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ClinicalTrials.gov public records Last synced Oct 27, 2026, 1:31 PM EDT

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Showing 745–768 of 10,339 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Completed Phase 1 Interventional Results available

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

NCT00430768
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
rAAV1-CB-hAAT
Biological
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
2
States / cities
Gainesville, Florida • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 15, 2016 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Not applicable Interventional

Fetal Endotracheal Occlusion (FETO) in Severe and Extremely Severe Congenital Diaphragmatic Hernia

NCT00881660
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Goldballoon Detachable Balloon and delivery microcatheter
Device
Lead sponsor
Michael A Belfort
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional

A Clinical Trial in Subjects With Atopic Dermatitis (Part 1)

NCT05717920
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
ADX-629 (Open-label)
Drug
Lead sponsor
Aldeyra Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Bexley, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 4, 2024 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy

NCT02239224
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Placebo, ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Rochester, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 11, 2021 · Synced Oct 27, 2026, 1:31 PM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)

NCT03514420
View directory record Official record
Conditions
Familial Partial Lipodystrophy
Interventions
AKCEA-ANGPTL3-LRx
Drug
Lead sponsor
Akcea Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Oct 27, 2026, 1:31 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Rapid Whole Genome Sequencing Study

NCT03385876
View directory record Official record
Conditions
Genetic Diseases, Genetic Syndrome
Interventions
Genomic sequencing and molecular diagnostic results, if any
Genetic
Lead sponsor
Rady Pediatric Genomics & Systems Medicine Institute
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2050
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 23, 2021 · Synced Oct 27, 2026, 1:31 PM EDT
Suspended Phase 2 Interventional

A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome

NCT01325740
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
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 20, 2012 · Synced Oct 27, 2026, 1:31 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Energy Requirements in Critically Ill Newborns

NCT00006274
View directory record Official record
Conditions
Respiratory Distress Syndrome
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
24 Hours to 120 Hours
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1997
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

NCT04007991
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
53
States / cities
Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua (AMT-061)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
4
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2024 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 3 Interventional

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

NCT02932618
View directory record Official record
Conditions
Von Willebrand Disease
Interventions
Vonicog alfa, Antihemophilic Factor (Recombinant)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
Up to 17 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
16
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Jacksonville, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

NCT01384513
View directory record Official record
Conditions
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Aplastic Anemia, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Myelodysplastic Syndromes, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Essential Thrombocythemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Juvenile Myelomonocytic Leukemia, Mastocytosis, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Polycythemia Vera, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Anemia, Refractory Anemia With Ringed Sideroblasts, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Myelodysplastic Syndromes, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenström Macroglobulinemia
Interventions
Fludarabine, Busulfan, Total Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI), Cyclophosphamide (CY), Tacrolimus, Mycophenolate mofetil, Allogeneic hematopoietic stem cell transplantation, Peripheral blood stem cell transplantation (PBSCT)
Drug · Radiation · Biological + 2 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Terminated Phase 1 Interventional

Initiating Transdermal Estradiol Therapy in Turner's Syndrome

NCT00870220
View directory record Official record
Conditions
Turner's Syndrome
Interventions
Norditropin, Menostar 14mcg patch, Vivelle dot 25mcg patch
Drug
Lead sponsor
University of Chicago
Other
Eligibility
138 Months to 13 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
3
States / cities
Chicago, Illinois • Baltimore, Maryland • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 11, 2014 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 3 Interventional

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Terminated No phase listed Observational

Hemodynamic Assessment of Distal Revascularization Interval Ligation

NCT03054623
View directory record Official record
Conditions
Steal Syndrome, Chronic Kidney Diseases, Arterio-Venous Fistula
Interventions
DRIL procedure
Procedure
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
2
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jan 24, 2025 · Synced Oct 27, 2026, 1:31 PM EDT
Recruiting No phase listed Observational

Patterns of Neurodevelopmental Disorders

NCT06213090
View directory record Official record
Conditions
Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome, Down Syndrome, Epilepsy, Mitochondrial Encephalomyopathies, Cerebral Folate Deficiency
Interventions
Not listed
Lead sponsor
Richard Frye
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Recruiting Phase 3 Interventional

A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

NCT07285460
View directory record Official record
Conditions
Hemophilia
Interventions
Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
Drug · Biological
Lead sponsor
Sanofi
Industry
Eligibility
1 Year to 11 Years · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
8
States / cities
Los Angeles, California • Orange, California • Torrance, California + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional

Bisphosphonate Treatment of Osteogenesis Imperfecta

NCT00063479
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Zoledronic Acid
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
3 Months to 17 Years
Enrollment
158 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2007
U.S. locations
10
States / cities
Los Angeles, California • Wilmington, Delaware • Boise, Idaho + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2017 · Synced Oct 27, 2026, 1:31 PM EDT
Not listed No phase listed Observational

Pulmonary Health in Children With Down Syndrome

NCT04398862
View directory record Official record
Conditions
Down Syndrome, Dysphagia, Oropharyngeal
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
3 Years to 18 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 26, 2024 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Phase 2b Study to Evaluate the Efficacy and Safety of ISB 830 in Adults With Moderate to Severe Atopic Dermatitis

NCT03568162
View directory record Official record
Conditions
Moderate to Severe Atopic Dermatitis
Interventions
ISB 830 - Part 1 Group 1, ISB 830 - Part 1 Group 2, ISB 830 - Part 1 Group 3, Placebo - Part 1 Group 4, ISB 830 - Part 2 Group 5, Placebo - Part 2 Group 6
Drug
Lead sponsor
Ichnos Sciences SA
Industry
Eligibility
18 Years and older
Enrollment
462 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
34
States / cities
Birmingham, Alabama • Clovis, California • Rolling Hills Estates, California + 29 more
Source: ClinicalTrials.gov public record
Updated Aug 23, 2022 · Synced Oct 27, 2026, 1:31 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 29, 2020 · Synced Oct 27, 2026, 1:31 PM EDT
Completed Phase 4 Interventional Results available

Preterm Infant Inhaled Albuterol Dosing

NCT02447250
View directory record Official record
Conditions
Bronchopulmonary Dysplasia, Very Low Birth Weight
Interventions
Varied albuterol dose response
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
14 Days and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 23, 2019 · Synced Oct 27, 2026, 1:31 PM EDT
Recruiting Not applicable Interventional

Gastric Feeding Versus Transpyloric Feeding in Infants with Severe Bronchopulmonary Dysplasia, a Crossover Study

NCT06821776
View directory record Official record
Conditions
Bronchopulmonary Dysplasia, Feeding Intolerance
Interventions
Gastric feeding, Transpyloric feeding
Procedure
Lead sponsor
Le Bonheur Children's Hospital
Other
Eligibility
1 Month to 1 Year
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 12, 2025 · Synced Oct 27, 2026, 1:31 PM EDT