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Showing 1–24 of 524 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 25, 2026, 11:07 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 25, 2026, 11:07 AM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06617429
View directory record Official record
Conditions
Angelman Syndrome
Interventions
GTX-102, Sham-LP
Drug · Procedure
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
4 Years to 17 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Jun 25, 2026, 11:07 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 25, 2026, 11:07 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 25, 2026, 11:07 AM EDT
Recruiting No phase listed Observational

UTHealth Turner Syndrome Research Registry

NCT03185702
View directory record Official record
Conditions
Turner Syndrome
Interventions
Research genetic tests
Genetic
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Female only
Enrollment
200 participants
Timeline
2015 – 2035
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 27, 2023 · Synced Jun 25, 2026, 11:07 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 25, 2026, 11:07 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 25, 2026, 11:07 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 25, 2026, 11:07 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Trial of Levodopa in Angelman Syndrome

NCT01281475
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 14, 2020 · Synced Jun 25, 2026, 11:07 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

GEM: Impact of a Video Education Tool on Decisional Conflict Among Prenatal Patients

NCT06771453
View directory record Official record
Conditions
Aneuploidy, Genetic Counseling, Informed Consent, Decisional Conflict
Interventions
Video Educational Tool Regarding Prenatal Genetics
Other
Lead sponsor
Women and Infants Hospital of Rhode Island
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Sep 17, 2025 · Synced Jun 25, 2026, 11:07 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 25, 2026, 11:07 AM EDT
Terminated No phase listed Observational

Development of a NIPTT for Detecting Copy Number Variations

NCT04774640
View directory record Official record
Conditions
Copy Number Variations
Interventions
MaterniT GENOME
Device
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years to 89 Years · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 8, 2023 · Synced Jun 25, 2026, 11:07 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 25, 2026, 11:07 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 25, 2026, 11:07 AM EDT
Completed Phase 4 Interventional Results available

Body Composition in Infants With Klinefelter Syndrome and Effects of Testosterone Treatment

NCT02408445
View directory record Official record
Conditions
Klinefelter Syndrome
Interventions
testosterone cypionate 200mg/ml
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
42 Days to 108 Days · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 14, 2020 · Synced Jun 25, 2026, 11:07 AM EDT
Completed Phase 3 Interventional Results available

A Multicenter Phase 3, Open-Label Study of Bosutinib Versus Imatinib in Adult Patients With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia

NCT02130557
View directory record Official record
Conditions
Leukemia, Myelogenous, Chronic, Breakpoint Cluster Region-Abelson Proto-oncogene (BCR-ABL) Positive
Interventions
Bosutinib, Imatinib
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
536 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
65
States / cities
Anaheim, California • Atlanta, Georgia • Honolulu, Hawaii + 40 more
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Jun 25, 2026, 11:07 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 25, 2026, 11:07 AM EDT
Terminated Phase 2 Interventional Results available

Pegasys in Patients With Chronic Myeloid Leukemia (CML)

NCT01392170
View directory record Official record
Conditions
Leukemia
Interventions
PEG-IFNá-2a
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
16 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 3, 2015 · Synced Jun 25, 2026, 11:07 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Aneuploidy Rates in Advanced Maternal Age Patients Supplemented With Coenzyme Q10 (CoQ10) Versus Those That Are Not: a Pilot Study

NCT02119117
View directory record Official record
Conditions
Mitochondrial DNA, Aneuploidy
Interventions
CoQ10, Placebo
Dietary Supplement
Lead sponsor
Reproductive Endocrinology Associates of Charlotte
Other
Eligibility
36 Years to 42 Years · Female only
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated May 3, 2018 · Synced Jun 25, 2026, 11:07 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

NCT06772597
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Jun 25, 2026, 11:07 AM EDT
Completed Phase 3 Interventional

Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

NCT03714373
View directory record Official record
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 Jun 25, 2026, 11:07 AM EDT
Not yet recruiting Phase 2 Interventional

Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome

NCT07328529
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jun 25, 2026, 11:07 AM EDT
Withdrawn Phase 2 Interventional

Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome

NCT05198362
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 12, 2022 · Synced Jun 25, 2026, 11:07 AM EDT