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ClinicalTrials.gov public records Last synced Oct 22, 2026, 2:57 PM EDT

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Showing 673–696 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Not applicable Interventional Results available

Low-Cost Platelet-Rich Plasma for Hemarthropathy

NCT06543368
View directory record Official record
Conditions
Hemarthrosis, Hemophilia
Interventions
Intra articular PRP Injection
Biological
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Oct 22, 2026, 2:57 PM EDT
Recruiting Phase 2Phase 3 Interventional

Maralixibat in Patients With Cystic Fibrosis and Constipation

NCT06413368
View directory record Official record
Conditions
Constipation Chronic Idiopathic, Cystic Fibrosis
Interventions
Maralixibat 9.5 MG/ML [Livmarli]
Drug
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
1 Year to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Not applicable Interventional

Regeneron 1331 Kinetics Sub-Study HoFH

NCT04722068
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
Kinetics test
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Oct 22, 2026, 2:57 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

A Prospective Observational Study to Assess the Reliability and Validity of the MLSDT

NCT06805474
View directory record Official record
Conditions
Macular Degeneration, Geographic Atrophy, Stargardt Disease
Interventions
Observational
Other
Lead sponsor
Nanoscope Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 24, 2025 · Synced Oct 22, 2026, 2:57 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

NCT06089018
View directory record Official record
Conditions
Myotonic Dystrophy 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
12 Years to 90 Years
Enrollment
125 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Phase 3 Interventional Results available

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

NCT05076149
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA)
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
597 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
62
States / cities
Birmingham, Alabama • Tucson, Arizona • Little Rock, Arkansas + 55 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial of Rituximab for Graves' Ophthalmopathy

NCT00595335
View directory record Official record
Conditions
Thyroid-associated Ophthalmopathy
Interventions
Rituximab, Saline, Methylprednisolone
Drug
Lead sponsor
Rebecca Bahn
Other
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 1, 2016 · Synced Oct 22, 2026, 2:57 PM EDT
Recruiting Phase 2 Interventional

Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial

NCT06260891
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
25 mg/day zinc, 40 mg/day zinc
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
15 Years to 40 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
6
States / cities
Oakland, California • Durham, North Carolina • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 22, 2026, 2:57 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

PatientSpot Formerly Known as ArthritisPower

NCT03840928
View directory record Official record
Conditions
Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
Interventions
Not listed
Lead sponsor
Global Healthy Living Foundation
Other
Eligibility
19 Years and older
Enrollment
40,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Upper Nyack, New York
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 22, 2026, 2:57 PM EDT
Terminated No phase listed Observational

Screening for Lysosomal Acid Lipase Deficiency

NCT02926872
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
There is no intervention in the study
Other
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 16 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Atlanta, Georgia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 16, 2017 · Synced Oct 22, 2026, 2:57 PM EDT
Enrolling by invitation Phase 3 Interventional

Long-term Efficacy and Safety of OLE LIB003 in HoFH, HeFH, and High-risk CVD Patients Requiring Further LDL-C Reduction

NCT04798430
View directory record Official record
Conditions
Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol, Familial Hypercholesterolemia
Interventions
lerodalcibep
Drug
Lead sponsor
LIB Therapeutics LLC
Industry
Eligibility
10 Years and older
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
4
States / cities
Evanston, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 22, 2026, 2:57 PM EDT
Terminated Phase 2 Interventional Results available

A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis

NCT03738800
View directory record Official record
Conditions
Lamellar Ichthyosis
Interventions
CD5789 Cream 200 µg/g, CD5789 Cream 100 µg/g, CD5789 Cream Vehicle
Drug
Lead sponsor
Mayne Pharma International Pty Ltd
Industry
Eligibility
12 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
10
States / cities
San Diego, California • Aurora, Colorado • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2023 · Synced Oct 22, 2026, 2:57 PM EDT
Active, not recruiting Phase 3 Interventional Accepts healthy volunteers

Molecular Imaging of Myocardial Fibrosis in Cardiac Amyloidosis

NCT05619302
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[68Ga]CBP8 PET/MRI
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 22, 2026, 2:57 PM EDT
Terminated Phase 4 Interventional Results available

INdividualized ITI Based on Fviii(ATE) Protection by VWF

NCT03204539
View directory record Official record
Conditions
Hemophilia A With Inhibitor, Hemophilia A
Interventions
Wilate
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
3
States / cities
Sacramento, California • San Diego, California • New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated May 29, 2020 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Phase 1 Interventional

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

NCT04428281
View directory record Official record
Conditions
Angelman Syndrome
Interventions
RO7248824
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
1 Year to 12 Years
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
7
States / cities
Los Angeles, California • San Diego, California • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2025 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Phase 1 Interventional

Use of Busulfan as Conditioning Agent for a Second Stem Cell Transplant

NCT00092937
View directory record Official record
Conditions
Granulomatous Disease, Chronic
Interventions
Busulfan
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 22, 2026, 2:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Cherubism Observational Study

NCT01916772
View directory record Official record
Conditions
Cherubism
Interventions
no interventions
Other
Lead sponsor
The Cleveland Clinic
Other
Eligibility
Not listed
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 10, 2017 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

NCT04729751
View directory record Official record
Conditions
Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
Interventions
Maralixibat
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
0 Days to 364 Days
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
7
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 22, 2026, 2:57 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Bone Loss, Physical Function and Frailty in Older Women With Sickle Cell Trait Sickle Cell Trait

NCT07083531
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Trait
Interventions
Healthy volunteers without SCT, Healthy volunteers with SCT
Other
Lead sponsor
UConn Health
Other
Eligibility
50 Years and older · Female only
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Farmington, Connecticut
Source: ClinicalTrials.gov public record
Updated Jul 24, 2025 · Synced Oct 22, 2026, 2:57 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders

NCT00579137
View directory record Official record
Conditions
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
Interventions
Campath -1H, Fludarabine, Anti-CD45, Stem cell infusion
Biological · Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 2, 2013 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Phase 3 Interventional Results available

Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain

NCT02471339
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibromas
Interventions
Acceptance and Commitment Therapy (ACT)
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
16 Years to 59 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 14, 2022 · Synced Oct 22, 2026, 2:57 PM EDT
Terminated Phase 2 Interventional Results available

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 18, 2019 · Synced Oct 22, 2026, 2:57 PM EDT
Completed No phase listed Observational Results available

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

NCT02354651
View directory record Official record
Conditions
Pompe Disease
Interventions
Diaphragm conditioning via phrenic nerve stimulation
Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 22, 2026, 2:57 PM EDT
Completed Not applicable Interventional

Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

NCT02197845
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Patient Navigator, Recruitment into Specialty Care
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
15 Years and older
Enrollment
353 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 2, 2023 · Synced Oct 22, 2026, 2:57 PM EDT