CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)

NCT ID: NCT03848481

Last Updated: 2024-11-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-11-23
• Study Completion Date: 2024-10-31

Brief Summary

This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.

Detailed Description

This clinical research trial aims to study the feasibility and safety of cannabidivarin (CBDV), in children and young adults with Prader-Willi Syndrome (PWS). CBDV has effects independent of CB1 and CB2 receptor activation and a good safety profile. This proposal addresses the Foundation for Prader Willi Research's PWS Research Plan: Program 1, Clinical Care Research: seeks to evaluate treatments that aim to reduce behavioral symptoms, such as irritability, in order to improve the quality of life of both the individual with PWS and their families. GW Pharmaceuticals will provide the CBDV drug and matching placebo.

Conditions

Prader-Willi Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Cannabidivarin (CBDV)

Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks

Group Type: EXPERIMENTAL

CBDV Compound

Intervention Type: DRUG

CBDV is obtained from the Cannabis sativa L. plant and contains a negligible quantity (less than 0.2%) of THC

Matched Placebo

Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo oral solution contains matching excipients.

Interventions

CBDV Compound

CBDV is obtained from the Cannabis sativa L. plant and contains a negligible quantity (less than 0.2%) of THC

Intervention Type: DRUG

Placebo

Placebo oral solution contains matching excipients.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male or Female outpatients aged 5 to 30 years.

2. Diagnosis of PWS confirmed by genetic testing and patient medical records and history.

3. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.

4. Have a physical exam and laboratory results that are within the norms for PWS

5. Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the patient's development and behavior throughout the study. Child Assent will be obtained if the subject is 7 years of age or older and has the mental capacity to understand and sign a written assent form and/or give verbal assent.

6. Score on the Clinical Global Impression Scale Severity (CGI-S) ≥ 4 (moderate severity) at baseline.

7. Score of ≥18 on the Aberrant Behavior Checklist-Irritability (ABC-I) at baseline.

8. Agree not to drive or operate machinery.

Exclusion Criteria

1. Exposure to any investigational agent in the 30 days prior to randomization.

2. Prior chronic treatment with CBD or CBDV.

3. Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.

4. History of Drug Abuse Disorder including Cannabis Use Disorder

5. A primary psychiatric diagnosis other than PWS, including bipolar disorder, psychosis, schizophrenia, PTSD or MDD. These patients will be excluded due to potential confounding results.

6. A medical condition that severely impacts the subject's ability to participate in the study, interferes with the conduct of the study, confounds interpretation of study results or endangers the subject's well-being (including but not limited to hepatic or renal impairment and cardiovascular disease).

7. Known or suspected allergy to CBDV or excipients used in the formulation (i.e. sesame).

8. Clinical indications of renal, pancreatic, or hematologic dysfunction as evidenced by values above upper limits of normal for BUN/creatinine, values twice the upper limit of normal for serum lipase and amylase, platelets <80,000 /mcL, WBC<3.0 103 /mcL. or > 2 X UNL values of AST or ALT.

10. Female subjects who are pregnant will be excluded from the study. If a female subject is able to become pregnant, she will be given a pregnancy test before entry into the study. Female subjects will be informed not become pregnant while taking CBDV. Female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if they become pregnant during the study.

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Foundation for Prader-Willi Research

OTHER

Sponsor Role: collaborator

GW Pharmaceuticals Ltd

INDUSTRY

Sponsor Role: collaborator

Montefiore Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Eric Hollander, MD

Role: PRINCIPAL_INVESTIGATOR

Montefiore Medical Center/Albert Einstein College of Medicine

Locations

Montefiore Medical Center, Albert Einstein College of Medicine

The Bronx, New York, United States

Countries

United States

Other Identifiers

2019-9914

Identifier Type: -

Identifier Source: org_study_id