Independent directory Public ClinicalTrials.gov records United States
Clear filters
ClinicalTrials.gov public records Last synced Jul 21, 2026, 7:11 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Phelan-McDermid Syndrome, Autism Spectrum Disorder, Intellectual Disability
Interventions
No Intervention
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
22 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
6
States / cities
Stanford, California • Chicago, Illinois • Bethesda, Maryland + 3 more
Conditions
Fragile X Syndrome
Interventions
placebo, oxytocin 24IU, oxytocin 48IU
Drug
Lead sponsor
Stanford University
Other
Eligibility
13 Years to 29 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Stanford, California
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Down Syndrome Memantine Follow-up Study

NCT02304302
Conditions
Down Syndrome, Intellectual Disability
Interventions
Memantine, Placebo
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
15 Years to 32 Years
Enrollment
160 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
1
States / cities
Cleveland, Ohio
Conditions
Fragile X Syndrome
Interventions
AFQ056, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 17 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
8
States / cities
Sacramento, California • Decatur, Georgia • Chicago, Illinois + 5 more
Conditions
Asthma in Children, Intellectual Disabilities (F70-F79), Developmental Disability, Autism Spectrum Disorder (ASD)
Interventions
Use of smart inhaler to enhance asthma inhaler proficiency
Behavioral
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
10 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Down Syndrome (Trisomy 21), Edwards Syndrome (Trisomy 18), Patau Syndrome (Trisomy 13), Turner Syndrome
Interventions
Not listed
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
7
States / cities
La Jolla, California • San Diego, California • San Jose, California + 4 more
Not listed No phase listed Observational Accepts healthy volunteers

Glucose Metabolic, Amyloid, and Tau Brain Imaging in Down Syndrome and Dementia

NCT00966017
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
National Institute on Aging (NIA)
NIH
Eligibility
45 Years and older
Enrollment
108 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Obesity, Prader Willi Syndrome
Interventions
High carbohydrate meal, High fat meal
Other
Lead sponsor
Duke University
Other
Eligibility
5 Years to 17 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Epilepsy
Interventions
Divalproex
Drug
Lead sponsor
University of Kansas
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Kansas City, Kansas
Conditions
Alzheimer's Disease in Down Syndrome
Interventions
Not listed
Lead sponsor
LuMind IDSC Foundation
Other
Eligibility
25 Years and older
Enrollment
252 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
14
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 11 more
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
1 Year to 64 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Down Syndrome, Intellectual Disability
Interventions
Memantine
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
15 Years to 32 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cleveland, Ohio
Conditions
Creatine Deficiency, X-linked
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Months to 65 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
9
States / cities
La Jolla, California • Chicago, Illinois • Bethesda, Maryland + 6 more
Conditions
Developmental Delay Disorders, Chromosome Deletion, Mental Retardation, Sleep Disorders, Circadian Rhythm, Self Injurious Behavior
Interventions
dTR Melatonin (NIH CC PDS), Phototherapy (Bright Light), Melatonin CR
Drug · Device
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
3 Years to 45 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Problem Behavior, Intellectual Disability, Developmental Disorder
Interventions
Functional communication training
Behavioral
Lead sponsor
Western Kentucky University
Other
Eligibility
3 Years to 11 Years
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
Bowling Green, Kentucky
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 8 Days
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Fragile X Syndrome, Autism Spectrum Disorder, Autistic Disorder, Asperger Syndrome
Interventions
Alpha Auditory Entrainment, Sham
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years to 10 Years
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Approved For Marketing No phase listed Expanded access

Copper Histidinate Treatment for Menkes Disease

NCT04074512
Conditions
Menkes Disease
Interventions
copper histidinate
Drug
Lead sponsor
Sentynl Therapeutics, Inc.
Industry
Eligibility
0 Years to 6 Years
U.S. locations
20
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 17 more
Recruiting No phase listed Observational Accepts healthy volunteers

Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome

NCT07569445
Conditions
Rett Syndrome
Interventions
Electroencephalogram (EEG) Recording
Procedure
Lead sponsor
University of Rochester
Other
Eligibility
2 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Rochester, New York
Conditions
Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
Interventions
Epi-ON corneal cross-linking (CXL)
Combination Product
Lead sponsor
Woolfson Eye Institute
Other
Eligibility
8 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
12
States / cities
Atlanta, Georgia • Canton, Georgia • Cumming, Georgia + 8 more
Completed Phase 2 Interventional Results available

ERG/5-HTP in Fragile X Syndrome (FXS)

NCT05030129
Conditions
Fragile X Syndrome
Interventions
5-Hydroxytryptophan, Ergoloid Mesylates, Matching placebo for Ergoloid mesylates, Matching placebo for 5-Hydroxytryptophan
Drug
Lead sponsor
Elizabeth Berry-Kravis
Other
Eligibility
18 Years to 45 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Completed No phase listed Observational Accepts healthy volunteers

Development of the ORCA Communication Measure for Rett Syndrome

NCT04920110
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
18 Years and older
Enrollment
272 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Down Syndrome, Intellectual Disability, Sexuality, Parent-Child Relations
Interventions
Parent training
Behavioral
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Park Ridge, Illinois
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