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

Recruitment & Eligibility

Status: A

Sex: Female

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 her parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
• Patient and her 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 she has one) and consultant (if she has one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• 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).
• Patient is 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 thereafter.
• Patient has been previously enrolled and dosed in this trial.
• Patient is unwilling to abstain from donation of blood during the trial.

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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