Condition

Prader-Willi Syndrome clinical trials and research

• 13Clinical trials
• 2NIH research projects
• 16Countries

Newest Prader-Willi Syndrome clinical trials

All recruiting trials (13)

• Group therapy to help teens with Prader-Willi regulate emotions

  Teens with Prader-Willi Syndrome and their families will try a group therapy program to help calm emotions and reduce irritability and anxiety.

  Recruiting Children's Mercy Hospital Kansas City · Kansas City, Missouri · NCT07607730

• CSTI-500 in Prader-Willi Syndrome: a dose-escalation study for safety and efficacy

  This study tests CSTI-500 in people aged 13–50 with Prader-Willi syndrome to see if it is safe and helps with symptoms, using increasing doses guided by blood levels.

  Recruiting Phase 2 ConSynance Therapeutics · Nashville, Tennessee · NCT07348601

• Using Tirzepatide to treat obesity in young adults with Prader-Willi Syndrome and hypothalamic obesity

  This study is testing if a weight loss medication called Tirzepatide can help young adults with Prader-Willi Syndrome and hypothalamic obesity lose weight and improve their health.

  Recruiting at 3 sites Phase 4 Seattle Children's Hospital · Saint Paul, Minnesota and 2 other locations · NCT06901245

• Examining autistic traits and sensory profiles in children with Prader-Willi Syndrome

  This study looks at how autistic traits and sensory experiences show up in children with Prader-Willi Syndrome to better understand their needs and see if early oxytocin treatment makes a difference.

  Recruiting University Hospital, Toulouse · Toulouse · NCT06877715

• Inclusive physical activity program for adults with intellectual disabilities

  This study is testing a new fitness program for adults with intellectual disabilities to see if it helps them be more active and feel better over 16 weeks.

  Recruiting University of North Carolina, Chapel Hill · Fayetteville, Arkansas and 1 other locations · NCT06740162

• Using vagus nerve stimulation to improve emotion and thinking in Prader-Willi Syndrome

  This study is testing whether a new treatment using vagus nerve stimulation can help improve emotions and thinking skills in adults with Prader-Willi Syndrome.

  Recruiting at 3 sites University of Bordeaux · Bordeaux and 3 other locations · NCT06720571

• Registry for Rare Diseases

  This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.

  Recruiting Hospital Italiano de Buenos Aires · Buenos Aires, Buenos Aires · NCT06573723

• Evaluating pitolisant for excessive daytime sleepiness in Prader-Willi syndrome

  This study is testing if a medication called pitolisant can help reduce excessive daytime sleepiness in people aged 6 and older with Prader-Willi syndrome.

  Recruiting at 52 sites Phase 3 Harmony Biosciences Management, Inc. · Los Angeles, California and 56 other locations · NCT06366464

• Observing metabolic profiles in patients with imprinting disorders

  This study looks at the metabolic health of people, both adults and children, with imprinting disorders to see if there are common patterns and how these conditions affect their overall health.

  Recruiting at 8 sites Institut National de la Santé Et de la Recherche Médicale, France · Angers and 19 other locations · NCT05945576

• Using brain stimulation to improve appetite control in Prader-Willi syndrome

  This study tests if a brain stimulation technique can help people with Prader-Willi syndrome control their appetite and feel full.

  Recruiting Brigham and Women's Hospital · Belmont, Massachusetts · NCT05938543

• Using prebiotics and probiotics to manage obesity in children with Prader-Willi syndrome

  This study is testing whether giving probiotics and prebiotics can help children with Prader-Willi syndrome manage their weight and appetite better.

  Recruiting Children's Hospital of Fudan University · Shanghai, Shanghai Municipality · NCT05791604

• Observing the health management of patients with rare genetic syndromes

  This study looks at how patients with rare genetic syndromes like Prader-Willi and Klinefelter manage their health as they move from children's care to adult care to see what support they need.

  Recruiting Erasmus Medical Center · Rotterdam, South Holland · NCT04463316

• Register of Patients With Prader-Willi Syndrome

  This study is creating a national register to collect information about people with Prader-Willi Syndrome to better understand their needs and improve their care.

  Recruiting University Hospital, Toulouse · Toulouse · NCT02829684

NIH-funded research (2)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Investigating gene therapy for Prader-Willi syndrome

  This study is exploring a new gene therapy for Prader-Willi syndrome that aims to replace missing genes using a tiny virus, which could help improve treatments for people living with this condition.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-10771280

• Understanding the genetic causes of Prader-Willi syndrome

  This study is looking at how certain genes related to Prader-Willi syndrome work, especially a group called SNORD116, to see if turning on the right genes can help improve brain function and find new ways to treat the condition.

  NIH research University of Connecticut Sch of Med/dnt · Farmington, United States · NIH-10662498

Related topics

Quick answers

How many Prader-Willi Syndrome clinical trials are recruiting right now?

13, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 16 countries. The most sites are in United States (6 trials), France (5 trials), Argentina (1 trial), China (1 trial), Australia (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 4: 1, Phase 3: 1, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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