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ClinicalTrials.gov public records Last synced Oct 22, 2026, 4:18 PM EDT

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Showing 457–480 of 559 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 31, 2013 · Synced Oct 22, 2026, 4:18 PM EDT
Recruiting Phase 2 Interventional

A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)

NCT04546399
View directory record Official record
Conditions
Down Syndrome, Recurrent B Acute Lymphoblastic Leukemia
Interventions
3-Dimensional Conformal Radiation Therapy, Asparaginase Erwinia chrysanthemi, Biospecimen Collection, Blinatumomab, Bone Marrow Aspiration, Bone Marrow Biopsy, Calaspargase Pegol, Cytarabine, Dexamethasone, Hydrocortisone Sodium Succinate, Lumbar Puncture, Mercaptopurine, Methotrexate, Nivolumab, Pegaspargase, Vincristine Sulfate
Radiation · Drug · Procedure + 1 more
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 30 Years
Enrollment
461 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
211
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 151 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 22, 2026, 4:18 PM EDT
Completed No phase listed Observational

Natural History Study of Serious Medical Events in PWS

NCT03718416
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Not listed
Lead sponsor
Foundation for Prader-Willi Research
Other
Eligibility
5 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Walnut, California
Source: ClinicalTrials.gov public record
Updated Feb 29, 2024 · Synced Oct 22, 2026, 4:18 PM EDT
Completed No phase listed Observational

Surgical Treatment of Children With OSA and Small Tonsils or Down Syndrome

NCT05154214
View directory record Official record
Conditions
Obstructive Sleep Apnea of Child
Interventions
Adenotonsillectomy, Drug-induced sleep endoscopy directed surgery
Procedure
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years to 18 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 22, 2026, 4:18 PM EDT
Enrolling by invitation No phase listed Observational

Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome

NCT07450664
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
VYKAT XR
Drug
Lead sponsor
Soleno Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 22 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 2 Interventional

The Effects of Hormones in Growth Hormone-Treated Girls With Turner Syndrome

NCT00001343
View directory record Official record
Conditions
Dwarfism, Turner's Syndrome
Interventions
Humatrope, Oxandrolone
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
10 Years to 14 Years · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 22, 2026, 4:18 PM EDT
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 22, 2026, 4:18 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism

NCT00837616
View directory record Official record
Conditions
Turner Syndrome, Hypogonadism, Premature Ovarian Failure
Interventions
17 B estradiol orally, 17 B estradiol
Drug
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
13 Years to 20 Years · Female only
Enrollment
41 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2012
U.S. locations
2
States / cities
Jacksonville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 16, 2023 · Synced Oct 22, 2026, 4:18 PM EDT
Completed No phase listed Observational

Development of Non-invasive Prenatal Test for Microdeletion and Other Genetic Syndromes Based on Cell Free DNA

NCT02109770
View directory record Official record
Conditions
Microdeletion Syndromes, Trisomy 21, Trisomy 18, Trisomy 13, Sex Chromosome Abnormalities
Interventions
Not listed
Lead sponsor
Natera, Inc.
Industry
Eligibility
Not listed
Enrollment
216 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
2
States / cities
San Carlos, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 26, 2019 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 2 Interventional Results available

Revlimid in Patients With Acute Myelogenous Leukemia and Myelodysplastic Syndrome

NCT00360672
View directory record Official record
Conditions
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Interventions
Revlimid
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 14, 2013 · Synced Oct 22, 2026, 4:18 PM EDT
Completed No phase listed Observational

Evaluation of Patients With Unresolved Chromosome Abnormalities

NCT00001639
View directory record Official record
Conditions
Abnormalities, Failure to Thrive, Mental Retardation, Microcephaly
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
263 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 4:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

NCT05462106
View directory record Official record
Conditions
Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
Interventions
Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
35 Years to 85 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
13
States / cities
Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 4 Interventional Results available

Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study

NCT01735955
View directory record Official record
Conditions
Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST), Acute Lymphoblastic Leukemia (ALL), Receptor Tyrosine Kinase (KIT) Mutated Melanoma
Interventions
Nilotinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Albany, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 8, 2024 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

Building Sentences With Preschoolers Who Use AAC

NCT03538925
View directory record Official record
Conditions
Down Syndrome, Speech and Language Disorder, Speech Disorders in Children, Speech Intelligibility, Speech Sound Disorder
Interventions
AAC Generative Language Intervention, Standard of Care / Business as Usual
Behavioral
Lead sponsor
University of Central Florida
Other
Eligibility
3 Years to 4 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
2
States / cities
Orlando, Florida • Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Jan 8, 2024 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 2Phase 3 Interventional

A Trial of Metformin in Individuals With Fragile X Syndrome

NCT03479476
View directory record Official record
Conditions
Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked, Genetic Diseases, X-Linked, Trinucleotide Repeat Expansion, Fra(X) Syndrome, Intellectual Disability, FXS, Neurobehavioral Manifestations, Sex Chromosome Disorders
Interventions
Placebo Medication, Metformin
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
6 Years to 25 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2025 · Synced Oct 22, 2026, 4:18 PM 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 22, 2026, 4:18 PM EDT
Recruiting Phase 2 Interventional

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

NCT07284641
View directory record Official record
Conditions
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions
Hematopoietic stem cell transplant (HSCT)
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 22, 2026, 4:18 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Angelman Syndrome Video Assessment (ASVA) Source Material Study

NCT05637697
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
1 Year and older
Enrollment
73 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 22, 2026, 4:18 PM EDT
Terminated No phase listed Observational

Infection in DiGeorge Following CHD Surgery

NCT00278005
View directory record Official record
Conditions
DiGeorge Syndrome, Congenital Heart Defects
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 15 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2008
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Mar 16, 2012 · Synced Oct 22, 2026, 4:18 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 22, 2026, 4:18 PM EDT
Terminated Phase 2 Interventional Results available

Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial

NCT00675025
View directory record Official record
Conditions
Down Syndrome
Interventions
Donepezil hydrochloride (Aricept)
Drug
Lead sponsor
Eisai Inc.
Industry
Eligibility
10 Years to 17 Years
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
31
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 24 more
Source: ClinicalTrials.gov public record
Updated Mar 29, 2021 · Synced Oct 22, 2026, 4:18 PM EDT
Active, not recruiting Phase 2 Interventional

Medications for Obstructive Sleep Apnea to Improve Cognition in Children With Down Syndrome

NCT05933603
View directory record Official record
Conditions
Obstructive Sleep Apnea, Down Syndrome
Interventions
Atomoxetine and Oxybutynin (ato-oxy)
Drug
Lead sponsor
University of Arizona
Other
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 2 Interventional Results available

Thymus Transplantation Dose in DiGeorge #932

NCT00576836
View directory record Official record
Conditions
DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
Interventions
Cultured Thymus Tissue Implantation (CTTI), Cultured Thymus Tissue Implantation with Parathyroid Transplantation
Biological · Other
Lead sponsor
Sumitomo Pharma Switzerland GmbH
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 25, 2022 · Synced Oct 22, 2026, 4:18 PM EDT
Completed Phase 1 Interventional

Oxytocin Trial in Prader-Willi Syndrome

NCT02013258
View directory record Official record
Conditions
Prader Willi Syndrome
Interventions
Intranasal oxytocin, Placebo
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 11 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
3
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2018 · Synced Oct 22, 2026, 4:18 PM EDT