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These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 24, 2026, 9:04 AM EDT

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

Showing 529–552 of 10,333 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all

Conditions: Gout
Interventions: RDEA3170 10 mg, allopurinol 300 mg; Drug
Lead sponsor: Ardea Biosciences, Inc.; Industry
Eligibility: 18 Years to 75 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Overland Park, Kansas

Conditions: Healthy, Atopic Dermatitis
Interventions: LY3872386, Prednisone, Placebo; Drug
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 18 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2024
U.S. locations: 2
States / cities: Anaheim, California • Daytona Beach, Florida

Conditions: Klinefelter Syndrome, XXY Syndrome
Interventions: Testosterone gel 1%, Placebo gel; Drug
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 8 Years to 18 Years · Male only
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2017
U.S. locations: 1
States / cities: Aurora, Colorado

Conditions: Sanfilippo Syndrome Type C
Interventions: Not listed
Lead sponsor: Phoenix Nest; Industry
Eligibility: 12 Months and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Hemophilia A
Interventions: Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC), Emicizumab; Drug · Biological
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 2
States / cities: Los Angeles, California • Flowood, Mississippi

Conditions: Heart Diseases, Arrhythmogenic Right Ventricular Dysplasia
Interventions: Not listed
Lead sponsor: University of Arizona; Other
Eligibility: 12 Years to 90 Years
Enrollment: 320 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2010
U.S. locations: 1
States / cities: Tucson, Arizona

Conditions: Gorlin Syndrome
Interventions: Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib; Drug
Lead sponsor: Sol-Gel Technologies, Ltd.; Industry
Eligibility: 18 Years and older
Enrollment: 113 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 24
States / cities: Rogers, Arkansas • Fremont, California • Los Angeles, California + 21 more

Conditions: Pectus Excavatum, Pain, Postoperative
Interventions: Ultrasound-guided Sub-Paraspinal Block, PCA only; Procedure
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: 12 Years to 17 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 1
States / cities: Jacksonville, Florida

Conditions: Neurofibromatosis, Neurofibromas
Interventions: Gleevec; Drug
Lead sponsor: Kent Robertson; Other
Eligibility: 3 Years to 65 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Indianapolis, Indiana

Conditions: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions: Genetic characterization; Genetic
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 18 Years and older · Female only
Enrollment: 250 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2030
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Hyperphagia, Prader-Willi Syndrome
Interventions: Placebo, ARD-101; Drug
Lead sponsor: Aardvark Therapeutics, Inc.; Industry
Eligibility: 7 Years and older
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 17
States / cities: Birmingham, Alabama • Encinitas, California • Orange, California + 14 more

Conditions: Sphingomyelin Lipidosis
Interventions: Olipudase alfa; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Up to 17 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: New York, New York

Conditions: Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
Interventions: Beta blocker; Drug
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 65 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Tourette Syndrome
Interventions: D-serine, Riluzole, Placebo; Drug
Lead sponsor: Johns Hopkins University; Other
Eligibility: 8 Years to 17 Years
Enrollment: 39 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Neurofibromatosis 1, Plexiform Neurofibroma
Interventions: Selumetinib; Drug
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 1 Year to 8 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 15
States / cities: Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 11 more

Conditions: Lysosomal Storage Disease, Cholesterol Metabolism
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 1 Day to 99 Years
Enrollment: 344 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2023
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Angelman Syndrome
Interventions: Alogabat; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 5 Years to 17 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 6
States / cities: Chicago, Illinois • Boston, Massachusetts • New York, New York + 3 more

Conditions: Cystic Fibrosis
Interventions: VX-121/TEZ/D-IVA; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 1 Year to 11 Years
Enrollment: 210 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2030
U.S. locations: 19
States / cities: Orange, California • Palo Alto, California • Aurora, Colorado + 16 more

Conditions: Sensorineural Hearing Loss, Long QT Syndrome
Interventions: Not listed
Lead sponsor: Pediatrix; Other
Eligibility: 1 Day to 90 Days
Enrollment: 600 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2020
U.S. locations: 12
States / cities: Anchorage, Alaska • Phoenix, Arizona • Waterford, Connecticut + 6 more

Conditions: Cholesterol, HDL, Lipid Metabolism, Inborn Errors, Tangier Disease, LCAT Deficiency, Cholesteryl Ester Transfer Protein (CETP) Deficiency
Interventions: 3H-cholesterol bound to albumin; Drug
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years to 75 Years
Enrollment: 17 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Leukocyte-Adhesion Deficiency Syndrome
Interventions: modified stem cell transplant; Procedure
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Not listed
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2003
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Epidermolysis Bullosa
Interventions: APR-TD011; Combination Product
Lead sponsor: Northwestern University; Other
Eligibility: 6 Months and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Retinopathy of Prematurity
Interventions: Bevacizumab; Drug
Lead sponsor: Jaeb Center for Health Research; Other
Eligibility: Up to 6 Months
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 11
States / cities: Atlanta, Georgia • Indianapolis, Indiana • Baltimore, Maryland + 8 more

Conditions: Cystic Fibrosis
Interventions: Essential amino acid intake + Leucine vs total AA supplement; Dietary Supplement
Lead sponsor: Texas A&M University; Other
Eligibility: 10 Years to 21 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 1
States / cities: Little Rock, Arkansas

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RDEA3170 and Allopurinol Combination Study in Gout Subjects

A Study of LY3872386 in Healthy Participants and Participants With Atopic Dermatitis

Study of Psychological and Motor Effects of Testosterone in Adolescents With XXY/Klinefelter Syndrome

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Multidisciplinary Study of Right Ventricular Dysplasia

Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome

Sub-Paraspinal Block in Nuss Patients. A Pilot Project

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

N-of-1 for Beta-Blockers in Cardiac Amyloidosis

Developing New Treatments for Tourette Syndrome: Therapeutic Trials With Modulators of Glutamatergic Neurotransmission

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

Study of Inborn Errors of Cholesterol Synthesis and Related Disorders

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Determine the Incidence of Long QT Amongst a Large Cohort of Subjects Diagnosed With Unilateral or Bilateral Sensorineural Hearing Loss.

Mendelian Reverse Cholesterol Transport Study

Stem Cell Transplantation to Treat Leukocyte Adhesion Deficiency

Characterization of the Microbiome in Colonized Dystrophic and Junctional Epidermolysis Bullosa Wounds Before and After Use of APR-TD011 ® Spray Solution

Phase 1 Trial of Bevacizumab Treatment for Severe Retinopathy of Prematurity

Leucine-enriched Essential Amino Acid Intake to Optimize Protein Anabolism in Children With Cystic Fibrosis