Clinical Trials Opportunities

PWS Anxiety and Distress Questionnaire

Got Anxiety? Want to help pave the way for new medicines to treat anxiety in PWS? Levo Therapeutics and the Foundation for Prader-Willi Research are working to develop a new questionnaire for use in clinical studies of novel treatments for PWS. We want to know more about how anxiety is expressed in people with PWS.

PWS Activity, Sleep and Eating Pattern Pilot Study

Dr. Panda at the Salk Institute for Biological Studies, is conducting a pilot study to examine the daily patterns in activity, sleep, and food/beverage intake in individuals diagnosed with Prader-Willi Syndrome.

DCCR for the Treatment of Hyperphagia in PWS

Study Purpose Soleno Therapuetics is conducting a Phase 3 study of DCCR to measure the drugs efficacy for treating hyperphagia in PWS. The study seeks to enroll 105 patients with PWS ages 8 years and older. The study will be a 15 week, randomized, double-blind, placebo-controlled study of Diazoxide Choline Controlled-Release Tablet (DCCR) and will require a once daily oral administration. Five sites are now open with additional sites expected to open soon.

A Study to Assess Weight Change Over 6 months in Prader-Willi Syndrome (PWS)

FPWR is conducting a text-messaging based research study to assess weight changes in Prader-Willi syndrome (PWS) over a period of six months. We’d like to learn more about the normal variation in weight, over time, in the PWS population.  We hope to recruit 150-300  individuals across the US and Canada to participate, so we can get a good picture of how weight changes over time.  We anticipate that this study will inform future clinical trials for hyperphagia/obesity related therapies, and provide a basis for understanding how well potential therapies are working. Patients should not be enrolled in other clinical trials for the duration of this study. The study can be done from your home, with no visit to a clinic.

Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in PWS

Study Purpose This phase 2 randomized double blind 8-week treatment trial of intranasal oxytocin (IN-OXT) will assess IN-OXT's affect on: eating behaviors, repetitive behaviors, weight and body composition, quality of life, and salivary OXT and hormone levels.  Data from the study will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS.

Oxytocin and the Autonomic Nervous System in PWS

There is a reduction in the number of neurons that produce oxytocin in people with PWS. This, along with a range of other evidence supports the likelihood that abnormalities in the oxytocin system are key to the problems of PWS. However, studies examining the levels of oxytocin in PWS as well as clinical trials evaluating the efficacy of oxytocin on PWS symptoms has led to mixed results. We want to better understand these mixed findings. This will help to develop more effective interventions in the future.

Feasibility of a Mindfulness-Based Intervention for Temper Outbursts

The purpose of the study is to evaluate the feasibility and acceptability of a mindfulness-based intervention for managing temper outbursts in PWS. We will also be looking at the impact on anxiety.

Developing Objective Biomarkers of Hyperphagia in Children with PWS

Vanderbilt and Case Western Universities are collaborating in a new study to better understand how eye tracking technology can be used to measure how children process images of food vs. objects.

Evaluating the Play-based Remote Enrichment To Enhance Development (PRETEND) Program

This 8-week telehealth (remote) intervention program focuses on building play skills, emotional understanding and regulation, and social skills. The study involves two in-person visits, one at the beginning and one at the end, that would be conducted in the family's local area and then the 8-week intervention, which is all done via remote videoconferencing.

Study of Carbetocin in PWS

Study Purpose Carbetocin (LV-101) is an investigational drug that was created to have effects in the body like oxytocin.  Carbetocin is not approved in the United States, but is approved in some other countries for treatment of women with excessive bleeding after giving birth via caesarean section. This Phase 3 study of intranasal carbetocin (LV-101) is a randomized, double-blind study with an 8-week, placebo controlled period, followed by a long-term follow-up period of 56 weeks during which all participants will receive active treatment with LV-101. At Week 8, participants who were randomized to placebo in the placebo-controlled period will be randomized to one of the two LV-101 doses, administered three times per day before meals.

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