Clinical Trials Opportunities Archives - Foundation for Prader-Willi Research | Clinical Trials Opportunities (2)

DCCR for the Treatment of Hyperphagia in PWS

Study Purpose Soleno Therapeutics is conducting a Phase 3 study of DCCR to measure the efficacy of the DCCR for treating hyperphagia in PWS. The study seeks to enroll 105 patients with PWS ages 4 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. The initial 15-week study will be followed by a 9-month extension during which time all patients will receive DCCR. Protocol Update: A recent amendment to the study protocol now allows patients between 20kg and 130kg to enroll in the study, provided they meet all eligibility criteria. Individual study sites will be able to enroll patients in the new weight band (20-30kg) once the appropriate tablet strengths come available, which will likely be in the September timeframe. Patients who are interested in enrolling should contact their nearest study coordinator.

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

[NOTE: This study is now closed. Learn about the results here.] 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

Study Purpose The Mindfulness-Based Intervention for Temper Outbursts intends to evaluate the feasibility and acceptability of a mindfulness-based intervention for managing temper outbursts in PWS. The study will also evaluate the interventions  impact on anxiety.  This study will be conducted remotely via phone, email and video-conference and is, therefore, open to families both within and outside of Australia. Eligible participants with PWS, their primary caregiver (parent or resident carer) and a teacher or support worker will be invited to participate in an 18-week trial. Two weeks before the start of the trial primary caregivers will be trained to use and teach the mindfulness-based intervention. The 18-weeks will be divided into three six-week periods termed baseline, intervention and follow-up. Throughout the 18-weeks, primary caregivers and a teacher support worker will be asked to monitor the person’s behaviours. During the intervention period, primary caregivers will teach the mindfulness-based intervention to the person with PWS. A webinar sharing details on this study was recorded May, 2019. You can watch the webinar below or read a transcription of the webinar on our blog. 

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.

Play-based Remote Enrichment To Enhance Development (PRETEND) Research Study

This 8-week telehealth (remote) intervention program focuses on building play skills, emotional understanding and regulation, and social skills in Prader-Willi syndrome. 

Carbetocin for the Treatment of 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.  

A Study of GLWL-01 in Patients With Prader-Willi Syndrome

Study Purpose GLWL is conducting a Phase 2 clinical trial to to evaluate the efficacy, safety, and pharmacokinetics of GLWL-01 in treating hyperphagia in patients with PWS. Participants will be assigned to one of two treatment sequences (GLWL-01/Placebo or Placebo/GLWL-01), with each sequence consisting of two treatment periods separated by a washout period.

Survey Measuring the Impact of PWS on Caregivers

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