- Conditions
- Prader-Willi Syndrome, Excessive Daytime Sleepiness, Hyperphagia, Body Weight, Mood, Behavior
- Interventions
- Sham Light, Bright Light Therapy
- Other
- Lead sponsor
- Maimonides Medical Center
- Other
- Eligibility
- 6 Years to 88 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Brooklyn, New York
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- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Prader-Willi Syndrome
- Interventions
- RGH-706, Placebo
- Drug
- Lead sponsor
- Gedeon Richter Plc.
- Industry
- Eligibility
- 17 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 6
- States / cities
- San Diego, California • Chicago, Illinois • Brooklyn, New York + 3 more
- Conditions
- Prader-Willi Syndrome
- Interventions
- Cannabidiol, Placebo
- Drug
- Lead sponsor
- Radius Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Years to 17 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 7
- States / cities
- Tucson, Arizona • San Diego, California • Iowa City, Iowa + 4 more
- Conditions
- Prader Willi Syndrome
- Interventions
- Intranasal oxytocin, Placebo
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 5 Years to 11 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015
- U.S. locations
- 3
- States / cities
- Orange, California • Gainesville, Florida • Kansas City, Kansas
- Conditions
- Prader-Willi Syndrome
- Interventions
- DCCR
- Drug
- Lead sponsor
- Soleno Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 17
- States / cities
- Orange, California • Palo Alto, California • San Diego, California + 14 more
- Conditions
- Intellectual Disability, Neurodevelopmental Disorders, Autism Spectrum Disorder, Down Syndrome, Fragile X Syndrome, Cri-du-Chat Syndrome, De Lange Syndrome, Mental Retardation, X-Linked, Prader-Willi Syndrome, Rubinstein-Taybi Syndrome, Trisomy 13 Syndrome, WAGR Syndrome, Williams Syndrome
- Interventions
- PACE Program
- Behavioral
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 376 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2028
- U.S. locations
- 2
- States / cities
- Fayetteville, Arkansas • Chapel Hill, North Carolina
- Conditions
- Prader-Willi Syndrome
- Interventions
- RAD011, Placebo
- Drug
- Lead sponsor
- Radius Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Years to 65 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 8
- States / cities
- Los Angeles, California • San Diego, California • Iowa City, Iowa + 5 more
- Conditions
- Hyperphagia in Prader-Willi Syndrome
- Interventions
- Carbetocin
- Drug
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Industry
- Eligibility
- 5 Years to 30 Years
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Orange, California + 16 more
- Conditions
- Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature, Healthy
- Interventions
- somatropin
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years to 40 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011
- U.S. locations
- 1
- States / cities
- Evansville, Indiana
- Conditions
- Obesity, Over-weight, Prader-Willi Syndrome
- Interventions
- ZGN-440 sterile diluent, 1.2 mg ZGN-440 for injectable suspension, 1.8 mg ZGN-440 for injectable suspension
- Drug
- Lead sponsor
- Zafgen, Inc.
- Industry
- Eligibility
- 16 Years to 65 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Prader-Willi Syndrome
- Interventions
- ARD-101
- Drug
- Lead sponsor
- Aardvark Therapeutics, Inc.
- Industry
- Eligibility
- 17 Years to 65 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 2
- States / cities
- Palo Alto, California • Aurora, Colorado
- Conditions
- Prader-Willi Syndrome
- Interventions
- Nutropin AQ
- Drug
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Other
- Eligibility
- 16 Years to 60 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2012
- U.S. locations
- 1
- States / cities
- Kansas City, Missouri
- Conditions
- Prader-Willi Syndrome
- Interventions
- Regulating Together group therapy
- Behavioral
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Other
- Eligibility
- 13 Years to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Kansas City, Missouri
- Conditions
- Prader Willi Syndrome, Obsessive-compulsive Disorder
- Interventions
- Cognitive-behavioral therapy, Wait-list control
- Behavioral
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- St. Petersburg, Florida
- Conditions
- Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
- Interventions
- Tirzepatide
- Drug
- Lead sponsor
- Grace Kim
- Other
- Eligibility
- 18 Years to 26 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 3
- States / cities
- Saint Paul, Minnesota • Nashville, Tennessee • Seattle, Washington
- Conditions
- Prader-Willi Syndrome
- Interventions
- VYKAT XR
- Drug
- Lead sponsor
- Soleno Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 15
- States / cities
- Los Angeles, California • Atlanta, Georgia • Chicago, Illinois + 12 more
- Conditions
- Prader-Willi Syndrome
- Interventions
- DCCR, Placebo for DCCR
- Drug
- Lead sponsor
- Soleno Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 127 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 20
- States / cities
- Orange, California • Palo Alto, California • San Diego, California + 17 more
- Conditions
- Hyperphagia, Prader-Willi Syndrome
- Interventions
- Placebo, ARD-101
- Drug
- Lead sponsor
- Aardvark Therapeutics, Inc.
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 17
- States / cities
- Birmingham, Alabama • Encinitas, California • Orange, California + 14 more
- Conditions
- Prader-Willi Syndrome
- Interventions
- Octreotide, Placebo
- Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- 5 Years to 21 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2010
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Prader-Willi Syndrome
- Interventions
- Interview
- Other
- Lead sponsor
- Soleno Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 59 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Plymouth, Massachusetts
- Conditions
- Prader-Willi Syndrome, Obesity
- Interventions
- NBT-NM108
- Drug
- Lead sponsor
- Rutgers, The State University of New Jersey
- Other
- Eligibility
- 18 Years to 35 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- New Brunswick, New Jersey
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
- Interventions
- Parent-Infant Inter(X)action Intervention (PIXI)
- Behavioral
- Lead sponsor
- RTI International
- Other
- Eligibility
- Up to 99 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Interventions
- Part A: RM-718 or placebo (matched to specific RM-718 dose cohort), Part B: RM-718 or placebo (matched to specific RM-718 dose cohort), Part C: RM-718, Part D: RM-718
- Drug
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2028
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Chicago, Illinois • Boston, Massachusetts + 3 more