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ClinicalTrials.gov public records Last synced Oct 31, 2026, 8:57 PM EDT

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Showing 2329–2352 of 10,816 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Recruiting Not applicable Interventional

MULTIsite Feasibility of MUSIc Therapy to Address Quality Of Life in Sickle Cell Disease

NCT06853158
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Music therapy, Health education
Behavioral · Other
Lead sponsor
University of California, Irvine
Other
Eligibility
14 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
2
States / cities
Cleveland, Ohio • Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Oct 31, 2026, 8:57 PM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

NCT06597006
View directory record Official record
Conditions
Familial Hypercholesterolemia - Homozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
4
States / cities
San Francisco, California • Washington D.C., District of Columbia • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs

NCT01033552
View directory record Official record
Conditions
Epidermolysis Bullosa
Interventions
Cyclophosphamide, Fludarabine, Anti-thymocyte globulin, Myeloablative Busulfan, Mesenchymal stem cell transplantation, Total body irradiation, Bone marrow or umbilical cord blood (UCG) stem cell transplantation
Drug · Procedure · Radiation
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 25 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2021
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 31, 2026, 8:57 PM EDT
Completed No phase listed Observational

The Microvascular Complications Study

NCT00222508
View directory record Official record
Conditions
Cystic Fibrosis Related Diabetes
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 2, 2012 · Synced Oct 31, 2026, 8:57 PM EDT
Completed No phase listed Observational

Telemedicine Approaches to Evaluating Acute-phase ROP

NCT01264276
View directory record Official record
Conditions
Retinopathy of Prematurity
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
32 Weeks to 40 Weeks
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
11
States / cities
Louisville, Kentucky • Baltimore, Maryland • Boston, Massachusetts + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2015 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Phase 2 Interventional Results available

CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

NCT01095510
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
CINRYZE
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
8
States / cities
Colorado Springs, Colorado • Tampa, Florida • Chevy Chase, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2021 · Synced Oct 31, 2026, 8:57 PM EDT
Enrolling by invitation Not applicable Interventional

Applying Population Management Best Practices to Preventative Genomic Medicine Trial

NCT07053813
View directory record Official record
Conditions
Neoplastic Syndromes, Hereditary
Interventions
Web resources, Personalized outreach
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 75 Years
Enrollment
900 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
2
States / cities
Denver, Colorado • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 31, 2026, 8:57 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 31, 2026, 8:57 PM EDT
Completed Not applicable Interventional

Evaluation and Treatment of Heart Disease in Patients Not Participating in Research

NCT00001460
View directory record Official record
Conditions
Arrhythmia, Congenital Heart Defect, Heart Disease
Interventions
inpatient evaluation and management
Behavioral
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 5, 2008 · Synced Oct 31, 2026, 8:57 PM EDT
Not listed No phase listed Observational

Impact of Thrombocytopenia and Platelet Transfusions on Neonatal Bleeding and Inflammation

NCT03848923
View directory record Official record
Conditions
Neonatal Thrombocytopenia
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
0 Days to 6 Months
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 31, 2026, 8:57 PM EDT
Recruiting Not applicable Interventional

Computerized Decision Support for Identification and Management of Familial Hypercholesterolemia

NCT06743659
View directory record Official record
Conditions
Familial Hypercholesterolaemia
Interventions
Alert-based computerized decision support
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Phase 4 Interventional Results available

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

NCT03335488
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 99 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Gainesville, Florida • New York, New York • Cleveland, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 31, 2026, 8:57 PM EDT
Recruiting Phase 2 Interventional

Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL)

NCT06919003
View directory record Official record
Conditions
Kidney Transplant
Interventions
Berinert, Placebo for Berinert
Biological · Other
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
4
States / cities
Los Angeles, California • Chicago, Illinois • Kansas City, Kansas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Phase 4 Interventional Results available

Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

NCT00243386
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
7 Years to 65 Years
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
9
States / cities
Los Angeles, California • Chicago, Illinois • Indianapolis, Indiana + 6 more
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Oct 31, 2026, 8:57 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569
View directory record Official record
Conditions
GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
AXO-AAV-GM2
Drug
Lead sponsor
Terence Flotte
Other
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 31, 2026, 8:57 PM EDT
Not listed No phase listed Observational

A Retrospective Analysis of Cerefolin NAC® in Cognitively Impaired Patients

NCT01594541
View directory record Official record
Conditions
Mild Cognitive Impairment
Interventions
Not listed
Lead sponsor
Pamlab, Inc.
Industry
Eligibility
55 Years to 89 Years
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Springfield, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 24, 2014 · Synced Oct 31, 2026, 8:57 PM EDT
Recruiting No phase listed Observational

Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform

NCT06999954
View directory record Official record
Conditions
Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia
Interventions
Not listed
Lead sponsor
Shwachman-Diamond Syndrome Alliance Inc
Other
Eligibility
Not listed
Enrollment
8,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2088
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 31, 2025 · Synced Oct 31, 2026, 8:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genes Causing Ebstein's Anomaly

NCT00497705
View directory record Official record
Conditions
Heart Septal Defects, Ventricle, Heart Defects, Congenital, Double Outlet Right Ventricle, Truncus Arteriosus, Persistent
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 31, 2026, 8:57 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Evaluation of ETHY-001 for Safety, Tolerability, and Clinical Activity in Thyroid Eye Disease

NCT07885605
View directory record Official record
Conditions
Thyroid Eye Disease (TED), Graves' Disease, Eye Diseases, Hereditary, Eye Diseases, Exophthalmos, Orbital Diseases, Goiter, Thyroid Diseases, Endocrine System Diseases, Hyperthyroidism, Autoimmune Diseases, Immune System Diseases, Graves Ophthalmopathy, Proptosis
Interventions
ETHY-001, Placebo
Drug · Other
Lead sponsor
Ethyreal Bio, Inc.
Industry
Eligibility
18 Years and older
Enrollment
72 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
3
States / cities
Orland Park, Illinois • Las Vegas, Nevada • Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 26, 2026 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Telemedicine vs. Face-to-Face Cancer Genetic Counseling

NCT00609505
View directory record Official record
Conditions
Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome
Interventions
Telemedicine, Face-to-Face
Other
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2011
U.S. locations
4
States / cities
Henderson, North Carolina • Laurinburg, North Carolina • Lumberton, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2012 · Synced Oct 31, 2026, 8:57 PM EDT
Not listed Phase 2 Interventional

Rifaximin to Modify the Disease Course in Sickle Cell Disease

NCT03719729
View directory record Official record
Conditions
Sickle Cell Disease, Antibiotics
Interventions
Rifaximin
Drug
Lead sponsor
New York Medical College
Other
Eligibility
18 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Mar 14, 2019 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Not applicable Interventional Results available

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

NCT00244010
View directory record Official record
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 31, 2026, 8:57 PM EDT
Terminated Phase 2 Interventional Results available

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

NCT00837824
View directory record Official record
Conditions
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
9
States / cities
Farmington, Connecticut • Coral Springs, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 31, 2026, 8:57 PM EDT
Completed Phase 2 Interventional Results available

Study Evaluating the Mechanism of Action of PF-04965842 Monotherapy for Moderate-to-severe Atopic Dermatitis

NCT03915496
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
PF-04965842 200 mg, PF-04965842 100 mg, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
9
States / cities
Encinitas, California • Los Angeles, California • Largo, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2023 · Synced Oct 31, 2026, 8:57 PM EDT