What side effects are associated with this type of intervention?

Common treatments for Prader-Willi Syndrome (PWS) that interact with the endocannabinoid system, such as Cannabidivarin (CBDV), may have side effects including drowsiness, fatigue, diarrhea, and changes in appetite or weight. Additionally, cannabinoid-based treatments can sometimes cause dizziness, dry mouth, and mood changes. It is important to monitor these side effects and discuss them with a healthcare provider to ensure the benefits outweigh the risks.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments for Prader-Willi Syndrome that specifically target the endocannabinoid system, affecting neurotransmitter release and neuroinflammation, similar to Cannabidivarin (CBDV). The primary FDA-approved treatment for PWS focuses on growth hormone therapy to address growth deficiencies and improve muscle tone. Other treatments are symptomatic, addressing issues such as obesity, behavioral problems, and hormonal deficiencies.

What other types of research exist?

Promising alternatives to Cannabidivarin (CBDV) for Prader-Willi Syndrome patients include other cannabinoids such as THC and CBD, which have shown potential in modulating neuroinflammation and neurotransmitter release. Additionally, omega-3 fatty acids may be considered due to their role in neuroinflammation and neurotransmitter modulation.

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Cannabinoid

CBDV for Prader-Willi Syndrome

Phase 2

Waitlist Available

Led By Eric Hollander, MD

Research Sponsored by Montefiore Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or Female outpatients aged 5 to 30 years.

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

Must not have

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).

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to week 12

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects of CBDV are not detailed in this summary but would typically include monitoring for any adverse reactions compared to the placebo group since it's a feasibility and safety trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 5 and 30 years old.

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any health conditions that would make it unsafe for me to join the study.

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I do not have bipolar disorder, psychosis, schizophrenia, PTSD, or major depression.

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My blood tests show kidney, pancreas, or blood issues.

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I have been on long-term treatment with CBD or CBDV.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to week 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to week 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to week 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Aberrant Behavior Checklist-Irritability Subscale (ABC-I)

Secondary study objectives

Lethargy

ActiGraph GT9X-BT activity monitors

Caregiver Strain Questionnaire (CSQ)

+5 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Cannabidivarin (CBDV)Experimental Treatment1 Intervention

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

Group II: Matched PlaceboPlacebo Group1 Intervention

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

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Prader-Willi Syndrome (PWS) often target the endocannabinoid system, which is crucial for regulating neurotransmitter release and neuroinflammation. Cannabidivarin (CBDV), for example, interacts with this system to potentially alleviate hyperphagia, behavioral issues, and cognitive impairments in PWS patients. This targeted approach is significant as it offers a way to manage symptoms that are challenging to control with traditional therapies, improving the quality of life for those affected by PWS.

Cannabinoids and Dementia: A Review of Clinical and Preclinical Data.The marijuana component cannabidiol inhibits beta-amyloid-induced tau protein hyperphosphorylation through Wnt/beta-catenin pathway rescue in PC12 cells.