Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Jun 25, 2026, 4:08 PM EDT

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Showing 1–24 of 628 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Fragile X Syndrome
Interventions
arbaclofen, Placebo
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
5 Years to 11 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
28
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 24 more
Conditions
Prader-Willi Syndrome, Obesity
Interventions
ZGN-440 for Injectable Suspension, ZGN-440 Placebo for Injectable Suspension
Drug
Lead sponsor
Zafgen, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
15
States / cities
Sacramento, California • San Diego, California • Aurora, Colorado + 12 more
Conditions
Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
Interventions
Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Conditions
Cerebral Palsy, Neurological Conditions, Mental Retardation, Sialorrhea
Interventions
Oral Glycopyrrolate Liquid
Drug
Lead sponsor
Shionogi
Industry
Eligibility
3 Years to 18 Years
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
7
States / cities
Lakewood, Colorado • Atlanta, Georgia • Bayside, New York + 4 more
Conditions
Fragile X Syndrome
Interventions
MG01CI extended-release tablet
Drug
Lead sponsor
Alcobra Ltd.
Industry
Eligibility
15 Years to 55 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
11
States / cities
Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 8 more
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
Conditions
Hypothyroidism, Trisomy 21
Interventions
Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
Drug
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
2 Months to 4 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
Kansas City, Missouri
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
Conditions
Down Syndrome, Sleep Disordered Breathing
Interventions
Adenotonsillectomy
Procedure
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
3 Years to 13 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Ann Arbor, Michigan • Cincinnati, Ohio • Cleveland, Ohio + 3 more
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Conditions
Down Syndrome
Interventions
ACI-24 low dose, ACI-24 high dose, Placebo
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
25 Years to 45 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
4
States / cities
Phoenix, Arizona • La Jolla, California • Baltimore, Maryland + 1 more
Conditions
Prader-Willi Syndrome, Obesity
Interventions
Group 1, Group 2
Other
Lead sponsor
University of Florida
Other
Eligibility
Up to 60 Years
Enrollment
392 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
4
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 1 more
Conditions
Rett Syndrome
Interventions
dextromethorphan, donepezil hydrochloride
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Year to 15 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Down Syndrome
Interventions
Ubiquinol-10 Syrup, syrup (placebo)
Dietary Supplement
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 16 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Conditions
Fragile X Syndrome, Intellectual Disability
Interventions
Social gaze training
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
8 Years to 18 Years · Male only
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Stanford, California
Conditions
Prader-Willi Syndrome, Obesity, Hyperphagia
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Gainesville, Florida
Completed Not applicable Interventional Accepts healthy volunteers

A Family-Based Weight Loss Intervention for Youth With Intellectual Disability

NCT02033642
Conditions
Obesity
Interventions
Family Based Behavioral Intervention, Maintenance
Behavioral
Lead sponsor
University of Massachusetts, Boston
Other
Eligibility
14 Years to 22 Years
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
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
Conditions
Aggressive Behavior in Fragile X Syndome
Interventions
SRX246, Placebo
Drug
Lead sponsor
Azevan Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Conditions
Prader-Willi Syndrome
Interventions
Placebo, Tesomet
Other · Drug
Lead sponsor
Saniona
Industry
Eligibility
13 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Lansing, Michigan
Conditions
Rett Syndrome
Interventions
NGN-401
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years and older · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
13
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 10 more
Conditions
Hunter Syndrome
Interventions
No treatment
Other
Lead sponsor
Shire
Industry
Eligibility
Up to 70 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more