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ClinicalTrials.gov public records Last synced Oct 9, 2026, 4:30 PM EDT

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Showing 49–72 of 246 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Mental Retardation, X Linked Clear all
Terminated Phase 3 Interventional Results available

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

NCT04252586
View directory record Official record
Conditions
Rett Syndrome, RTT
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • La Jolla, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 8, 2022 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting No phase listed Observational

Cognitive Function in Rett Syndrome During Trofinetide Treatment

NCT06705816
View directory record Official record
Conditions
Rett Syndrome
Interventions
None interventional
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 99 Years
Enrollment
20 participants
Timeline
2024 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101 Treatment
Genetic
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Completed Phase 2 Interventional Results available

A Study of Rucaparib in Patients With Metastatic Castration-resistant Prostate Cancer and Homologous Recombination Gene Deficiency

NCT02952534
View directory record Official record
Conditions
Metastatic Castration Resistant Prostate Cancer
Interventions
Rucaparib
Drug
Lead sponsor
pharmaand GmbH
Industry
Eligibility
18 Years and older · Male only
Enrollment
277 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
58
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 47 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2023 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Speech-in-noise Perception in Autism and Fragile X

NCT06088589
View directory record Official record
Conditions
Autism Spectrum Disorder, Fragile X Syndrome
Interventions
Mismatch negativity
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
15 Years to 35 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Terminated No phase listed Observational

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674
View directory record Official record
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2013 · Synced Oct 9, 2026, 4:30 PM EDT
Active, not recruiting Phase 3 Interventional

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCT05606614
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
6 Years to 21 Years · Female only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
8
States / cities
Duarte, California • La Jolla, California • Oakland, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863
View directory record Official record
Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Completed Phase 2 Interventional

A Study of RO4917523 in Patients With Fragile X Syndrome

NCT01517698
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Placebo, RO4917523 0.5 mg, RO4917523 1.5 mg
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
14 Years to 50 Years
Enrollment
185 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
20
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2016 · Synced Oct 9, 2026, 4:30 PM EDT
Terminated Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228
View directory record Official record
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 12, 2023 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting No phase listed Observational

The Rett Syndrome Global Registry

NCT04900493
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
1
States / cities
Trumbull, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Completed Phase 1 Interventional

An Initial Study of AZD7325 in Adults With Fragile X Syndrome

NCT03140813
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AZD7325 (High-Dose), AZD7325 (Low-Dose), Placebo oral capsule
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 50 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 8, 2021 · Synced Oct 9, 2026, 4:30 PM EDT
Completed Phase 3 Interventional

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome

NCT04977986
View directory record Official record
Conditions
Fragile X Syndrome, Fragile X Syndrome (FXS)
Interventions
ZYN002, Placebo
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
3 Years to 29 Years
Enrollment
257 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
18
States / cities
Phoenix, Arizona • Culver City, California • Irvine, California + 15 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Enrolling by invitation Phase 1 Interventional

Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities

NCT05301361
View directory record Official record
Conditions
Intellectual Disability, Fragile X Syndrome, Down Syndrome, Attention Deficit Hyperactivity Disorder
Interventions
Methylphenidate Oral Solution
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
6 Years to 24 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 9, 2026, 4:30 PM EDT
Active, not recruiting No phase listed Observational

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting No phase listed Observational

Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders

NCT07493096
View directory record Official record
Conditions
Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD), Neurodevelopmental Disorders and Developmental Abnormalities, Developmental Delay (Disorder), Cerebral Palsy (CP), Cerebral Palsy Hemiparetic Cerebral Palsy Spasticity Gait Disorders, Neurologic Postural Balance Impairment, Cerebral Palsy Infantile, Cerebral Palsy Spastic Hemiplegic, Cerebral Palsy, Dyskinetic, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD, Hypoxic Ischemic Encephalopathy, Hypoxic Ischemic Encephalopathy (HIE), Traumatic Brain Injury (TBI), Sensory Processing Disorder, Chromosomal Abnormalities, Genetic Disorders, Down Syndrome (Trisomy 21), Fragile X Syndrome (FXS), RETT Syndrome With Proven MECP2 Mutation, Williams Syndrome, 22q11.2 Deletion Syndrome, Sensorimotor Integration
Interventions
Not listed
Lead sponsor
Healing Hope International
Other
Eligibility
4 Years to 12 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
The Woodlands, Texas
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 9, 2026, 4:30 PM EDT
Completed Phase 2 Interventional Results available

Pharmacological Treatment of Rett Syndrome With Statins

NCT02563860
View directory record Official record
Conditions
Rett Syndrome
Interventions
Lovastatin
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
3 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Jul 12, 2019 · Synced Oct 9, 2026, 4:30 PM EDT
Recruiting No phase listed Observational

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

NCT06092346
View directory record Official record
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 9, 2026, 4:30 PM 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 9, 2026, 4:30 PM EDT
Terminated Phase 1 Interventional

An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome

NCT02562820
View directory record Official record
Conditions
Rett Syndrome
Interventions
Intravenous infusion
Procedure
Lead sponsor
The Cleveland Clinic
Other
Eligibility
5 Years to 25 Years · Female only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 30, 2017 · Synced Oct 9, 2026, 4:30 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

NCT00069641
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
iduronate-2-sulfatase enzyme replacement therapy, Placebo
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 9, 2026, 4:30 PM EDT
Terminated Phase 3 Interventional Results available

Open-Label Extension Study of Trofinetide for Rett Syndrome

NCT04776746
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
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2024 · Synced Oct 9, 2026, 4:30 PM EDT