- Conditions
- Angelman Syndrome
- Interventions
- MVX-220
- Genetic
- Lead sponsor
- MavriX Bio, LLC
- Industry
- Eligibility
- 4 Years to 50 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2031
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Chicago, Illinois • Boston, Massachusetts
Search Results
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Showing 1–24
of 33
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Angelman Syndrome
- Interventions
- OV101 Regimen 1, OV101 regimen 2, Placebo
- Drug · Other
- Lead sponsor
- Healx AI
- Industry
- Eligibility
- 13 Years to 49 Years
- Enrollment
- 88 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 12
- States / cities
- Phoenix, Arizona • San Diego, California • Gainesville, Florida + 9 more
- Conditions
- Angelman Syndrome
- Interventions
- ION582, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 50 Years
- Enrollment
- 158 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 17
- States / cities
- Los Angeles, California • San Diego, California • Aurora, Colorado + 13 more
- Conditions
- Angelman Syndrome, Prader-Willi Syndrome
- Interventions
- Newborn Screening Assay
- Diagnostic Test
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- Not listed
- Enrollment
- 11 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Microdeletion Syndromes, Trisomy 21, Trisomy 18, Trisomy 13, Sex Chromosome Abnormalities
- Interventions
- Not listed
- Lead sponsor
- Natera, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 216 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 2
- States / cities
- San Carlos, California • Philadelphia, Pennsylvania
- Conditions
- Trisomy 21, Trisomy 18, Trisomy 13, Sex Chromosome Abnormalities, Microdeletion Syndromes
- Interventions
- Not listed
- Lead sponsor
- Natera, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1,059 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2020
- U.S. locations
- 7
- States / cities
- San Carlos, California • San Gabriel, California • Washington D.C., District of Columbia + 4 more
- Conditions
- Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
- Interventions
- Gaboxadol, Placebo
- Drug
- Lead sponsor
- Healx AI
- Industry
- Eligibility
- 2 Years to 12 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • San Diego, California • Chicago, Illinois + 6 more
- Conditions
- Sleep Disorders
- Interventions
- Circadin 2/5/10 mg, Placebo
- Drug
- Lead sponsor
- Neurim Pharmaceuticals Ltd.
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 125 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 18
- States / cities
- Phoenix, Arizona • Miami Lakes, Florida • Orange City, Florida + 14 more
- Conditions
- Angelman Syndrome
- Interventions
- Levodopa/Carbidopa (4:1)
- Drug
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Angelman Syndrome
- Interventions
- Levodopa, Placebo Oral Capsule
- Drug
- Lead sponsor
- Wen-Hann Tan
- Other
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 7
- States / cities
- San Diego, California • San Francisco, California • Boston, Massachusetts + 4 more
- Conditions
- Angelman Syndrome
- Interventions
- GTX-102
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 4 Years to 17 Years
- Enrollment
- 74 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 6
- States / cities
- Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
- Conditions
- Angelman Syndrome
- Interventions
- GTX-102
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 255 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
- Conditions
- Angelman Syndrome
- Interventions
- Alogabat
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 5 Years to 17 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 6
- States / cities
- Chicago, Illinois • Boston, Massachusetts • New York, New York + 3 more
- Conditions
- Rett Syndrome, Prader-Willi Syndrome, Angelman Syndrome, Sleep Problems
- Interventions
- Not listed
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 804 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2013
- U.S. locations
- 9
- States / cities
- Birmingham, Alabama • Irvine, California • San Diego, California + 6 more
- Conditions
- Angelman Syndrome
- Interventions
- No intervention, GTX-102
- Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 1 Year to 64 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 5 more
- Conditions
- Angelman Syndrome
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 1 Year and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Lexington, Massachusetts
- 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
- Angelman Syndrome
- Interventions
- OV101
- Drug
- Lead sponsor
- Healx AI
- Industry
- Eligibility
- 13 Years to 49 Years
- Enrollment
- 141 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • San Diego, California • Atlanta, Georgia + 7 more
- Conditions
- Angelman Syndrome
- Interventions
- ION582
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 0 Years to 50 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 6
- States / cities
- San Diego, California • Aurora, Colorado • Chicago, Illinois + 3 more
- Conditions
- Angelman Syndrome
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2031
- U.S. locations
- 8
- States / cities
- Los Angeles, California • San Diego, California • Aurora, Colorado + 5 more
- Conditions
- Angelman Syndrome, Nervous System Diseases
- Interventions
- Betaine, Creatine, Metafolin, Vitamin B12
- Drug
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 1 Day to 5 Years
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2010
- U.S. locations
- 4
- States / cities
- San Diego, California • Boston, Massachusetts • Greenwood, South Carolina + 1 more
- Conditions
- Angelman Syndrome
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 1 Day to 60 Years
- Enrollment
- 302 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 6
- States / cities
- San Diego, California • Boston, Massachusetts • Cincinnati, Ohio + 3 more
- Conditions
- Angelman Syndrome
- Interventions
- Nutritional Formulation Containing Exogenous Ketones, Placebo Formulation
- Dietary Supplement
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 4 Years to 11 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Angelman Syndrome, Chromosome 15q Duplication (dup15q) Syndrome
- Interventions
- Lumbar Puncture, Blood Collection
- Procedure
- Lead sponsor
- Biogen
- Industry
- Eligibility
- Up to 50 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 5
- States / cities
- San Diego, California • Chicago, Illinois • Boston, Massachusetts + 2 more