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Showing 1–24 of 1,254 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 3 Interventional

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome

NCT01325220
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 30, 2013 · Synced Jun 26, 2026, 12:42 AM EDT
Terminated Phase 3 Interventional

Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome

NCT02179151
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 25, 2017 · Synced Jun 26, 2026, 12:42 AM EDT
Recruiting Not applicable Interventional

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

NCT06139172
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jun 26, 2026, 12:42 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy Study of Oral Glycopyrrolate Liquid for the Treatment of Pathologic (Chronic Moderate to Severe) Drooling in Pediatric Patients 3 to 18 Years of Age With Cerebral Palsy or Other Neurologic Conditions

NCT00491894
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 8, 2012 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Phase 2 Interventional

A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome

NCT02126995
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 5, 2016 · Synced Jun 26, 2026, 12:42 AM EDT
Approved For Marketing No phase listed Expanded access

Copper Histidinate Treatment for Menkes Disease

NCT04074512
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Jun 26, 2026, 12:42 AM EDT
Terminated Phase 4 Interventional Results available

Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients

NCT04747275
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced Jun 26, 2026, 12:42 AM EDT
Recruiting Phase 3 Interventional Accepts healthy volunteers

Exploring Sympathetic Nervous System Function in Individuals With Down Syndrome

NCT07238465
View directory record Official record
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 Jun 26, 2026, 12:42 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Investigating Phenotypic, Epigenetic, and NeuroGenetic Traits in Rare and Ultra-rare Neurodevelopmental Disorders (Project PENGUIN)

NCT07329257
View directory record Official record
Conditions
Baker Gordon Syndrome, Rare Neurodevelopmental Conditions, Rare Neurogenetic Conditions, Syt-1 Disorder, Epilepsy, Seizure, Genetic Mutations, Autism in Children, Developmental Delay (Disorder)
Interventions
No Intervention: Observational Cohort
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
Up to 99 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jun 26, 2026, 12:42 AM EDT
Completed No phase listed Observational

Healthy Sleep for Children With Down Syndrome

NCT03922165
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 1, 2022 · Synced Jun 26, 2026, 12:42 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Aspirin in Reducing Events in the Elderly

NCT01038583
View directory record Official record
Conditions
Functional Disability, Dementia, Heart Disease, Stroke, Cancer, Bleeding, Depression
Interventions
100 mg enteric-coated aspirin, Placebo
Drug
Lead sponsor
Hennepin Healthcare Research Institute
Other
Eligibility
65 Years and older
Enrollment
19,114 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2024
U.S. locations
33
States / cities
Birmingham, Alabama • Palo Alto, California • Washington D.C., District of Columbia + 26 more
Source: ClinicalTrials.gov public record
Updated Apr 4, 2021 · Synced Jun 26, 2026, 12:42 AM EDT
Recruiting No phase listed Observational

Characterization of Dysmorphology in Subjects With Creatine Transporter Deficiency

NCT05600946
View directory record Official record
Conditions
Cognitive Disorder, Metabolic Disease, Autism Spectrum Disorder
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 40 Years · Male only
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Jun 26, 2026, 12:42 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Eye Movements and Reading Disabilities

NCT01860027
View directory record Official record
Conditions
Reading Disability, Dyslexia
Interventions
Not listed
Lead sponsor
Scripps Health
Other
Eligibility
7 Years to 13 Years
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Nov 21, 2023 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Retrieval-Based Word Learning in Developmental Language Disorder During Book Reading

NCT06026124
View directory record Official record
Conditions
Developmental Language Disorder, Specific Language Impairment, Language Development
Interventions
Retrieval-based word learning: Repeated Spaced Retrieval condition, Retrieval-based word learning: Repeated Study condition
Behavioral
Lead sponsor
Purdue University
Other
Eligibility
48 Months to 71 Months
Enrollment
27 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
2
States / cities
Indianapolis, Indiana • West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Phase 1 Interventional Results available

Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome

NCT02738450
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 14, 2021 · Synced Jun 26, 2026, 12:42 AM EDT
Terminated No phase listed Observational

PLAY LONG: The Effect of Sports and Recreation Participation for Young chiLdren With physicAl disabilitY

NCT03256318
View directory record Official record
Conditions
Physical Disability, Quality of Life, Social Interaction, Health, Subjective
Interventions
No intervention
Other
Lead sponsor
Allina Health System
Other
Eligibility
5 Years to 10 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Golden Valley, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 5, 2020 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Not applicable Interventional Results available

A Novel Transition Program to Reduce Disability After Stroke

NCT03485820
View directory record Official record
Conditions
Ischemic Stroke, Hemorrhagic Stroke
Interventions
COMPASS, Stroke education
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
50 Years and older
Enrollment
185 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 26, 2025 · Synced Jun 26, 2026, 12:42 AM EDT
Completed No phase listed Observational

Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity

NCT00375089
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 21, 2014 · Synced Jun 26, 2026, 12:42 AM EDT
Not listed Phase 3 Interventional

Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome

NCT00069550
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jun 26, 2026, 12:42 AM EDT
Withdrawn Phase 2 Interventional

Liq-NOL Efficacy in Pediatric Patients With Down Syndrome

NCT00891917
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 8, 2020 · Synced Jun 26, 2026, 12:42 AM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 2, 2024 · Synced Jun 26, 2026, 12:42 AM EDT
Completed Phase 1Phase 2 Interventional

Effects of Social Gaze Training on Brain and Behavior in Fragile X Syndrome

NCT02616796
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 8, 2023 · Synced Jun 26, 2026, 12:42 AM EDT