply:
• Any history of suicidal behavior or any suicidal ideation in the last month or at Visit 1.
• Patient has clinically relevant abnormalities in the electrocardiogram measured at Visit 1 (including QT interval, corrected by Bazett's correction formula [QTcB] > 450 msec, average of 3 measurements).
• Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP (active and placebo), such as sesame oil.
• Patient has moderately impaired hepatic function at screening, defined as serum ALT or AST > 3 × ULN or total bilirubin [TBL] > 2 × ULN.
This criterion can only be confirmed once the Visit 1 laboratory results are available. If Visit 1 laboratory results indicate the patient is not eligible, the patient must be withdrawn. Prior to withdrawal for the transaminase elevations noted above, the investigator may choose to confirm the transaminase elevations by repeating the following laboratory tests within 24 to 48 hours: ALT, AST, TBL, international normalized ratio, % eosinophils, gamma-glutamyl transferase, alkaline phosphatase, and eosinophils. Should the above transaminase elevation criteria be confirmed, the patient must be withdrawn from the trial.
• Patient has received an IMP (other than GWND18064 IMP) since participation in GWND18064.
• Patient has been taking felbamate for less than 1 year prior to Visit 1
• Patient is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex
® [nabiximols]) or cannabidiol oral solutions (excluding GWND18064 IMP) within the 3

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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