CBDV for Prader-Willi Syndrome

Overseen By Eric Hollander, MD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Montefiore Medical Center

Must not be taking: CBD, THC

Disqualifiers: Drug abuse, Bipolar, Schizophrenia, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

14 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This study tests if a safe cannabis compound that doesn't cause a high can help reduce irritability in children and young adults with Prader-Willi Syndrome.

Will I have to stop taking my current medications?

The trial requires that your current medications be stable for at least 4 weeks before starting and throughout the study, so you should not stop taking them if they are stable.

Is CBDV safe for humans?

CBDV has been studied for safety in conditions like Rett syndrome and epilepsy, showing it is generally well-tolerated in humans, including children, without causing hallucinations. ^{1 2 3 4 5}

How does the drug CBDV differ from other treatments for Prader-Willi Syndrome?

CBDV is unique because it is a non-psychotropic compound derived from Cannabis sativa, which means it doesn't cause the 'high' associated with cannabis. It has shown potential in treating neurological conditions like epilepsy and autism spectrum disorders by interacting with specific receptors in the brain, which may offer a novel approach for managing symptoms of Prader-Willi Syndrome. ^{1 2 4 6 7}

What data supports the effectiveness of the drug CBDV for Prader-Willi Syndrome?

Research shows that CBDV, a compound similar to CBD, has been beneficial in improving neurological and social deficits in conditions like Rett syndrome, which shares some symptoms with Prader-Willi Syndrome. This suggests potential benefits of CBDV for Prader-Willi Syndrome, although direct evidence is not yet available. ^{1 2 4 5 7}

Who Is on the Research Team?

Eric Hollander, MD

Principal Investigator

Montefiore Medical Center/Albert Einstein College of Medicine

Are You a Good Fit for This Trial?

This trial is for children and young adults aged 5 to 30 with Prader-Willi Syndrome, confirmed by genetic testing. Participants must have stable treatments for 4 weeks prior and during the study, no drug abuse history, and not be pregnant or become pregnant. They should score moderately severe on specific behavioral scales.

Inclusion Criteria

I have a guardian who can consent for me and help with my study assessments.

I am between 5 and 30 years old.

My Prader-Willi Syndrome diagnosis is confirmed by genetic tests.

Exclusion Criteria

I am not pregnant and will take a pregnancy test if I can become pregnant. I agree not to become pregnant while taking CBDV.

You have a known or suspected allergy to CBDV or any of the ingredients used in the medicine, like sesame.

You have a history of drug addiction, including problems with cannabis.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive weight-based dosing of 10 mg/kg/day of CBDV or placebo for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CBDV Compound
• Placebo

Trial Overview The study tests cannabidivarin (CBDV) against a placebo in managing PWS symptoms. It assesses CBDV's safety and effectiveness through physical exams, lab results within PWS norms, caregiver assessments, and behavior checklists.

How Is the Trial Designed?

Group I: Cannabidivarin (CBDV) Experimental Treatment 1 Intervention

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

Group II: Matched Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Montefiore Medical Center

Lead Sponsor

Trials

468

Recruited

599,000+

GW Pharmaceuticals Ltd

Industry Sponsor

Trials

14

Recruited

1,700+

Foundation for Prader-Willi Research

Collaborator

Trials

14

Recruited

1,200+

Jazz Pharmaceuticals

Industry Sponsor

Trials

252

Recruited

35,100+

Bruce C. Cozadd

Jazz Pharmaceuticals

Chief Executive Officer since 2009

BA in Economics from Yale University, MBA from Stanford University

Dr. Austin

Jazz Pharmaceuticals

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

Published Research Related to This Trial

In a study involving 34 children aged 4-10 with Dravet syndrome, purified cannabidiol (CBD) was found to be generally well-tolerated, with a dose-proportional increase in exposure to CBD and its metabolites, indicating effective pharmacokinetics.

While CBD treatment resulted in more adverse events (AEs) compared to placebo, such as pyrexia and sedation, no significant safety concerns were identified, and all patients recovered from any elevated liver enzymes without meeting criteria for drug-induced liver injury.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized, dose-ranging safety trial of cannabidiol in Dravet syndrome. Devinsky, O., Patel, AD., Thiele, EA., et al.[2022]

Cannabidivarin (CBDV), a non-psychotomimetic cannabinoid, is gaining attention for its potential therapeutic effects in managing neurological conditions, particularly epilepsy and autism spectrum disorder.

This review compiles both animal and human studies to provide a comprehensive overview of CBDV's therapeutic potential, highlighting its promise as a treatment option alongside the more widely studied cannabidiol (CBD).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapeutic potential of cannabidivarin for epilepsy and autism spectrum disorder. Zamberletti, E., Rubino, T., Parolaro, D.[2022]

This systematic review analyzed nine randomized controlled trials (RCTs) on the use of cannabidiol (CBD) for neurodevelopmental disorders, revealing inconsistent evidence for its efficacy, which makes it difficult to recommend for clinical use.

While some studies suggest potential benefits of CBD for conditions like ADHD and autism, the overall variability in study design and outcomes indicates that more rigorous research is needed to establish its safety and effectiveness as a treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A systematic review of cannabidiol trials in neurodevelopmental disorders. Parrella, NF., Hill, AT., Enticott, PG., et al.[2023]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Cannabidivarin completely rescues cognitive deficits and delays neurological and motor defects in male Mecp2 mutant mice. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Randomized, dose-ranging safety trial of cannabidiol in Dravet syndrome. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Chronic treatment with the phytocannabinoid Cannabidivarin (CBDV) rescues behavioural alterations and brain atrophy in a mouse model of Rett syndrome. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of cannabidivarin treatment of epilepsy in girls with Rett syndrome: A phase 1 clinical trial. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Therapeutic potential of cannabidivarin for epilepsy and autism spectrum disorder. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

A systematic review of cannabidiol trials in neurodevelopmental disorders. [2023]

7. Netherlands pubmed.ncbi.nlm.nih.gov

Cannabis sativa: Much more beyond &#916;9-tetrahydrocannabinol. [2021]