An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

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Brief Title

An Open-label Extension 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

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.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Number of participants with any adverse event

Secondary Outcome

 Change from prerandomization Baseline of the Randomized Controlled Trial (RCT) to Week 105 in Rett Syndrome Behaviour Questionnaire (RSBQ) scores

Condition

Rett Syndrome

Intervention

GWP42003-P

Study Arms / Comparison Groups

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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

252

Start Date

February 28, 2020

Completion Date

June 2022

Primary Completion Date

June 2022

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
      

Gender

All

Ages

2 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT04252586

Organization ID

GWND19002

Secondary IDs

2019-001605-24

Responsible Party

Sponsor

Study Sponsor

GW Research Ltd


Study Sponsor

, , 


Verification Date

April 2020