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ClinicalTrials.gov public records Last synced Sep 9, 2026, 8:30 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed No phase listed Observational

Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease

NCT01959958
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Questionnaire/Interview
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
6 Years to 17 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 10, 2016 · Synced Sep 9, 2026, 8:30 AM EDT
Active, not recruiting No phase listed Observational

Genetic and Physical Study of Childhood Nerve and Muscle Disorders

NCT01568658
View directory record Official record
Conditions
Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Day to 100 Years
Enrollment
2,586 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 9, 2026, 8:30 AM EDT
Not listed Phase 1Phase 2 Interventional

Metformin in Patients With Fragile X

NCT04141163
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Metformin, Placebo oral tablet
Drug
Lead sponsor
Rowan University
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Stratford, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 30, 2019 · Synced Sep 9, 2026, 8:30 AM EDT
Not listed Phase 2 Interventional

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044
View directory record Official record
Conditions
Rett Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Center for Rare Neurological Diseases, Norcross, GA
Other
Eligibility
2 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Norcross, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 16, 2020 · Synced Sep 9, 2026, 8:30 AM EDT
Completed No phase listed Observational

Memory Disorders Registry

NCT00654563
View directory record Official record
Conditions
Dementia, Alzheimer's Disease, Parkinson Disease, Huntington Disease
Interventions
Not listed
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 26, 2012 · Synced Sep 9, 2026, 8:30 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

NCT04988867
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
2 Years to 5 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced Sep 9, 2026, 8:30 AM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

NCT07439887
View directory record Official record
Conditions
Stargardt Disease
Interventions
RTx-021
Genetic
Lead sponsor
Ray Therapeutics, Inc.
Industry
Eligibility
16 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Bakersfield, California • Pittsburgh, Pennsylvania • Bellaire, Texas
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Terminated No phase listed Observational

A Clinical Test for the Treatment of Multifocal Lung Cancers Using Genome Sequencing

NCT02705404
View directory record Official record
Conditions
Lung Neoplasms, Carcinoma Non-small-cell Lung
Interventions
Multifocal Tissue Banking
Genetic
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 13, 2022 · Synced Sep 9, 2026, 8:30 AM EDT
Completed Phase 4 Interventional Results available

Dexmedetomidine and Propofol for Pediatric MRI Sedation

NCT03513757
View directory record Official record
Conditions
Headache, Tumor, Seizure Disorder, Neurofibromatoses, Hydrocephalus, Abdominal Neoplasm, Spine Deformity
Interventions
propofol, Dexmedetomidine, Glycopyrrolate, Lidocaine 1% Injectable Solution, Nitrous Oxide, Sevoflurane
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
12 Months to 60 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 20, 2020 · Synced Sep 9, 2026, 8:30 AM EDT
Completed No phase listed Observational

Recovery and Outcomes From Stroke

NCT04007757
View directory record Official record
Conditions
Intracerebral Hemorrhage
Interventions
Not listed
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
8
States / cities
Chicago, Illinois • Louisville, Kentucky • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Sep 9, 2026, 8:30 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Testing AZD9291 as Potentially Targeted Treatment in Cancers With EGFR Genetic Changes (MATCH-Subprotocol E)

NCT06303167
View directory record Official record
Conditions
Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Multiple Myeloma
Interventions
Biopsy Procedure, Biospecimen Collection, Echocardiography Test, Multigated Acquisition Scan, Osimertinib, Radiologic Examination
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Recruiting No phase listed Observational

National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases

NCT06491615
View directory record Official record
Conditions
Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 120 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2054
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia

NCT01108770
View directory record Official record
Conditions
Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
Edimer Pharmaceuticals
Industry
Eligibility
1 Year and older · Male only
Enrollment
62 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010
U.S. locations
1
States / cities
Colorado Springs, Colorado
Source: ClinicalTrials.gov public record
Updated Aug 20, 2012 · Synced Sep 9, 2026, 8:30 AM EDT
Completed No phase listed Observational

Inflammation, Cardiac Sympathetic Innervation, and Arrhythmic Sudden Death

NCT01919983
View directory record Official record
Conditions
Ischemic Cardiomyopathy, Dilated Cardiomyopathy, Inflammation, Sudden Cardiac Death
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Sep 9, 2026, 8:30 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Studies of Disorders With Increased Susceptibility to Fungal Infections

NCT01222741
View directory record Official record
Conditions
Fungal Infections, Primary Immune Deficiencies
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 85 Years
Enrollment
850 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 23, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Terminated Phase 1 Interventional Results available

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

NCT03362502
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
22
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 14, 2025 · Synced Sep 9, 2026, 8:30 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Investigating Phenotypic, Epigenetic, and NeuroGenetic Traits in Rare and Ultra-rare Neurodevelopmental Disorders (Project PENGUIN)

NCT07329257
View directory record Official record
Conditions
Baker Gordon Syndrome, Rare Neurodevelopmental Conditions, Rare Neurogenetic Conditions, Syt-1 Disorder, Epilepsy, Seizure, Genetic Mutations, Autism in Children, Developmental Delay (Disorder)
Interventions
No Intervention: Observational Cohort
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
Up to 99 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Completed Not applicable Interventional

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

NCT00002273
View directory record Official record
Conditions
Colitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
21 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 9, 2026, 8:30 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of Brain Activity Changes After a Behavior Change Weight Loss Intervention

NCT04522245
View directory record Official record
Conditions
Obesity, Behavior, Health, Weight Loss
Interventions
Noom Healthy Weight; mobile behavior change program for weight loss., Matched Control
Behavioral · Other
Lead sponsor
Noom Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Commack, New York
Source: ClinicalTrials.gov public record
Updated Jul 19, 2021 · Synced Sep 9, 2026, 8:30 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Research for Individualized Therapeutics in Rare Genetic Disease

NCT05236595
View directory record Official record
Conditions
Rare Genetic Disease, Undiagnosed Diseases
Interventions
Individualized drug matching per genetic disease
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
3
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Minneota, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Recruiting Phase 3 Interventional

A Long-term Study of the Medicine Called Abrocitinib in Children Aged 2 Years and Older With Moderate to Severe Eczema

NCT06807281
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Abrocitinib
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 11 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
11
States / cities
Birmingham, Alabama • North Little Rock, Arkansas • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 9, 2026, 8:30 AM EDT
Completed No phase listed Observational

The Second Multicenter Hemophilia Cohort Study

NCT00341705
View directory record Official record
Conditions
Liver Decompensation, Hepatocellular Carcinoma, Non-Hodgkin Lymphoma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
13 Years and older
Enrollment
2,565 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
39
States / cities
Tucson, Arizona • Davis, California • Orange, California + 31 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Sep 9, 2026, 8:30 AM EDT
Completed Phase 3 Interventional Results available

Study of Ruxolitinib Cream in Adolescents With Atopic Dermatitis

NCT05456529
View directory record Official record
Conditions
Atopic Dermatitis (AD)
Interventions
Ruxolitinib Cream
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
12 Years to 17 Years
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
North Little Rock, Arkansas • Fountain Valley, California • Los Angeles, California + 21 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2025 · Synced Sep 9, 2026, 8:30 AM EDT