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ClinicalTrials.gov public records Last synced Oct 3, 2026, 10:59 AM EDT

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Showing 25–48 of 246 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Mental Retardation, X-Linked Clear all
Completed Phase 3 Interventional Results available

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

NCT04279314
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
5 Years to 21 Years · Female only
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2024 · Synced Oct 3, 2026, 10:59 AM EDT
Completed No phase listed Observational Results available

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

NCT01449240
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 3, 2026, 10:59 AM EDT
Recruiting Phase 1Phase 2 Interventional

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

NCT06430385
View directory record Official record
Conditions
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
ION440, Sham procedure
Drug · Procedure
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
8
States / cities
San Diego, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 1Phase 2 Interventional

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
5 Years to 17 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

NCT00920647
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
Other · Drug
Lead sponsor
Shire
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Oct 3, 2026, 10:59 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
ZYN002 - Cannabidiol Transdermal Gel
Drug
Lead sponsor
Zynerba Pharmaceuticals, Inc.
Industry
Eligibility
3 Years to 29 Years
Enrollment
476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
19
States / cities
Phoenix, Arizona • Culver City, California • Irvine, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 1 Interventional

Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

NCT01482143
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
3 Years to 11 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 8, 2020 · Synced Oct 3, 2026, 10:59 AM EDT
Completed No phase listed Observational

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

NCT01938014
View directory record Official record
Conditions
Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
1 Day to 18 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2019 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747
View directory record Official record
Conditions
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
16
States / cities
Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 3, 2026, 10:59 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis Type II Natural History

NCT03529786
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 26, 2022 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Not applicable Interventional

Teaching Math Skills to Individuals With Fragile X Syndrome

NCT01204151
View directory record Official record
Conditions
Developmental Disabilities, Fragile X Syndrome
Interventions
discrete-trial training
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
10 Years to 23 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 9, 2019 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 2 Interventional Results available

ERG/5-HTP in Fragile X Syndrome (FXS)

NCT05030129
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
5-Hydroxytryptophan, Ergoloid Mesylates, Matching placebo for Ergoloid mesylates, Matching placebo for 5-Hydroxytryptophan
Drug
Lead sponsor
Elizabeth Berry-Kravis
Other
Eligibility
18 Years to 45 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 3, 2026, 10:59 AM EDT
Not listed No phase listed Observational

A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

NCT03278899
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy
Interventions
Not listed
Lead sponsor
NeuroVia, Inc.
Industry
Eligibility
2 Years to 13 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 8, 2018 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 2 Interventional Results available

Ganaxolone Treatment in Children With Fragile X Syndrome

NCT01725152
View directory record Official record
Conditions
Fragile x Syndrome
Interventions
Ganaxolone, Placebo
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
6 Years to 17 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Apr 10, 2023 · Synced Oct 3, 2026, 10:59 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Aromatase Activity and Ovarian Growth Factors in African-American Versus Caucasian Women

NCT00334971
View directory record Official record
Conditions
Healthy, Fragile X Syndrome
Interventions
Follicle Aspiration
Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
116 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 6, 2013 · Synced Oct 3, 2026, 10:59 AM EDT
Recruiting Phase 2 Interventional

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
3
States / cities
La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Oct 3, 2026, 10:59 AM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Genetic HMI-203
Biological
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 45 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
5
States / cities
Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2023 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 3 Interventional

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

NCT05358886
View directory record Official record
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 10, 2026 · Synced Oct 3, 2026, 10:59 AM EDT
Terminated Phase 2 Interventional Results available

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01348087
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
10
States / cities
Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated May 25, 2016 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

NCT01043640
View directory record Official record
Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions
Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 5, 2018 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 2 Interventional Results available

Randomized Controlled Study of Donepezil in Fragile X Syndrome

NCT01120626
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
donepezil, sugar pill
Drug
Lead sponsor
Stanford University
Other
Eligibility
12 Years to 29 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2016 · Synced Oct 3, 2026, 10:59 AM EDT
Withdrawn Phase 1 Interventional

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

NCT01787578
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
Interventions
Sobetirome
Drug
Lead sponsor
Thomas S. Scanlan
Other
Eligibility
18 Years to 64 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 14, 2014 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107

NCT00965432
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
STX107
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 8, 2010 · Synced Oct 3, 2026, 10:59 AM EDT
Completed Phase 3 Interventional Results available

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 24, 2024 · Synced Oct 3, 2026, 10:59 AM EDT