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Prader–Willi syndrome

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Prader–Willi syndrome

14 trials tracked for Prader–Willi syndrome: 4 in phase 3 or 4 and 3 with published results. The most-cited published study has 51 citations.

14Trials tracked

4Phase 3 & 4

0Recruiting

3With published results

Phase distribution

Phase 3 4 Phase 2 8 Other / NA 2

Phase 3 Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome Completed · 51 cited
Phase 3 A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome Completed · 50 cited
Phase 3 Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS Completed
Phase 3 Growth Hormone Use in Adults With Prader-Willi Syndrome Completed
Phase 2 Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants Completed
Phase 2 Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome Completed

Show 8 more trials

Phase 2 A Study of GLWL-01 in Patients With Prader-Willi Syndrome Completed
Phase 2 A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension Completed
Phase 2 Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome Completed
Phase 2 Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome Completed
Phase 2 Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome Completed
Phase 2 Intranasal Oxytocin for Infants With Prader-Willi Syndrome Completed
N/A Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome Completed
N/A Pilot Study of Startle-response Test to Assess Transcranial Direct Current Stimulation-induced Modulation of Hyperphagia in Prader-Willi Syndrome Completed

Showing the 14 most-cited and recently-updated of 14 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Prader–Willi syndrome: what the trials found

Clinical trials have evaluated various pharmacological interventions for managing symptoms associated with Prader-Willi syndrome. DCCR demonstrated a statistically significant improvement in Clinical Global Impression of Improvement (CGI-I) at week 13 ¹. Somatropin was evaluated in both adult and pediatric cohorts, showing a reduction in body fat percentage in the adult group (-3.09%) and mixed results in the pediatric groups (-4.59% for GH-naïve; 1.34% for GH-treated) ³. Nutropin AQ showed significant improvements in body composition, blood chemistry, and physical activity levels ⁴.

Oxytocin and its intranasal formulation (IN-OXT) have been studied for managing hyperphagia. Intranasal oxytocin demonstrated a statistically significant reduction in the 'Drive Factor Score' of the Hyperphagia Questionnaire ¹⁰, while other metrics such as total score and behavior factor did not reach statistical significance ¹⁰. Other studies on oxytocin and intranasal oxytocin were conducted regarding hyperphagia, anxiety, and suck/swallow competency without reaching statistically significant primary outcomes in those specific categories [2, 12].

Recent results — preliminary, needs further review

[ ] Pitolisant oral tablets are currently being evaluated in Phase 2 trials.

For the clinician treating this condition

DCCR is associated with improved clinical global impression of improvement in patients with Prader-Willi syndrome ¹.
Somatropin may be utilized to reduce body fat percentage, though efficacy varies between pediatric and adult cohorts ³.
Nutropin AQ has shown statistically significant improvements in physical activity and body composition ⁴.
Intranasal oxytocin specifically targets the 'Drive Factor' of hyperphagia as measured by standardized questionnaires ¹⁰.

AI synthesis of 4 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.