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ClinicalTrials.gov public records Last synced Nov 4, 2026, 12:58 PM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 2761–2784 of 10,824 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Developmental Defect Clear all

Conditions: Fabry Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: 18 Years and older · Male only
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Skin Cancer, Skin Condition, Skin Diseases, Skin Lesion, Skin Abnormalities
Interventions: VIO System; Device
Lead sponsor: Enspectra Health; Industry
Eligibility: 2 Years to 90 Years
Enrollment: 122 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Mountain View, California

Conditions: Hemophilia A
Interventions: Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose), BIVV001 (High Dose); Biological
Lead sponsor: Bioverativ, a Sanofi company; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 7
States / cities: Los Angeles, California • Indianapolis, Indiana • Iowa City, Iowa + 4 more

Conditions: Hemophilia A, Hemophilia B
Interventions: MOD-5014; Drug
Lead sponsor: OPKO Health, Inc.; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 11
States / cities: Duarte, California • Los Angeles, California • Sacramento, California + 7 more

Conditions: Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Day to 100 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Fucosidosis
Interventions: Not listed
Lead sponsor: JCR Pharmaceuticals Co., Ltd.; Industry
Eligibility: Not listed
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 2
States / cities: Orange, California • Minneapolis, Minnesota

Conditions: Keratoconus, Corneal Opacity, Corneal Dystrophy
Interventions: Optical Coherence Tomography; Device
Lead sponsor: Oregon Health and Science University; Other
Eligibility: 14 Years to 85 Years
Enrollment: 445 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2031
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Muscle Weakness, Problems Moving Their Arms and Legs
Interventions: Extension assist knee ankle foot orthosis (EA-KAFO); Device
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 5 Years to 25 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Familial Hypercholesterolemia
Interventions: Genetic testing; Other
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 378 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2018
U.S. locations: 2
States / cities: Lancaster, Pennsylvania • Philadelphia, Pennsylvania

Conditions: Sleep Disturbance in Infancy (Disorder), Neonatal Disease
Interventions: Novel Real-time Neonatal Sleep Stage Detection Algorithm; Other
Lead sponsor: University of Michigan; Other
Eligibility: 1 Day to 1 Year
Enrollment: 39 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Cystic Fibrosis, Advanced Lung Disease
Interventions: Lumacaftor, Ivacaftor; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years and older
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 6
States / cities: Denver, Colorado • Tampa, Florida • Chicago, Illinois + 3 more

Conditions: Infant, Premature, Congenital Heart Disease, Patent Ductus Arteriosus, Necrotizing Enterocolitis
Interventions: Near-infrared spectroscopy (NIRS); Procedure
Lead sponsor: University of Utah; Other
Eligibility: 12 Days to 6 Months
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2022
U.S. locations: 3
States / cities: Murray, Utah • Salt Lake City, Utah

Conditions: Bronchopulmonary Dysplasia, Neurally Adjusted Ventilatory Assist, Mechanical Ventilation
Interventions: NAVA, CMV; Device
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Up to 24 Months
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Providence, Rhode Island

Conditions: Gaucher Disease
Interventions: eliglustat; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 79 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2017
U.S. locations: 3
States / cities: Miami, Florida • Saint Paul, Minnesota • Knoxville, Tennessee

Conditions: Neurofibromatosis Type 1
Interventions: Selumetinib granule formulation, Selumetinib capsule formulation; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 1 Year to 6 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 4
States / cities: Akron, Ohio • Philadelphia, Pennsylvania • Houston, Texas + 1 more

Conditions: Bone Marrow Transplant, Sickle Cell Disease, G-CSF
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 816 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2021
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cystinuria
Interventions: Dapagliflozin; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Tourette Syndrome
Interventions: NBI-98854, Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 98 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 44
States / cities: Sun City, Arizona • Springdale, Arkansas • Long Beach, California + 41 more

Conditions: Amyloid Cardiomyopathy, Transthyretin-Related
Interventions: Acoramidis (AG10); Drug
Lead sponsor: Eidos Therapeutics, a BridgeBio company; Industry
Eligibility: 18 Years to 90 Years
Enrollment: 389 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 26
States / cities: Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more

Conditions: Sickle Cell Disease
Interventions: Ticagrelor, Placebo; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 193 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 7
States / cities: Fort Lauderdale, Florida • Miami, Florida • Chicago, Illinois + 4 more

Conditions: Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions: VAD044 Part I, VAD044 Part II; Drug
Lead sponsor: Vaderis Therapeutics AG; Industry
Eligibility: 18 Years and older
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Down Syndrome
Interventions: Placebo, RO5186582; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 6 Years to 11 Years
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 9
States / cities: Phoenix, Arizona • Decatur, Georgia • Chicago, Illinois + 5 more

Conditions: Nemaline Myopathy
Interventions: Not listed
Lead sponsor: Stanford University; Other
Eligibility: 0 Years to 18 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 5
States / cities: Palo Alto, California • Bethesda, Maryland • Boston, Massachusetts + 2 more

Conditions: DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome, Complete Atypical DiGeorge Syndrome
Interventions: Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw, Rabbit anti-thymocyte globulin, Cyclosporine, Tacrolimus, Methylprednisolone or Prednisolone, Daclizumab, Mycophenolate mofetil; Biological · Other · Procedure + 1 more
Lead sponsor: Sumitomo Pharma Switzerland GmbH; Industry
Eligibility: Not listed
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2017
U.S. locations: 1
States / cities: Durham, North Carolina

Search by objective public record fields.

Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity

Non-Linear Imaging of Skin In Vivo

A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)

A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

A Long-Term Observational Study of Patients With Fucosidosis

OCT in Diagnosis of Irregular Corneas

Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults

IN-TANDEM Familial Hypercholesterolemia Pilot Study

Real-time State of Vigilance Monitor for the Neonatal Intensive Care Unit

Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

Splanchnic and Renal Tissue Oxygenation During Enteral Feedings in Neonates With Patent Ductus Arteriosus

NAVA vs. CMV Crossover in Severe BPD

A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

Screening for Subjects to Participate in Studies of Blood Disorders

Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics

Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease

Assess Safety and Efficacy of VAD044 in HHT Patients

A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age

Nemaline Myopathy Clinical Research Network (NM-CTRN)

Phase I/II Thymus Transplantation With Immunosuppression #950