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ClinicalTrials.gov public records Last synced Oct 24, 2026, 6:49 PM EDT

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Showing 601–624 of 10,333 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Terminated Phase 1Phase 2 Interventional Results available

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

NCT00467831
View directory record Official record
Conditions
Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Metabolic Disease
Interventions
Losartan, Zileuton, N-Acetylcysteine, Pravastatin, Erythromycin
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 2, 2013 · Synced Oct 24, 2026, 6:49 PM EDT
Completed No phase listed Observational

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Not applicable Interventional

Sargramostim in Reducing Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplantation for Hematologic Cancer or Aplastic Anemia

NCT00053157
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
sargramostim
Biological
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
5 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Oct 5, 2022 · Synced Oct 24, 2026, 6:49 PM EDT
Terminated Phase 1 Interventional

Effect of Potassium and Acetazolamide on People With Andersen-Tawil Syndrome

NCT00839501
View directory record Official record
Conditions
Andersen-Tawil Syndrome
Interventions
Potassium, Acetazolamide
Dietary Supplement · Drug
Lead sponsor
University of Rochester
Other
Eligibility
10 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 20, 2012 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Phase 2Phase 3 Interventional

Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901

NCT00157053
View directory record Official record
Conditions
Hemophilia A
Interventions
Antihemophilic factor, recombinant, manufactured protein-free
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
10 Years and older · Male only
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
11
States / cities
Aurora, Colorado • Atlanta, Georgia • Peoria, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2021 · Synced Oct 24, 2026, 6:49 PM EDT
Terminated Phase 3 Interventional Results available

Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI

NCT02352753
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Denosumab
Drug
Lead sponsor
Amgen
Industry
Eligibility
2 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
13
States / cities
Los Angeles, California • Torrance, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2022 · Synced Oct 24, 2026, 6:49 PM EDT
Active, not recruiting Phase 3 Interventional

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
0 Years to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 24, 2026, 6:49 PM EDT
Recruiting No phase listed Observational

The Duchenne Registry

NCT02069756
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier, Dystrophinopathy Female Carrier
Interventions
Not listed
Lead sponsor
The Duchenne Registry
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2047
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Oct 24, 2026, 6:49 PM EDT
Recruiting No phase listed Observational

Forced Oscillometry in Infants With Bronchopulmonary Dysplasia

NCT04270045
View directory record Official record
Conditions
Infant, Premature, Diseases, Bronchopulmonary Dysplasia, Respiratory Distress Syndrome
Interventions
Non-invasive forced airway oscillometry
Device
Lead sponsor
Winston Manimtim
Other
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Phase 2 Interventional Results available

MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

NCT02582775
View directory record Official record
Conditions
Epidermolysis Bullosa
Interventions
Thymoglobulin, Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone marrow infusion, Tacrolimus, Mycophenolate Mofetil, Donor mesenchymal stem cell infusions, Busulfan
Drug · Radiation · Procedure + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 25 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 24, 2026, 6:49 PM EDT
Terminated Phase 2 Interventional Results available

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

NCT04638153
View directory record Official record
Conditions
Achondroplasia
Interventions
Recifercept
Biological
Lead sponsor
Pfizer
Industry
Eligibility
3 Months to 10 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
6
States / cities
Aliso Viejo, California • Irvine, California • Long Beach, California + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2024 · Synced Oct 24, 2026, 6:49 PM EDT
Not listed No phase listed Observational

Electrophysiologic and Morphologic Assessment of the Substrate to Guide Implantation of Defibrillators in Dilated Cardiomyopathy

NCT01378572
View directory record Official record
Conditions
Dilated Cardiomyopathy, Primary Prevention of Sudden Cardiac Death, Implantable Cardioverter Defibrillator
Interventions
Not listed
Lead sponsor
Catholic University of the Sacred Heart
Other
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Jun 22, 2011 · Synced Oct 24, 2026, 6:49 PM EDT
Recruiting Phase 1 Interventional

Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load

NCT06974877
View directory record Official record
Conditions
Cryopyrin-Associated Periodic Syndromes, Amyloidosis
Interventions
124I AT-01
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 120 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Phase 2 Interventional Results available

Tolvaptan Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT00413777
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Dominant
Interventions
Tolvaptan
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
11
States / cities
Denver, Colorado • Jacksonville, Florida • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Not applicable Interventional

Intestinal Function in Neonates With Complex Congenital Heart Disease

NCT01475357
View directory record Official record
Conditions
Congenital Heart Defects, Growth Failure
Interventions
Arm 1: NPO by mouth pre-operative, Arm 2: Fresh Breast Milk pre-operative
Other
Lead sponsor
Medical University of South Carolina
Other
Eligibility
37 Weeks to 40 Weeks
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 15, 2014 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Phase 3 Interventional Results available

Growth Hormone Therapy in Osteogenesis Imperfecta

NCT00001305
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Humatrope
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 16 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 29, 2019 · Synced Oct 24, 2026, 6:49 PM EDT
Completed No phase listed Observational

4-D Visualization Using Magnetic Resonance Imaging (MRI) in the Congenital Heart Disease Population

NCT00165945
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 2, 2012 · Synced Oct 24, 2026, 6:49 PM EDT
Terminated Phase 4 Interventional Accepts healthy volunteers Results available

Efficient Study of ACULAR in Inhibiting Proliferative Retinopathy in Prematurity

NCT00634972
View directory record Official record
Conditions
Retinopathy of Prematurity, Retinal Detachment, Blindness
Interventions
ACULAR, REFRESH TEARS, placebo
Drug
Lead sponsor
University of South Alabama
Other
Eligibility
10 Days to 15 Days
Enrollment
83 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2008
U.S. locations
1
States / cities
Mobile, Alabama
Source: ClinicalTrials.gov public record
Updated Dec 8, 2020 · Synced Oct 24, 2026, 6:49 PM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

NCT06079372
View directory record Official record
Conditions
Hypophosphatasia
Interventions
ALXN1850, asfotase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 11 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Hartford, Connecticut • Baltimore, Maryland • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 24, 2026, 6:49 PM EDT
Completed No phase listed Observational

Familial Prostate Cancer

NCT00001377
View directory record Official record
Conditions
Neoplastic Syndromes, Hereditary, Prostatic Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety Study of AeroVanc for the Treatment of Persistent MRSA Lung Infection in Cystic Fibrosis Patients

NCT01746095
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Vancomycin hydrochloride inhalation powder, Placebo inhalation powder
Drug
Lead sponsor
Savara Inc.
Industry
Eligibility
12 Years and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
38
States / cities
Mobile, Alabama • Little Rock, Arkansas • Los Angeles, California + 32 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Oct 24, 2026, 6:49 PM EDT
Completed No phase listed Observational

Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)

NCT03478839
View directory record Official record
Conditions
Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets Type2
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Year to 110 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 24, 2026, 6:49 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Impact of Extended CPAP on Bronchopulmonary Dysplasia

NCT05547139
View directory record Official record
Conditions
Bronchopulmonary Dysplasia, Prematurity
Interventions
Protocolized weaning of respiratory support
Other
Lead sponsor
Christiana Care Health Services
Other
Eligibility
22 Weeks to 30 Weeks
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Newark, Delaware
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 24, 2026, 6:49 PM EDT
Recruiting Phase 1 Interventional

Testing for Safety and Colorectal Cancer Preventive Effects of ONC201

NCT05630794
View directory record Official record
Conditions
Colorectal Adenomatous Polyp, Colorectal Carcinoma, Familial Adenomatous Polyposis, Multiple Adenomatous Polyps
Interventions
Biopsy Procedure, Biospecimen Collection, Colonoscopy, Dordaviprone Hydrochloride, Endoscopic Procedure, Questionnaire Administration, Sigmoidoscopy
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Ann Arbor, Michigan • St Louis, Missouri • Cleveland, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 24, 2026, 6:49 PM EDT