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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04252586
Recruitment Status : Enrolling by invitation
First Posted : February 5, 2020
Last Update Posted : April 28, 2020
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

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.

Condition or disease Intervention/treatment Phase
Rett Syndrome RTT Drug: GWP42003-P Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 252 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : February 28, 2020
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : June 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: GWP42003-P
100 milligrams per milliliter (mg/mL) GWP42003-P oral solution, taken twice daily (morning and evening).
Drug: GWP42003-P
GWP42003-P presented as an oral solution containing cannabidiol in the excipients sesame oil with anhydrous ethanol, sweetener (sucralose), and strawberry flavoring
Other Names:
  • Cannabidiol
  • CBD
  • Epidiolex
  • CBD-OS




Primary Outcome Measures :
  1. Number of participants with any adverse event [ Time Frame: up to Week 110 ]
  2. Number of participants with any clinically significant clinical laboratory parameter value [ Time Frame: up to Week 106 ]
  3. Number of participants with any clinically significant vital sign value [ Time Frame: up to Week 106 ]
  4. Number of participants with any clinically significant physical examination procedure value [ Time Frame: up to Week 106 ]
  5. Number of participants with any clinically significant 12-lead electrocardiogram (ECG) value [ Time Frame: up to Week 106 ]
  6. Number of participants with any clinically significant effects on their menstruation cycle [ Time Frame: up to Week 105 ]
  7. Number of participants with suicidality, as assessed by the investigator via a clinical interview with the caregiver [ Time Frame: up to Week 106 ]
  8. Number of participants with any change in growth and development by measurement of height, weight, serum insulin-like growth factor-1 (IGF-1) levels, and Tanner Staging [ Time Frame: up to Week 106 ]

Secondary Outcome Measures :
  1. Change from prerandomization Baseline of the Randomized Controlled Trial (RCT) to Week 105 in Rett Syndrome Behaviour Questionnaire (RSBQ) scores [ Time Frame: from Baseline up to Week 105 ]
  2. Change from prerandomization Baseline of the RCT to Week 105 in Clinical Global Impressions - Improvement (CGI-I) scores [ Time Frame: from Baseline up to Week 105 ]
  3. Change from prerandomization Baseline of the RCT to Week 105 in Clinician Global Impressions - Severity Scale (CGI-S) scores [ Time Frame: from Baseline up to Week 105 ]
  4. Change from prerandomization Baseline of the RCT to Week 105 in 9-item Motor Behavioral Assessment (MBA-9) scores [ Time Frame: from Baseline up to Week 105 ]
  5. Change from prerandomization Baseline of the RCT to Week 105 in Children's Sleep Habits Questionnaire (CSHQ) scores [ Time Frame: from Baseline up to Week 105 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04252586


Locations
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United States, Alabama
Clinical Trial Site
Birmingham, Alabama, United States, 35294-0021
United States, California
Clinical Trial Site
La Jolla, California, United States, 92093
United States, Colorado
Clinical Trial Site
Aurora, Colorado, United States, 80045
United States, Illinois
Clinical Trial Site
Chicago, Illinois, United States, 60612
United States, Maryland
Clinical Trial Site
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States, 02115
United States, Minnesota
Clinical Trial Site
Saint Paul, Minnesota, United States, 55101
United States, Missouri
Clinical Trial Site
Saint Louis, Missouri, United States, 63110-1093
United States, New York
Clinical Trial Site
Bronx, New York, United States, 10467
United States, Ohio
Clinical Trial Site
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Clinical Trial Site
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Clinical Trial Site
Greenwood, South Carolina, United States, 29646
United States, Tennessee
Clinical Trial Site
Nashville, Tennessee, United States, 37232
United States, Texas
Clinical Trial Site
Houston, Texas, United States, 77030
Australia
Clinical Trial Site
Perth, Australia
Clinical Trial Site
South Brisbane, Australia
Canada
Clinical Trial Site
Toronto, Canada
Clinical Trial Site
Vancouver, Canada
Italy
Clinical Trial Site
Genova, Italy
Clinical Trial Site
Messina, Italy
Clinical Trial Site
Milano, Italy
Clinical Trial Site
Rome, Italy
Clinical Trial Site
Siena, Italy
Spain
Clinical Trial Site #1
Barcelona, Spain
Clinical Trial Site #2
Barcelona, Spain
Clinical Trial Site #1
Madrid, Spain
Clinical Trial Site #2
Madrid, Spain
Clinical Trial Site
Valencia, Spain
United Kingdom
Clinical Trial Site
Edinburgh, United Kingdom
Clinical Trial Site
Liverpool, United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
GW Research Ltd
Additional Information:
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Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT04252586    
Other Study ID Numbers: GWND19002
2019-001605-24 ( EudraCT Number )
First Posted: February 5, 2020    Key Record Dates
Last Update Posted: April 28, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by GW Research Ltd:
Cannabidiol
CBD
Epidiolex
GWP42003-P
Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Epidiolex
Anticonvulsants