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ClinicalTrials.gov public records Last synced Oct 21, 2026, 2:09 PM EDT

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Showing 121–144 of 10,540 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disorders Clear all
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 21, 2026, 2:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Folate Metabolism in Normal Weight and Obese Women of Child-bearing Age

NCT01743196
View directory record Official record
Conditions
Obesity, Neural Tube Defects
Interventions
Not listed
Lead sponsor
University of Georgia
Other
Eligibility
18 Years to 35 Years · Female only
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Athens, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 21, 2015 · Synced Oct 21, 2026, 2:09 PM EDT
Recruiting No phase listed Observational

Natural History Study of Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL)

NCT03624374
View directory record Official record
Conditions
Leukoencephalopathies, LBSL, Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, White Matter Disease, Ataxia, Cerebellar, Genetic Disease
Interventions
Not listed
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Phase 2 Interventional Results available

A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases

NCT02633397
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Riociguat, Placebo
Drug
Lead sponsor
Mark Gladwin
Other
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
20
States / cities
Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2023 · Synced Oct 21, 2026, 2:09 PM EDT
Terminated No phase listed Observational

Descemet Membrane Endothelial Keratoplasty vs. Descemet's Stripping With Endothelial Keratoplasty vs. Descemet Stripping Only

NCT04527523
View directory record Official record
Conditions
Cystoid Macular Edema, Fuchs Dystrophy
Interventions
Optical Coherence Tomography
Diagnostic Test
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 13, 2023 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Phase 1 Interventional

Nitric Oxide and Transfusion Therapy for Sickle Cell Patients With Pulmonary Hypertension

NCT00023296
View directory record Official record
Conditions
Sickle Cell Anemia, Pulmonary Hypertension
Interventions
Nitric Oxide, INO Pulse - NO Delivery System
Drug · Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2015
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 21, 2026, 2:09 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 21, 2026, 2:09 PM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 53 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2038
U.S. locations
15
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Oct 21, 2026, 2:09 PM EDT
No Longer Available No phase listed Expanded access

Treatment Use of 3,4-Diaminopyridine

NCT01765140
View directory record Official record
Conditions
Myasthenic Syndromes, Congenital
Interventions
3,4-diaminopyridine
Drug
Lead sponsor
Vern C. Juel, M.D.
Other
Eligibility
Not listed
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 20, 2025 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Not applicable Interventional Results available

Assessing and Promoting Resilience in Patients With Adult Congenital Heart Disease

NCT04738474
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Promoting Resilience in Stress Management (PRISM), Usual Care
Behavioral · Other
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Feasibility Study: Lifting and Tightening of the Elbows

NCT01708382
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera System Treatment
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
21 Years to 65 Years
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 24, 2017 · Synced Oct 21, 2026, 2:09 PM EDT
Suspended No phase listed Observational

Intraoperative Laser Speckle Contrast Imaging of Cerebral Blood Flow

NCT04865718
View directory record Official record
Conditions
Aneurysm, Brain, Arteriovenous Malformation of Brain, Neurovascular Disorder
Interventions
ICGA imaging
Diagnostic Test
Lead sponsor
Andrew Dunn
Other
Eligibility
18 Years and older
Enrollment
20 participants
Timeline
2019 – 2028
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 21, 2026, 2:09 PM EDT
Recruiting Phase 3 Interventional

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

NCT06840496
View directory record Official record
Conditions
Rett Syndrome
Interventions
Bionetide, Placebo
Drug
Lead sponsor
Biomed Industries, Inc.
Industry
Eligibility
5 Years to 20 Years · Female only
Enrollment
210 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
14
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 21, 2026, 2:09 PM EDT
Completed No phase listed Observational

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609827
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 21, 2026, 2:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Bone Regeneration Using Bone Marrow Stromal Cells

NCT00001391
View directory record Official record
Conditions
Bone Disease, Craniofacial Abnormality, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Dental and Craniofacial Research (NIDCR)
NIH
Eligibility
18 Years to 70 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 2:09 PM EDT
Completed No phase listed Observational

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

NCT04201418
View directory record Official record
Conditions
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
27
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Jun 6, 2022 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Not applicable Interventional

Cognitive Remediation in 22q11DS

NCT01781923
View directory record Official record
Conditions
22q11.2 Deletion Syndrome, Velo-Cardio-Facial Syndrome
Interventions
Cognitive remediation program, Small group social skills training
Behavioral
Lead sponsor
Duke University
Other
Eligibility
11 Years to 17 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 10, 2015 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Not applicable Interventional

Impact of Antioxidant Juice Intake on Brain Injury and Placental Pathology in Infants With Intrauterine Growth Restriction (IUGR)

NCT04394910
View directory record Official record
Conditions
Intrauterine Growth Restriction
Interventions
Pomegranate Juice, Placebo Juice
Dietary Supplement
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 6, 2023 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study to Access the Relative Bioavailability of Subcutaneous Dose of Nemolizumab When Administered Via Auto-Injector Versus Dual-Chamber Syringe

NCT05405985
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Nemolizumab
Drug
Lead sponsor
Galderma R&D
Industry
Eligibility
18 Years to 65 Years
Enrollment
192 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
2
States / cities
Tempe, Arizona • Lincoln, Nebraska
Source: ClinicalTrials.gov public record
Updated Jan 3, 2025 · Synced Oct 21, 2026, 2:09 PM EDT
Recruiting Phase 3 Interventional

A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

NCT07656415
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Mitapivat Matched Placebo, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Indianapolis, Indiana • Grand Rapids, Michigan • Flowood, Mississippi
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Phase 3 Interventional Results available

A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT03777176
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
Dasiglucagon, Standard of Care
Drug · Other
Lead sponsor
Zealand Pharma
Industry
Eligibility
3 Months to 12 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
3
States / cities
Aurora, Colorado • Philadelphia, Pennsylvania • Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 21, 2026, 2:09 PM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

NCT06363760
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2040
U.S. locations
16
States / cities
Oakland, California • Aurora, Colorado • New Haven, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2025 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Not applicable Interventional

Up, Down, and All Around: Evaluating Mobility Devices for Young Children With Developmental Disabilities and Delays

NCT06591559
View directory record Official record
Conditions
Down Syndrome, Gross Motor Delay, Cerebral Palsy
Interventions
Permobil Explorer Mini, Portable Mobility Aid for Children (PUMA)
Device
Lead sponsor
University of Washington
Other
Eligibility
12 Months to 60 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Oct 21, 2026, 2:09 PM EDT
Completed Phase 4 Interventional Results available

The Impact of Treating Staphylococcus Aureus Infection and Colonization on the Clinical Severity of Atopic Dermatitis

NCT00179959
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Sodium hypochlorite (bleach) baths, Mupirocin ointment, Water, Petrolatum Ointment
Drug
Lead sponsor
Northwestern University
Other
Eligibility
6 Months to 17 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 8, 2014 · Synced Oct 21, 2026, 2:09 PM EDT