Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 7, 2026, 4:08 AM EDT

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Showing 1–10 of 10 matching trials from the live ClinicalTrials.gov search.
Conditions
Developmental Disability, Intellectual Disability, Autism Spectrum Disorder, Congenital Anomaly, Fragile X Syndrome, Facioscapulohumeral Muscular Dystrophy 1
Interventions
Standard of care genetic testing group
Other
Lead sponsor
Bionano Genomics
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
8
States / cities
Atlanta, Georgia • Augusta, Georgia • Iowa City, Iowa + 5 more
Recruiting No phase listed Observational

Rett Syndrome Registry

NCT05432349
Conditions
Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked, Intellectual Disability, Neurobehavioral Manifestations, Neurologic Manifestations, Neurologic Disorder, Neurodevelopmental Disorders, Nervous System Diseases
Interventions
Not listed
Lead sponsor
International Rett Syndrome Foundation
Other
Eligibility
0 Years to 99 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • Oakland, California + 16 more
Conditions
Rett Syndrome, MECP2 Duplication Disorder, Rett-related Disorder
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 8 more
Conditions
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Interventions
Enzyme testing
Other
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Completed Phase 2Phase 3 Interventional Results available

A Trial of Levodopa in Angelman Syndrome

NCT01281475
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
Completed No phase listed Observational

Characterization of Angelman Syndrome

NCT00296764
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
Completed No phase listed Observational Accepts healthy volunteers

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW

NCT02670694
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
Aspartylglucosaminuria, Fucosidosis, Galactosialidosis, Alpha Mannosidosis, Beta Mannosidosis, Mucolipidosis II, Mucolipidosis III, Schindler Disease, Sialidosis
Interventions
Non-interventional.
Other
Lead sponsor
Greenwood Genetic Center
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
North Charleston, South Carolina
Conditions
Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
Interventions
Not listed
Lead sponsor
Greenwood Genetic Center
Other
Eligibility
Up to 18 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Greenville, South Carolina
Completed Not applicable Interventional Results available

Dietary Supplements for the Treatment of Angelman Syndrome

NCT00348933
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