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ClinicalTrials.gov public records Last synced Oct 14, 2026, 8:19 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 241–264 of 10,529 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Withdrawn No phase listed Observational

The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)

NCT04214002
View directory record Official record
Conditions
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
Interventions
Not listed
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
6 Months and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Sep 17, 2021 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Changes in Adherence After Playing "My Life With CF" Game

NCT00688051
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
My Life with CF game
Other
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
12 Years to 21 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 5, 2014 · Synced Oct 14, 2026, 8:19 AM EDT
Completed No phase listed Observational

Immunoglobulin G Therapy Dose Optimization

NCT04818177
View directory record Official record
Conditions
Obesity, Immune Deficiency
Interventions
Institutional standard intravenous immune globulin treatment
Drug
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years to 75 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Somerville, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 14, 2026, 8:19 AM EDT
Withdrawn Not applicable Interventional

Open Label Safety Study of DCI-1020 in Pediatric Cystic Fibrosis (CF) Patients

NCT00663975
View directory record Official record
Conditions
Digestive System Diseases, Pancreatic Disease, Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions
DCI 1020
Drug
Lead sponsor
Digestive Care, Inc.
Industry
Eligibility
Up to 2 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated May 13, 2010 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 4 Interventional Results available

Testing the Effect of Adding Chronic Oral Azithromycin to Inhaled Tobramycin in People With Cystic Fibrosis (CF)

NCT02677701
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
azithromycin, placebo (for azithromycin), inhaled tobramycin
Drug
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
12 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
39
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 33 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of the Immediate Effect and Safety of DMA™ Novel Muscle Stimulation Technology

NCT04296201
View directory record Official record
Conditions
Skin Laxity, Muscle Contracture
Interventions
DMA treatment
Device
Lead sponsor
Lumenis Be Ltd.
Industry
Eligibility
25 Years to 45 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 2Phase 3 Interventional

Phase 2/3 Oxabact Study

NCT01037231
View directory record Official record
Conditions
Primary Hyperoxaluria
Interventions
Oxalobacter formigenes, Placebo
Biological · Drug
Lead sponsor
OxThera
Industry
Eligibility
2 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 15, 2013 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

NCT01362595
View directory record Official record
Conditions
Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
Interventions
leucine
Drug
Lead sponsor
Northwell Health
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2022 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Not applicable Interventional Results available

MN4000 for Treatment of CF and MND Patients in the Home Setting

NCT03797677
View directory record Official record
Conditions
Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
Interventions
MN 4000
Device
Lead sponsor
Hill-Rom
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 7, 2024 · Synced Oct 14, 2026, 8:19 AM EDT
Not listed Not applicable Interventional

Transcranial Photobiomodulation With Near-Infrared Light for Language in Individuals With Down Syndrome

NCT04668001
View directory record Official record
Conditions
Down Syndrome
Interventions
Near-Infrared Transcranial Photobiomodulation, Sham Transcranial Photobiomodulation
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
16 Years to 35 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 31, 2024 · Synced Oct 14, 2026, 8:19 AM EDT
Recruiting No phase listed Observational

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

NCT06271512
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2043
U.S. locations
6
States / cities
Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2025 · Synced Oct 14, 2026, 8:19 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations

NCT03278912
View directory record Official record
Conditions
Chronic Granulomatous Disease, Inflammatory Bowel Disease
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Not applicable Interventional

Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With NF1

NCT01058330
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Plyometric training program
Other
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
4 Years to 19 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
2
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Mar 23, 2021 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 1 Interventional

Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease

NCT02867800
View directory record Official record
Conditions
Sickle Cell Disease, Graft Versus Host Disease
Interventions
Diphenhydramine, Acetaminophen, Methylprednisolone, Meperidine, Alemtuzumab, Thymoglobulin, Fludarabine, Melphalan, Thiotepa, Cyclosporine, Tacrolimus, Methotrexate, Abatacept, Marrow infusion, Sirolimus, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Monica Bhatia
Other
Eligibility
3 Years to 20 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
7
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Oct 14, 2026, 8:19 AM EDT
Not listed Not applicable Interventional

Prosthetic Device in Children With Upper Congenital Limb Deficiency

NCT04059107
View directory record Official record
Conditions
Upper Limb Deformities, Congenital
Interventions
3D Printed Myoelectric Prosthetic Device
Device
Lead sponsor
Oregon Health and Science University
Other
Eligibility
6 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Orlando, Florida • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Mar 10, 2021 · Synced Oct 14, 2026, 8:19 AM EDT
Terminated Phase 4 Interventional Results available

Ketorolac Versus Ibuprofen to Treat Painful Episodes of Sickle Cell Disease

NCT00115336
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell
Interventions
Intravenous Ketorolac, Ibuprofen
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 8, 2020 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Not applicable Interventional

Scalable Communication Modalities for Returning Genetic Research Results

NCT04407611
View directory record Official record
Conditions
Hereditary Breast and Ovarian Cancer, Lynch Syndrome
Interventions
Online modality, Genetic counselor follow-up
Behavioral
Lead sponsor
Boston University
Other
Eligibility
40 Years and older · Female only
Enrollment
926 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 14, 2026, 8:19 AM EDT
Recruiting Phase 2 Interventional

Tocilizumab for Acute Chest Syndrome

NCT05640271
View directory record Official record
Conditions
Sickle Cell Disease, Acute Chest Syndrome
Interventions
Tocilizumab
Drug
Lead sponsor
University of Chicago
Other
Eligibility
12 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 1 Interventional

Amicar Pharmacokinetics of Children Having Craniofacial Surgery

NCT00912119
View directory record Official record
Conditions
Craniosynostosis
Interventions
Epsilon-Aminocaproic Acid
Drug
Lead sponsor
Paul Stricker
Other
Eligibility
2 Months to 24 Months
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 1, 2012 · Synced Oct 14, 2026, 8:19 AM EDT
Recruiting Not applicable Interventional

Parent Navigator Program (PNP) to Improve Outcomes in Latino/x Children and Parents

NCT07023367
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Parent Navigator Program, Standard of Care
Behavioral
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
0 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 12, 2025 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Oct 14, 2026, 8:19 AM EDT
Recruiting Phase 3 Interventional

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003, Placebo
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
7 Years to 11 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
3
States / cities
Little Rock, Arkansas • Flower Mound, Texas • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 14, 2026, 8:19 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)

NCT03346434
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Dupilumab, Matching placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
6 Months to 5 Years
Enrollment
202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
33
States / cities
Birmingham, Alabama • Gilbert, Arizona • Long Beach, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2022 · Synced Oct 14, 2026, 8:19 AM EDT