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ClinicalTrials.gov public records Last synced Oct 28, 2026, 9:35 AM EDT

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

Showing 1417–1440 of 8,015 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Terminated Phase 1 Interventional

Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I)

NCT03396575
View directory record Official record
Conditions
Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma
Interventions
TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning, Dose-Intensified TMZ, Td vaccine, Autologous Hematopoietic Stem Cells (HSC)
Biological · Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Years to 30 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 28, 2026, 9:35 AM EDT
Terminated Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

NCT00358657
View directory record Official record
Conditions
Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Sirolimus, Tacrolimus, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
3
States / cities
Nashville, Tennessee • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Optimizing BCI-FIT: Brain Computer Interface - Functional Implementation Toolkit

NCT04468919
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Brainstem Stroke, Muscular Dystrophies, Parkinson's Disease and Parkinsonism, Multiple System Atrophy, Brain Tumor Adult, Spinal Cord Injuries, Locked-in Syndrome
Interventions
BCI-FIT multi-modal access, BCI-FIT adaptive signal modeling, BCI-FIT active querying, BCI-FIT language modeling
Behavioral
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years to 89 Years
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 16, 2025 · Synced Oct 28, 2026, 9:35 AM EDT
Terminated Phase 2 Interventional Results available

Open-Label Study of AG10 in Patients With Cardiomyopathy

NCT03536767
View directory record Official record
Conditions
Amyloid Cardiomyopathy
Interventions
Acoramidis
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
9
States / cities
San Francisco, California • Stanford, California • New Haven, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Dent Disease Mutation Genotyping

NCT01783795
View directory record Official record
Conditions
Dent Disease
Interventions
Genetic Analysis
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2019
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 6, 2020 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 28, 2026, 9:35 AM EDT
Active, not recruiting Phase 2 Interventional

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

NCT06450639
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Satralizumab
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
8 Years to 17 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Little Rock, Arkansas • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Phase 3 Interventional

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

NCT03584165
View directory record Official record
Conditions
Choroideremia, X-Linked Retinitis Pigmentosa
Interventions
BIIB111, BIIB112
Genetic
Lead sponsor
NightstaRx Ltd, a Biogen Company
Other
Eligibility
18 Years and older · Male only
Enrollment
277 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional

Virtual Reality as an Adjuvant Therapy for Sickle Cell Vaso-Occlusive Crisis in the Pediatric Emergency Department

NCT05864092
View directory record Official record
Conditions
Sickle Cell Crisis
Interventions
Virtual Reality Goggles
Other
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
6 Years to 21 Years
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Jun 28, 2024 · Synced Oct 28, 2026, 9:35 AM EDT
Not listed No phase listed Observational

Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD

NCT04406636
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
18 Years to 63 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
San Diego, California • Englewood, Colorado • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2022 · Synced Oct 28, 2026, 9:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

NCT00688844
View directory record Official record
Conditions
Phenylketonuria
Interventions
KuvanTM Therapy
Drug
Lead sponsor
Emory University
Other
Eligibility
4 Years and older
Enrollment
58 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
2
States / cities
Atlanta, Georgia • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 28, 2026, 9:35 AM EDT
Terminated Not applicable Interventional Results available

Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

NCT00478244
View directory record Official record
Conditions
Epidermolysis Bullosa
Interventions
busulfan, cyclophosphamide, fludarabine phosphate, hematopoietic bone marrow transplantation
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 25 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional

Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy

NCT04529707
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Transdiagnostic Behavioral Sleep Intervention
Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
6 Years to 18 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 7, 2025 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Phase 2 Interventional Results available

Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis

NCT05719311
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
Interventions
adrulipase
Drug
Lead sponsor
Entero Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
3
States / cities
Orlando, Florida • Northfield, Illinois • Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 28, 2026, 9:35 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 28, 2026, 9:35 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)

NCT03945292
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Fazisiran Injection, Placebo
Drug · Other
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
16
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2025 · Synced Oct 28, 2026, 9:35 AM EDT
Completed No phase listed Observational

Role of Helicobacter Pylori and Its Toxins in Lung and Digestive System Diseases

NCT00366509
View directory record Official record
Conditions
Pulmonary Disease, Oropharyngeal Disease, Lymphangioleiomyomatosis, Pulmonary Fibrosis, Asthma, Sarcoidosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Phase 2 Interventional Results available

A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

NCT02951195
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-152, TEZ/IVA, IVA, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
16
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2021 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional

An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease

NCT04688411
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
Interventions
MED-Go App
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 28, 2026, 9:35 AM EDT
Available No phase listed Expanded access

Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

NCT06931834
View directory record Official record
Conditions
Mitochondrial Disease, Chronic Kidney Disease
Interventions
Probucol
Drug
Lead sponsor
RiboNova Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 17, 2025 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional Results available

Dietary Supplements for the Treatment of Angelman Syndrome

NCT00348933
View directory record Official record
Conditions
Angelman Syndrome, Nervous System Diseases
Interventions
Betaine, Creatine, Metafolin, Vitamin B12
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
1 Day to 5 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
4
States / cities
San Diego, California • Boston, Massachusetts • Greenwood, South Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2012 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Phase 2 Interventional Results available

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

NCT02418455
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Day to 5 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 30, 2019 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Korean Translation and Validation of the K-HEPI in NYC

NCT05544578
View directory record Official record
Conditions
Disaster, Vulnerable Populations
Interventions
Nurses Taking On Readiness Measures (N- TORM)
Behavioral
Lead sponsor
Hunter College of City University of New York
Other
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 15, 2024 · Synced Oct 28, 2026, 9:35 AM EDT
Completed Phase 2Phase 3 Interventional

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT03655678
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
6
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 28, 2026, 9:35 AM EDT