Want to participate in research? Check out our ongoing studies here!

Current Studies:

Phase II Clinical Trial: CSTI-500-005

The purpose of this study is to evaluate how an investigational drug, CSTI-500, works in patients with genetically confirmed Deletion PWS. The drug is orally administered and may work to slow the movement of the neurotransmitters dopamine, serotonin, and norepinephrine in the body, which play roles in mood and hunger. Participation in this study includes 7 on-site visits to Vanderbilt, which will include a physical examination, vital sign collection, an EEG, pregnancy testing (females only), blood sampling, and questionnaires for you to complete. There will also be 2 telehealth video calls (9 visits total) over the course of 84 days.  

For additional information, please visit ClinicalTrials.gov or this link.:

In Their Own Voices: My Hunger Questionnaire (MY-HQ) of Hyperphagic Symptoms for Individuals with Prader-Willi Syndrome

With the help of a generous grant from the Foundation for Prader-Willi Research, we are developing a self-report hyperphagia questionnaire with the help of some of our friends with PWS. Our goal is to understand what hunger means to people with PWS in their own words, then use these findings to create and validate a questionnaire through which people with PWS can report their own hunger.

To learn more, contact our research coordinator here: Email

 

 

Completed Studies:

HBS-101-CL-00

This is a Phase II clinical trial sponsored by Harmony Biosciences assessing the safety and efficacy of pitolisant in individuals with PWS. Pitolisant is a wake-promoting agent that is FDA approved for the treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy. Narcolepsy, obstructive sleep apnea, and EDS are all common ailments for people with PWS, so the goal of this study is to determine if pitolisant could be an effective treatment for these symptoms in PWS.

Click here to view topline results released by Harmony in November 2022.

CSTI-500-003

In this Phase I clinical trial sponsored by ConSynance Therapeutics, we gathered data to assess the safety of CSTI-500, a triple monoamine reuptake inhibitor, in teens and adults with PWS. We saw 10 individuals aged 13 and up between November 2022 and February 2023, with no major side effects reported. Each participant took a single, 10 mg dose of CSTI-500 with continuous monitoring of safety labs, as well as blood draws and finger sticks to measure the amount of study drug remaining in the system. We are still awaiting topline data results to learn more about how this drug is metabolized by people with PWS, as well as the comparability of blood draws to finger sticks in measuring the amount of drug in the bloodstream.

In the future, we hope to study this medicine in a Phase II trial, where we can assess not only safety, but efficacy. We will be looking for decreases in anxiety and behavioral problems in PWS, as well as decreases in hyperphagia.

LV-101

In this Phase III clinical trial sponsored by Levo Therapeutics, we monitored participants on a stable, long-term dose of intranasal carbetocin. We observed lowered hyperphagia as well as improved mood and behavior in those continuously using carbetocin.

Unfortunately, this study closed before carbetocin could receive FDA approval due to the acquisition of Levo by Acadia Pharmaceuticals, inc. We hope to continue studying carbetocin under Acadia some time in the future.

Parents who are interested in current studies should contact Elizabeth Roof at elizabeth.roof@vanderbilt.eduor 615-343-3330 to get more information about the study.

 

Sometimes Research Takes a Village!

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Elizabeth Roof Speaks at the 2022 FPWR Conference

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Interdisciplinary research, services for families, and education for the community on Prader-Willi Syndrome.

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We aim to study the whole person with PWS – their strengths as well as problems – and how these relate to genetics, families, neurochemicals, aging, and successful living.

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Music Campers Record with Country Music Stars

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Music Campers Perform at the Grand Ole Opry

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Research Subjects Participate in Electroencephalograms

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We aim to study the whole person with PWS – their strengths as well as problems – and how these relate to genetics, families, neurochemicals, aging, and successful living.

... KEEP READING

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Clinical research not only advances science and helps find new treatments, it can also be a way to have fun and make new friends!

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Clinical research not only advances science and helps find new treatments, it can also be a way to have fun and make new friends!

... KEEP READING

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Music Campers Take a Silly Photo Before Performing at the Grand Ole Opry.

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Prader-Willi Syndrome Walk

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ACM Lifting Lives Music Camp for Williams Syndrome

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