CBDV for Prader-Willi Syndrome

No longer recruiting at 1 trial location

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and feasibility of a compound called cannabidivarin (CBDV) for treating symptoms in children and young adults with Prader-Willi Syndrome (PWS), a genetic disorder affecting growth, metabolism, and behavior. Participants will receive either CBDV or a placebo for 12 weeks to determine if CBDV can help manage symptoms like irritability. The trial seeks participants aged 5 to 30 with a confirmed PWS diagnosis who have been stable on their current treatments for at least four weeks. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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 there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that cannabidivarin (CBDV) might help treat children and young adults with Prader-Willi Syndrome (PWS). Although limited safety information exists, CBDV is similar to CBD, which most people tolerate well. In studies of similar substances, most individuals have experienced only mild side effects, such as fatigue or changes in appetite.

This trial is in an early stage, so researchers are still carefully checking safety. However, reaching this stage indicates that CBDV is considered safe enough for testing on more people. Prospective participants should consult a healthcare professional about any concerns before joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Prader-Willi Syndrome?

Unlike the standard treatments for Prader-Willi Syndrome, which often focus on hormone therapies or appetite suppressants, the Cannabidivarin (CBDV) compound offers a novel approach by leveraging a compound derived from cannabis. Researchers are excited about CBDV because it potentially targets the neurological aspects of Prader-Willi Syndrome, which may help in reducing hyperphagia (excessive eating) and improving behavior. Additionally, CBDV is administered based on weight, which allows for more personalized dosing, potentially enhancing its effectiveness and minimizing side effects. This new direction could lead to a significant improvement in quality of life for those affected by the syndrome.

What evidence suggests that CBDV could be an effective treatment for Prader-Willi Syndrome?

Research has shown that cannabidivarin (CBDV), which participants in this trial may receive, might help alleviate symptoms of Prader-Willi Syndrome (PWS). While studies specifically on PWS are ongoing, CBDV has shown promise in similar conditions. It has effectively reduced irritability and repetitive behaviors, key symptoms of PWS. People with other conditions have generally tolerated CBDV well, suggesting its safety. Although more research is needed, early results indicate that CBDV could be a helpful treatment option for those with PWS. ^{1 2 3 5 6}

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]

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]

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]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT03848481

CBDV vs Placebo in Children and Adults up to Age 30 ...

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 ...

2.

fpwr.org

fpwr.org/fpwr-funded-projects/cannabidivarin-cbdv-vs-placebo-in-children-with-pws

Cannabidivarin (CBDV) vs Placebo in Children with PWS

We hypothesize that CBDV will be superior to placebo and will have a positive effect on the core symptoms of PWS including irritability, restricted/repetitive ...

3.

ctv.veeva.com

ctv.veeva.com/study/cbdv-vs-placebo-in-children-and-adults-up-to-age-30-with-prader-willi-syndrome-pws

CBDV vs Placebo in Children and Adults up to Age 30 With ...

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

4.

withpower.com

withpower.com/trial/phase-3-syndrome-10-2020-62242

CBDV for Prader-Willi Syndrome · Info for Participants

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 ...

5.

clinicaltrials.gov

clinicaltrials.gov/ct2/show/NCT03848481?term=cbdv&draw=2&rank=2

CBDV vs Placebo in Children and Adults up to Age 30 ...

An unfavorable change in the health of a participant, including abnormal laboratory findings, that happens during a clinical study or within a certain amount of ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9916985/

Clinical Trials in Prader–Willi Syndrome: A Review - PMC

The authors reported that the use of oxytocin in children over five days was safe, and a significant improvement was noted in anxiety and self-injurious ...