{"disease":{"id":"prader-willi-syndrome","name":"Prader-Willi syndrome","therapeutic_area":"","data":{},"enrichment_level":0,"last_enriched_at":null,"visit_count":0,"created_at":"2026-04-19T18:31:21.150Z","updated_at":"2026-04-19T18:39:44.960Z","meddra_pt":null,"meddra_code":null,"icd10_codes":null,"synonyms":[],"prevalence_global":null,"prevalence_us":null,"marketed_drug_count":5,"pipeline_drug_count":0,"trial_count":1},"drugs":{"marketed":[{"drug_id":"genotropin","indication_name":"Prader-Willi syndrome","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Omnitrope","generic_name":"Somatropin","company_name":"Serono","drug_phase":"marketed","molecular_target":"Dimeric GH receptors on cell membranes of target tissues","drug_class":"Recombinant human growth hormone","quality_score":10,"revenue":"446","mechanism":"Somatropin binds dimeric GH receptors on target cells, inducing intracellular signaling and GH-dependent protein synthesis including IGF-1."},{"drug_id":"somatropin-adult-cohort","indication_name":"Treatment of Prader-Willi syndrome in adults.","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"somatropin - adult cohort","generic_name":"somatropin-adult-cohort","company_name":"Pfizer","drug_phase":"marketed","molecular_target":"Growth Hormone Receptor","drug_class":"Growth Hormone","quality_score":10,"revenue":"446","mechanism":""},{"drug_id":"carbetocin","indication_name":"Prader-Willi syndrome","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Pabal","generic_name":"CARBETOCIN","company_name":"Ferring Pharmaceuticals Ltd","drug_phase":"marketed","molecular_target":"Oxytocin receptor","drug_class":"carbetocin","quality_score":17,"revenue":null,"mechanism":""},{"drug_id":"duratocin","indication_name":"Prader-Willi syndrome","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"Duratocin","company_name":"Dongyu Wang","drug_phase":"phase_1","molecular_target":"Oxytocin receptor, Vasopressin V1a receptor, Vasopressin V2 receptor","drug_class":"","quality_score":null,"revenue":null,"mechanism":""},{"drug_id":"omnitrope","indication_name":"Prader-Willi syndrome","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"Omnitrope","company_name":"Sandoz","drug_phase":"marketed","molecular_target":"Growth hormone receptor","drug_class":"","quality_score":16,"revenue":null,"mechanism":null},{"drug_id":"somatropine","indication_name":"Prader-Willi syndrome","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"somatropine","company_name":"Sanofi","drug_phase":"phase_3","molecular_target":"Growth hormone receptor","drug_class":"","quality_score":null,"revenue":null,"mechanism":null}],"pipeline":[],"offLabel":[],"totalMarketed":6,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT03655223","title":"Early Check: Expanded Screening in Newborns","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30000,"lead_sponsor_name":"RTI International","has_results":false},{"nct_id":"NCT03718416","title":"Natural History Study of Serious Medical Events in PWS","phase":"","overall_status":"COMPLETED","enrollment_count":700,"lead_sponsor_name":"Foundation for Prader-Willi Research","has_results":false},{"nct_id":"NCT04463316","title":"GROWing Up With Rare GENEtic Syndromes","phase":"","overall_status":"RECRUITING","enrollment_count":600,"lead_sponsor_name":"dr. Laura C. G. de Graaff-Herder","has_results":false},{"nct_id":"NCT06573723","title":"Institutional Registry of Rare Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":380,"lead_sponsor_name":"Hospital Italiano de Buenos Aires","has_results":false},{"nct_id":"NCT04768803","title":"Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity","phase":"","overall_status":"UNKNOWN","enrollment_count":300,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT03790865","title":"Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome","phase":"PHASE2, PHASE3","overall_status":"TERMINATED","enrollment_count":158,"lead_sponsor_name":"Millendo Therapeutics SAS","has_results":true},{"nct_id":"NCT06239116","title":"A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":150,"lead_sponsor_name":"Rhythm Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT03649477","title":"Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":130,"lead_sponsor_name":"Levo Therapeutics, Inc.","has_results":true},{"nct_id":"NCT05249998","title":"Improving Care of Prader-Willi Syndrome : Evaluation of a New Care Program Combining Adapted Physical Activity, Nutrition and Therapeutic Education","phase":"NA","overall_status":"COMPLETED","enrollment_count":128,"lead_sponsor_name":"Assistance Publique - Hôpitaux de Paris","has_results":false},{"nct_id":"NCT03440814","title":"A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":127,"lead_sponsor_name":"Soleno Therapeutics, Inc.","has_results":true},{"nct_id":"NCT03836300","title":"Parent and Infant Inter(X)Action Intervention (PIXI)","phase":"NA","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":120,"lead_sponsor_name":"RTI International","has_results":false},{"nct_id":"NCT03714373","title":"Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":115,"lead_sponsor_name":"Soleno Therapeutics, Inc.","has_results":false},{"nct_id":"NCT06144645","title":"A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":102,"lead_sponsor_name":"Foundation for Prader-Willi Research","has_results":false},{"nct_id":"NCT06900335","title":"Functional Connectome in Prader-Willi Syndrome: Neuroimaging and AI to Assess Therapeutic Impact","phase":"","overall_status":"COMPLETED","enrollment_count":101,"lead_sponsor_name":"Corporacion Parc Tauli","has_results":false},{"nct_id":"NCT05032326","title":"Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":80,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT06877715","title":"Autistic Symptomatology and Sensory Profile in Children With Prader-Willi Syndrome","phase":"","overall_status":"RECRUITING","enrollment_count":75,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT04257929","title":"A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":65,"lead_sponsor_name":"Harmony Biosciences Management, Inc.","has_results":true},{"nct_id":"NCT05791604","title":"The Intervention of Obesity in Children With Prader-Willi Syndrome Using Prebiotics and Probiotics","phase":"NA","overall_status":"RECRUITING","enrollment_count":60,"lead_sponsor_name":"Children's Hospital of Fudan University","has_results":false},{"nct_id":"NCT04102839","title":"PWS Outcomes Assessment Study","phase":"","overall_status":"COMPLETED","enrollment_count":59,"lead_sponsor_name":"Soleno Therapeutics, Inc.","has_results":false},{"nct_id":"NCT04283578","title":"Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":52,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT05761184","title":"Characterization of Serum Proteome in Subjects With Prader Willi Syndrome (PROTEOMARKER)","phase":"","overall_status":"COMPLETED","enrollment_count":52,"lead_sponsor_name":"Istituto Auxologico Italiano","has_results":false},{"nct_id":"NCT04526379","title":"Study of Emotion and Cognition Abilities of Children With PWS and Proposition of an Innovative Remediation","phase":"NA","overall_status":"COMPLETED","enrollment_count":50,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT06448871","title":"Ultrasound to Assess Sarcopenia in Prader Willi Syndrome","phase":"","overall_status":"COMPLETED","enrollment_count":49,"lead_sponsor_name":"Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation","has_results":false},{"nct_id":"NCT04685057","title":"Probiotic Treatment for Prader-Willi Syndrome","phase":"NA","overall_status":"COMPLETED","enrollment_count":41,"lead_sponsor_name":"Fundació Sant Joan de Déu","has_results":false},{"nct_id":"NCT02311673","title":"Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":40,"lead_sponsor_name":"Rhythm Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT05322096","title":"Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":39,"lead_sponsor_name":"Gedeon Richter Plc.","has_results":false},{"nct_id":"NCT03548480","title":"Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment","phase":"NA","overall_status":"COMPLETED","enrollment_count":39,"lead_sponsor_name":"Fundació Sant Joan de Déu","has_results":false},{"nct_id":"NCT01968187","title":"Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":38,"lead_sponsor_name":"Ferring Pharmaceuticals","has_results":true},{"nct_id":"NCT06295315","title":"Evaluation of the Psychological Profile of Adult Patients With Prader-Willi Syndrome","phase":"","overall_status":"COMPLETED","enrollment_count":36,"lead_sponsor_name":"Istituto Auxologico Italiano","has_results":false},{"nct_id":"NCT04697381","title":"Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":33,"lead_sponsor_name":"Pfizer","has_results":true},{"nct_id":"NCT03197662","title":"Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":32,"lead_sponsor_name":"Eric Hollander","has_results":true},{"nct_id":"NCT06851351","title":"Respiratory Restriction in Prader-Willi Syndrome","phase":"","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Istituto Auxologico Italiano","has_results":false},{"nct_id":"NCT01401244","title":"Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Novo Nordisk A/S","has_results":false},{"nct_id":"NCT05778032","title":"Biological Age Assessment in Adults With Prader-Willi Syndrome (ETABIOLPWS)","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":30,"lead_sponsor_name":"Istituto Auxologico Italiano","has_results":false},{"nct_id":"NCT03616509","title":"GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Corporacion Parc Tauli","has_results":false},{"nct_id":"NCT04484051","title":"Growth Hormone Study in Adults With Prader-Willi Syndrome","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":25,"lead_sponsor_name":"Erasmus Medical Center","has_results":false},{"nct_id":"NCT05298085","title":"Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome","phase":"PHASE2, PHASE3","overall_status":"COMPLETED","enrollment_count":25,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT06720571","title":"Effects of Transcutaneous Vagus Nerve Stimulation on Emotion Regulation and Executive Functioning in Prader-Willi Syndrome","phase":"NA","overall_status":"RECRUITING","enrollment_count":24,"lead_sponsor_name":"University of Bordeaux","has_results":false},{"nct_id":"NCT04700644","title":"Assessment of CAI in Adults With PWS.","phase":"","overall_status":"COMPLETED","enrollment_count":21,"lead_sponsor_name":"Medical University of Warsaw","has_results":false},{"nct_id":"NCT06279052","title":"Adolescent Projections During Transition in Prader-Willi Syndrome","phase":"NA","overall_status":"UNKNOWN","enrollment_count":20,"lead_sponsor_name":"University Hospital, Toulouse","has_results":false},{"nct_id":"NCT04597645","title":"Prader-Willi Syndrome Body Composition","phase":"NA","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation","has_results":false},{"nct_id":"NCT05153434","title":"A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":19,"lead_sponsor_name":"Aardvark Therapeutics, Inc.","has_results":false},{"nct_id":"NCT03274856","title":"A Study of GLWL-01 in Patients With Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":19,"lead_sponsor_name":"GLWL Research Inc.","has_results":true},{"nct_id":"NCT03149445","title":"Co-administration of Tesofensine/Metoprolol in Subjects With Prader-Willi Syndrome (PWS)","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":18,"lead_sponsor_name":"Saniona","has_results":true},{"nct_id":"NCT06772597","title":"A Study of Setmelanotide in Patients With Prader-Willi Syndrome","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":18,"lead_sponsor_name":"Rhythm Pharmaceuticals, Inc.","has_results":false},{"nct_id":"NCT03245762","title":"Intranasal Oxytocin for Infants With Prader-Willi Syndrome","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":15,"lead_sponsor_name":"University of Florida","has_results":true},{"nct_id":"NCT05657860","title":"Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":15,"lead_sponsor_name":"Maimonides Medical Center","has_results":false},{"nct_id":"NCT04150991","title":"Fiber Intervention on Gut Microbiota in Children With Prader-Willi Syndrome","phase":"NA","overall_status":"COMPLETED","enrollment_count":14,"lead_sponsor_name":"University of Alberta","has_results":false},{"nct_id":"NCT03848481","title":"CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":14,"lead_sponsor_name":"Montefiore Medical Center","has_results":false},{"nct_id":"NCT05541003","title":"Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"Rutgers, The State University of New Jersey","has_results":true}],"total":50},"guidelines":[],"source":"Drug Landscape verified database"}