Search | Clinical Trials Finder

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 9, 2026, 1:05 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 1441–1464 of 10,357 matching trials from the live ClinicalTrials.gov search.

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

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: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2039
U.S. locations: 9
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 5 more

Conditions: Advanced Solid Tumors
Interventions: Elimusertib (BAY1895344), Pembrolizumab (Keytruda®); Drug
Lead sponsor: Bayer; Industry
Eligibility: 18 Years and older
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 8
States / cities: Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more

Conditions: Genitourinary Abnormality, Breast Cancer Female, Vaginal Abnormality, Urinary Tract Infections, Dyspareunia, Dysuria
Interventions: Replens and coconut oil; Combination Product
Lead sponsor: Alta Bates Summit Medical Center; Other
Eligibility: Female only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Berkeley, California

Conditions: Esophageal Neoplasms, Cancer of Esophagus, Cancer of the Esophagus, Esophageal Cancer, Esophagus Cancer, Neoplasm, Esophageal, Esophagectomy, Abnormalities, Digestive System, Digestive System Abnormalities
Interventions: Mayo Clinic Upper Digestive Disease Survey on Paper, Upper Digestive Disease (UDD Monitor) App; Other
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2028
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Porphyria Cutanea Tarda
Interventions: Hydroxychloroquine, Phlebotomy; Drug · Procedure
Lead sponsor: The University of Texas Medical Branch, Galveston; Other
Eligibility: 18 Years to 100 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2021
U.S. locations: 1
States / cities: Galveston, Texas

Conditions: Burden, Caregiver
Interventions: Yoga; Support Group; Behavioral
Lead sponsor: University of Kansas Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Kansas City, Kansas

Conditions: Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma, Wilms Tumor, Sarcoma, Spindle Cell, Sarcoma Rhabdoid, Childhood Sarcoma of Soft Tissue
Interventions: Not listed
Lead sponsor: Florida International University; Other
Eligibility: Up to 21 Years
Enrollment: 14 participants
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Vestibular Schwannoma, Neurofibromatosis Type 2
Interventions: bevacizumab, laboratory biomarker analysis, quality-of-life assessment; Biological · Other · Procedure
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2014
U.S. locations: 3
States / cities: Baltimore, Maryland • Rockville, Maryland • Charlestown, Massachusetts

Conditions: Cystic Fibrosis
Interventions: ELX/TEZ/IVA, IVA; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years to 5 Years
Enrollment: 83 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 13
States / cities: Tucson, Arizona • Palo Alto, California • Aurora, Colorado + 10 more

Conditions: Leber Hereditary Optic Neuropathy
Interventions: GS010, Sham Intravitreal Injection; Genetic · Other
Lead sponsor: GenSight Biologics; Industry
Eligibility: 15 Years and older
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 3
States / cities: Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania

Conditions: Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
Interventions: Hematopoietic Stem Cell Transplant; Biological
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: 4 Months to 45 Years
Enrollment: 47 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 19
States / cities: Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more

Conditions: Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions: Myozyme; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2006
U.S. locations: 5
States / cities: Oakland, California • Orange, California • Omaha, Nebraska + 2 more

Conditions: Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor
Interventions: Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Erdafitinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging; Procedure · Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 21 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 114
States / cities: Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more

Conditions: Solid Tumor, Renal Cell Carcinoma (Kidney Cancer), Gastric and Gastroesophageal Junction (GEJ) Adenocarcinoma, Non-Small Cell Lung Cancer
Interventions: NDI-101150, Pembrolizumab; Drug
Lead sponsor: Nimbus Saturn, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 106 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 19
States / cities: Scottsdale, Arizona • New Haven, Connecticut • Washington D.C., District of Columbia + 15 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: Cystic Fibrosis
Interventions: Not listed
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 10 Years and older
Enrollment: 123 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2014
U.S. locations: 5
States / cities: Denver, Colorado • Indianapolis, Indiana • Ann Arbor, Michigan + 2 more

Conditions: Congenital Vertical Talus, Familial Encephalopathy With Neuroserpin Inclusion Bodies, Idiopathic Generalised Epilepsy, Familial Dementia, X-linked Mental Retardation
Interventions: Not listed
Lead sponsor: State University of New York - Upstate Medical University; Other
Eligibility: 6 Months and older
Enrollment: 176 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2015
U.S. locations: 1
States / cities: Syracuse, New York

Conditions: Chronic Renal Failure
Interventions: Blood Draw; Procedure
Lead sponsor: University of Oklahoma; Other
Eligibility: 18 Years to 90 Years
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Tulsa, Oklahoma

Conditions: Myelomeningocele, Pregnancy
Interventions: Open Fetal Repair of Myelomeningocele; Procedure
Lead sponsor: St. Louis University; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Gout, Hyperuricemia
Interventions: Xanthine Oxidase Inhibitor; Drug
Lead sponsor: LG Chem; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019
U.S. locations: 1
States / cities: Austin, Texas

Conditions: Healthy, Men, Male Contraception
Interventions: Dimethandrolone-Undecanoate, Levonorgestrel 0.03 MG, Placebo oral capsule; Drug
Lead sponsor: Premier Research; Other
Eligibility: 18 Years to 50 Years · Male only
Enrollment: 92 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2020
U.S. locations: 2
States / cities: Torrance, California • Seattle, Washington

Conditions: Progeria, Hutchinson-Gilford Syndrome
Interventions: Lonafarnib; Drug
Lead sponsor: Monica E. Kleinman; Other
Eligibility: 1 Year and older
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Congenital Disorders
Interventions: Not listed
Lead sponsor: Emory University; Other
Eligibility: 1 Day to 30 Days
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Atlanta, Georgia

Search by objective public record fields.

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.

A Long-term Follow-up Study in Participants Who Received CTX001

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

GSM (Genitourinary Syndrome Of Menopause) Management In Breast Cancer Survivors

Mayo Clinic Upper Digestive Disease Survey

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

Social Support and Stress Reduction for Caregivers of Young Adults With IDD

Drug Sensitivity and Mutation Profiling

Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)

Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years

RESCUE and REVERSE Long-term Follow-up

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

A Study of NDI 1150-101 in Patients With Solid Tumors

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

Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

Genetic Disease Gene Identification

A Study of Cytokine and Inflammatory Markers in Patients With Chronic Renal Failure

Fetal Myelomeningocele Repair With Maternal BMI Between 35.0 and 40.0

The Effect of Colchicine on the Pharmacokinetic Profile of LC350189 in Healthy Aldults.

Study of Spermatogenesis Suppression With DMAU Alone or With LNG Versus Placebo Alone in Normal Men

Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

Biomarkers for Detection of Brain Ischemia