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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 Nov 5, 2026, 12:06 AM 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 1177–1200 of 10,355 matching trials from the live ClinicalTrials.gov search.

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

Conditions: Cystic Fibrosis, Bronchiectasis
Interventions: BI 1291583, Placebo to BI 1291583; Drug
Lead sponsor: Boehringer Ingelheim; Industry
Eligibility: 18 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024
U.S. locations: 7
States / cities: Denver, Colorado • Altamonte Springs, Florida • South Portland, Maine + 3 more

Conditions: Apnea of Prematurity, Intermittent Hypoxemia, Bronchopulmonary Dysplasia of Newborn
Interventions: Chinstrap; Device
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 3 Days to 3 Months
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2027
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Healthy Adults (Full Study and Extension Phase), Hypertrophic Cardiomyopathy or Dilated Cardiomyopathy
Interventions: Family History + Whole Genome Sequencing, Family History Only; Other
Lead sponsor: Brigham and Women's Hospital; Other
Eligibility: 18 Years to 90 Years
Enrollment: 213 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2021
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Tourette Syndrome
Interventions: levodopa solution 2mg/ml for i.v. use, placebo; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 18 Years to 55 Years
Enrollment: 49 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Cystic Fibrosis
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 9 Years to 65 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2008
U.S. locations: 2
States / cities: Bethesda, Maryland • Milwaukee, Wisconsin

Conditions: Hypoplastic Left Heart Syndrome (HLHS)
Interventions: Collaborative Learning Network; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: Up to 15 Months
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2028
U.S. locations: 61
States / cities: Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 53 more

Conditions: Hypertrophic Cardiomyopathy (HCM), Cardiomyopathy, Genetic Disease, Cardiology
Interventions: Large Language Model; Other
Lead sponsor: Stanford University; Other
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Kidney, Polycystic, Abdominal Pain
Interventions: Octreotide, Placebo; Drug
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years to 80 Years
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Bronchopulmonary Dysplasia
Interventions: Tobramycin solution for inhalation 78mg dose, Tobramycin solution for inhalation 150mg dose, Tobramycin solution for inhalation 216mg dose, Tobramycin solution for inhalation 300mg dose; Drug
Lead sponsor: Erik Allen Jensen; Other
Eligibility: 4 Weeks and older
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Epidermolysis Bullosa, Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Dystrophica
Interventions: No intervention; Other
Lead sponsor: ProgenaBiome; Other
Eligibility: Not listed
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Ventura, California

Conditions: Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions: laronidase; Drug
Lead sponsor: Patricia I. Dickson, M.D.; Individual
Eligibility: 8 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 1
States / cities: Torrance, California

Conditions: Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
Interventions: Haploidentical Hematopoietic Cell Transplantation; Biological
Lead sponsor: Johns Hopkins All Children's Hospital; Other
Eligibility: 0 Years to 21 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 1
States / cities: St. Petersburg, Florida

Conditions: Limb Girdle Muscular Dystrophy
Interventions: SRP-6004; Genetic
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Respiratory Distress Syndrome, Newborn, Pulmonary Surfactants, Intensive Care Units, Neonatal
Interventions: Echography-guided Surfactant THERapy (ESTHER); Diagnostic Test
Lead sponsor: Connecticut Children's Medical Center; Other
Eligibility: 0 Hours to 2 Hours
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Hartford, Connecticut

Conditions: Bronchopulmonary Dysplasia
Interventions: Lung Ultrasound; Diagnostic Test
Lead sponsor: Phoenix Children's Hospital; Other
Eligibility: 0 Days and older
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Phoenix, Arizona

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

Conditions: Osteogenesis Imperfecta
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 10 Years to 100 Years
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2027
U.S. locations: 2
States / cities: Los Angeles, California • Houston, Texas

Conditions: Atopic Dermatitis
Interventions: Tradipitant, Placebo; Drug
Lead sponsor: Vanda Pharmaceuticals; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 375 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 70
States / cities: Birmingham, Alabama • Tempe, Arizona • Encino, California + 60 more

Conditions: Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
Interventions: SB-007; Genetic
Lead sponsor: Splice Bio; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 5
States / cities: Aurora, Colorado • Miami, Florida • Boston, Massachusetts + 2 more

Conditions: Ataxia-Telangiectasia
Interventions: vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine, dexamethasone, procarbazine, chemotherapy, intrathecal chemotherapy, steroid therapy; Drug · Procedure
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 10 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2013
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Friedreich Ataxia
Interventions: Idebenone; Drug
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Polycystic Kidney
Interventions: Lixivaptan; Drug
Lead sponsor: Palladio Biosciences; Industry
Eligibility: 15 Years to 60 Years
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Ewing Sarcoma, Rhabdomyosarcoma, Wilms Tumor, Neuroblastoma, Hepatoblastoma, Germ Cell Tumor
Interventions: Vorinostat; Drug
Lead sponsor: New York Medical College; Other
Eligibility: 1 Year to 30 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2027
U.S. locations: 1
States / cities: Valhalla, New York

Conditions: Hemophilia A
Interventions: AAV2/8-HLP-FVIII-V3; Biological
Lead sponsor: University College, London; Other
Eligibility: 18 Years and older · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2029
U.S. locations: 3
States / cities: Boise, Idaho • Lexington, Kentucky • Memphis, Tennessee

Search by objective public record fields.

A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairafly™)

Strap To Reduce Apnea in Preterm Infants: A Randomized Crossover Trial

A Pilot Project Exploring the Impact of Whole Genome Sequencing in Healthcare

Dopaminergic Effects on Cortical Function in Tourette's (Levodopa Protocol)

Tissue Collection From People With Cystic Fibrosis

National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

Large Language Models To Improve the Quality of Care of Cardiology Patients

Octreotide in Severe Polycystic Liver Disease

Inhaled Tobramycin in BPD

A Pilot Study to Explore the Role of Gut Flora in Epidermolysis Bullosa

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)

Echography-guided Surfactant THERapy (ESTHER) For Preterm Infants With Respiratory Failure

Point of Care Lung Ultrasound in Preterm Infants With Respiratory Distress

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

Dental Malocclusion and Craniofacial Development in OI

Evaluating the Effects of Tradipitant vs. Placebo in Atopic Dermatitis (EPIONE)

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Study for Treatment of Cancer in Children With Ataxia-telangiectasia

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

Lixivaptan in a Single Subject With Intractable Pain Due to Polycystic Kidney Disease

Vorinostat in Combination With Chemotherapy in Relapsed/Refractory Solid Tumors and CNS Malignancies

Gene Therapy for Haemophilia A.