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ClinicalTrials.gov public records Last synced Oct 1, 2026, 4:11 PM EDT

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Showing 1–24 of 246 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Mental Retardation, X Linked Clear all
Withdrawn No phase listed Observational

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Autism

NCT04100863
View directory record Official record
Conditions
Autism, Autism Spectrum Disorder, Autistic Disorder, Autism, Akinetic, Autism; Atypical, Autism; Psychopathy, Autism Fragile Syndrome X, Autism With High Cognitive Abilities, Autism-Related Speech Delay, Autism, Susceptibility to, 6, Autism Spectrum Disorder High-Functioning, Autistic Thinking, Autism, Susceptibility to, X-Linked 6, Autistic Behavior
Interventions
No Intervention
Other
Lead sponsor
ProgenaBiome
Other
Eligibility
6 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Ventura, California
Source: ClinicalTrials.gov public record
Updated Jun 5, 2025 · Synced Oct 1, 2026, 4:11 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Establishing a Diagnostic and Therapeutic Index in Autism Spectrum Disorder (ASD) and Fragile X Syndrome

NCT04869930
View directory record Official record
Conditions
Autism Spectrum Disorder, Fra(X) Syndrome
Interventions
specimen collection
Other
Lead sponsor
Nova Mentis Life Science Corp
Industry
Eligibility
6 Months to 21 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Oct 13, 2021 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 4 Interventional Results available

Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome

NCT02642653
View directory record Official record
Conditions
Fragile X Syndrome, Genetic Diseases
Interventions
Lovastatin, Placebo
Drug · Other
Lead sponsor
University of California, Davis
Other
Eligibility
10 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Sep 11, 2019 · Synced Oct 1, 2026, 4:11 PM EDT
Recruiting Phase 1Phase 2 Interventional

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease

NCT07398508
View directory record Official record
Conditions
Menkes Disease
Interventions
Droxidopa Oral Product, Placebo Control
Drug · Other
Lead sponsor
Stephen G. Kaler
Other
Eligibility
7 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 1, 2026, 4:11 PM EDT
Completed No phase listed Observational

Longitudinal Studies of Brain Structure and Function in MPS Disorders

NCT01870375
View directory record Official record
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 6, 2019 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 3 Interventional

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome

NCT01282268
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
arbaclofen, placebo
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
24
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2013 · Synced Oct 1, 2026, 4:11 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 2 Interventional

Study of ANAVEX2-73 in Patients With Rett Syndrome

NCT03758924
View directory record Official record
Conditions
Rett Syndrome
Interventions
ANAVEX2-73, Placebo
Drug
Lead sponsor
Anavex Life Sciences Corp.
Industry
Eligibility
18 Years to 45 Years · Female only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
8
States / cities
Birmingham, Alabama • Sacramento, California • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2021 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01253629
View directory record Official record
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 23, 2020 · Synced Oct 1, 2026, 4:11 PM EDT
Completed No phase listed Observational

Natural History of Rett Syndrome & Related Disorders

NCT02738281
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
1,044 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2021 · Synced Oct 1, 2026, 4:11 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age

NCT05957549
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Speech discrimination
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
24 Months to 4 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 10, 2025 · Synced Oct 1, 2026, 4:11 PM EDT
Recruiting No phase listed Observational

Adrenoleukodystrophy National Registry Study

NCT03789721
View directory record Official record
Conditions
ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Interventions
Medical Record Abstraction, Biospecimen Sample Collection
Other
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 2 Interventional Results available

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526
View directory record Official record
Conditions
Sanfilippo Syndrome
Interventions
Recombinant human heparan N-sulfatase [rhHNS]
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Torrance, California • Minneapolis, Minnesota • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Oct 1, 2026, 4:11 PM EDT
Completed No phase listed Observational

Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment

NCT01364818
View directory record Official record
Conditions
Autism Spectrum Disorders, Psychosis, Fragile X Syndrome
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
3 Years to 45 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 18, 2016 · Synced Oct 1, 2026, 4:11 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-up on Menkes Disease Patients

NCT04337684
View directory record Official record
Conditions
Menkes Disease
Interventions
Long Term Follow-Up
Drug
Lead sponsor
Sentynl Therapeutics, Inc.
Industry
Eligibility
Not listed
Enrollment
50 participants
Timeline
2019 – 2026
U.S. locations
1
States / cities
Solana Beach, California
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 1, 2026, 4:11 PM EDT
Completed No phase listed Observational

ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME

NCT04514549
View directory record Official record
Conditions
Rett Syndrome
Interventions
Emerald
Device
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Female only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 1, 2026, 4:11 PM EDT
Completed No phase listed Observational

The Role of Family Functioning in Adaptation to Being a Caregiver of an Individual With Rett Syndrome

NCT00891956
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
426 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
3
States / cities
Birmingham, Alabama • Baltimore, Maryland • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Not applicable Interventional Results available

Trial of Minocycline to Treat Children With Fragile X Syndrome

NCT01053156
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
minocycline hydrochloride, Placebo
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
42 Months to 16 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 1, 2026, 4:11 PM EDT
Not listed No phase listed Observational

The Natural History of Danon Disease

NCT03766386
View directory record Official record
Conditions
Danon Disease
Interventions
Not listed
Lead sponsor
University of California, San Diego
Other
Eligibility
0 Years to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated May 12, 2022 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 1, 2026, 4:11 PM EDT
Completed Phase 2 Interventional

A Study With RO4917523 in Patients With Fragile X Syndrome

NCT01015430
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Placebo (for RO4917523 ascending doses), Placebo (for RO4917523 fixed dose), RO4917523
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 50 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
5
States / cities
Sacramento, California • Decatur, Georgia • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 2, 2016 · Synced Oct 1, 2026, 4:11 PM EDT
Terminated 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 – 2026
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 1, 2026, 4:11 PM EDT
Recruiting No phase listed Observational

Brain Development Research Program

NCT00305305
View directory record Official record
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 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 1, 2026, 4:11 PM EDT