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ClinicalTrials.gov public records Last synced May 21, 2026, 10:42 PM EDT

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

Showing 1–24 of 606 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Down Syndrome, Dysphagia, Oropharyngeal
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
3 Years to 18 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 25, 2024 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
752 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D Drug Product (eli-cel)
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced May 21, 2026, 10:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
Conditions
X-linked Adrenoleukodystrophy
Interventions
exercise training
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
21 Years to 70 Years · Female only
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Prader-Willi Syndrome
Interventions
tVNS, intermittent stimulation, tVNS, continuous stimulation
Device
Lead sponsor
Foundation for Prader-Willi Research
Other
Eligibility
10 Years to 40 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
18
States / cities
Palo Alto, California • San Diego, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Tardive Dyskinesia, Intellectual Disability, Developmental Disabilities
Interventions
Deutetrabenazine Oral Capsule
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years to 89 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Fragile X Syndrome
Interventions
Sertraline, Placebo
Drug
Lead sponsor
Randi J. Hagerman, MD
Other
Eligibility
24 Months to 68 Months
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Sep 24, 2017 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Rett Syndrome
Interventions
Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 10 Years · Female only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2018 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Parents, Stress, Developmental Disability, Intellectual Disability, Autism Spectrum Disorder
Interventions
Mindfulness-Based Music and Songwriting
Behavioral
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 31, 2022 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Fragile X Syndrome
Interventions
BPN14770/ zatolmilast, Placebo
Drug
Lead sponsor
Tetra Discovery Partners
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
171 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
17
States / cities
Irvine, California • Orange, California • Sacramento, California + 14 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Mental Retardation
Interventions
BIS monitor
Device
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
12 Years to 65 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 31, 2016 · Synced May 21, 2026, 10:42 PM EDT
Not listed No phase listed Observational
Conditions
Brain Disorders, Aicardi Syndrome
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2025
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 9, 2023 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
2 Years to 5 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Prader-Willi Syndrome
Interventions
DCCR, Placebo for DCCR
Drug
Lead sponsor
Soleno Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
20
States / cities
Orange, California • Palo Alto, California • San Diego, California + 17 more
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced May 21, 2026, 10:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
Conditions
Down Syndrome
Interventions
Spatial Ability Experience
Behavioral
Lead sponsor
University of Alabama, Tuscaloosa
Other
Eligibility
4 Years to 25 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Tuscaloosa, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Fragile X Syndrome
Interventions
AFQ056, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
12
States / cities
Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Prader-willi Syndrome
Interventions
rimonabant, placebo
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 19, 2023 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Cornelia de Lange Syndrome
Interventions
Autonomic Dysfunction Testing
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
8 Years to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2018 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Down Syndrome, Autonomic Dysfunction
Interventions
Fear Response, Cold Stress, Pain Response, Caffeine, 12-Hour Fast, Maximal Dynamic Exercise
Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 50 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Down Syndrome, Obstructive Sleep Apnea
Interventions
Open-ended semi-structured interviews
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
6 Years to 17 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 24, 2023 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Down Syndrome
Interventions
Treadmill Training, Supramalleolar Orthoses
Behavioral · Device
Lead sponsor
University of Michigan
Other
Eligibility
6 Months to 36 Months
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 3, 2017 · Synced May 21, 2026, 10:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
Conditions
Language Development Disorders, Down Syndrome, Child Language
Interventions
Semantic cues (perform action), syntactic cues (sentence frame), or combined semantic and syntactic cues
Behavioral
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
3 Years to 17 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 6, 2024 · Synced May 21, 2026, 10:42 PM EDT
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 10:42 PM EDT