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 Oct 25, 2026, 12:01 AM EDT

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

Showing 625–648 of 10,333 matching trials from the live ClinicalTrials.gov search.

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

Conditions: Sickle Cell Disease
Interventions: Not listed
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 16 Years and older
Enrollment: 297 participants
Timeline: 2021 – 2022
U.S. locations: 11
States / cities: Birmingham, Alabama • Wilmington, Delaware • St. Petersburg, Florida + 8 more

Conditions: Thyroid Eye Disease
Interventions: SENSIMED Triggerfish; Device
Lead sponsor: Sensimed AG; Industry
Eligibility: 18 Years to 90 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013
U.S. locations: 1
States / cities: La Jolla, California

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: Hyperoxaluria, Primary
Interventions: Oxabact OC5 - Oxalobacter formigenes Strain HC-1; Biological
Lead sponsor: OxThera; Industry
Eligibility: 3 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Endometrial Cancer, Lynch Syndrome, Screening, Early Detection of Cancer
Interventions: Menstrual cup use; Device
Lead sponsor: Jessica D. St. Laurent, MD; Other
Eligibility: 18 Years and older · Female only
Enrollment: 25 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Pediatric Cancer, Sickle Cell Disease, Quality of Life, Depressive Symptoms, Coping Skills
Interventions: FOCUS; Behavioral
Lead sponsor: Georgia State University; Other
Eligibility: 12 Years to 18 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 2
States / cities: Atlanta, Georgia

Conditions: Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia, Organic Acidemia
Interventions: BBP-671, Placebo; Drug
Lead sponsor: CoA Therapeutics, Inc., a BridgeBio company; Industry
Eligibility: 15 Years to 55 Years
Enrollment: 79 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2023
U.S. locations: 3
States / cities: Topeka, Indiana • Pittsburgh, Pennsylvania • Austin, Texas

Conditions: Congenital Disorders
Interventions: Not listed
Lead sponsor: Children's Healthcare of Atlanta; Other
Eligibility: Up to 21 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Zidovudine, Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1990
U.S. locations: 3
States / cities: San Diego, California • New York, New York • Rochester, New York

Conditions: Congenital Diaphragmatic Hernia
Interventions: BALT GOLDBAL2 Balloon; Device
Lead sponsor: Inna Lobeck; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2032
U.S. locations: 1
States / cities: Kansas City, Missouri

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

Conditions: Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
Interventions: Not listed
Lead sponsor: Arbor Research Collaborative for Health; Other
Eligibility: Up to 25 Years
Enrollment: 1,675 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2029
U.S. locations: 16
States / cities: Los Angeles, California • San Francisco, California • Aurora, Colorado + 12 more

Conditions: Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Interventions: XOPH5 Ointment, Placebo; Drug
Lead sponsor: Topokine Therapeutics, Inc.; Industry
Eligibility: 40 Years to 70 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
Interventions: muromonab-CD3, natural killer cell therapy, fludarabine phosphate, methotrexate, thiotepa, gene expression analysis, flow cytometry, immunologic technique, allogeneic hematopoietic stem cell transplantation, in vitro-treated peripheral blood stem cell transplantation, total-body irradiation; Biological · Drug · Genetic + 3 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Up to 45 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 2
States / cities: Seattle, Washington

Conditions: Smith-Magenis Syndrome (SMS)
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 115 Years
Enrollment: 593 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease
Interventions: Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, Mesna; Procedure · Drug · Radiation
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: 5 Years to 45 Years
Enrollment: 95 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2025
U.S. locations: 32
States / cities: Birmingham, Alabama • Oakland, California • Aurora, Colorado + 24 more

Conditions: Turner Syndrome
Interventions: Not listed
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: 2 Weeks to 2 Years
Enrollment: 295 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2014
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Epidermolysis Bullosa Simplex
Interventions: TolaSure Topical Gel, Vehicle Topical Gel; Drug
Lead sponsor: BioMendics, LLC; Industry
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 1
States / cities: Redwood City, California

Conditions: Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy, Pseudohypoparathyroidism Type 1a
Interventions: Theophylline, Placebo; Drug
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 2 Years to 12 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Congenital Heart Disease, Heart Defects, Congenital
Interventions: Cardiopulmonary rehabilitation, Standard of care; Other
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 16 Years and older
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
Interventions: rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 5 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2001
U.S. locations: 2
States / cities: New York, New York • Chapel Hill, North Carolina

Conditions: Sickle Cell Anemia
Interventions: Blood smear education, standard education; Behavioral · Other
Lead sponsor: Duke University; Other
Eligibility: 2 Years to 17 Years
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2012
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Plagiocephaly, Nonsynostotic
Interventions: Serkel 3D CRO; Device
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 4 Months to 18 Months
Enrollment: 18 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome, Tuberous Sclerosis, Focal Seizures
Interventions: Turmeric; Dietary Supplement
Lead sponsor: NYU Langone Health; Other
Eligibility: 1 Year to 70 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 1
States / cities: New York, New York

Search by objective public record fields.

Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers

Twenty-four Hour Intraocular Pressure Patterns in Patients With Uncontrolled Thyroid Eye Disease

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

An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

Menstrual Cup for Early Endometrial Cancer Detection in Lynch Syndrome

FOCUS for Pediatric Sickle Cell Disease and Cancer

A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia

Cath Lab Performance Improvement

A Study of AZT Plus Ganciclovir in Patients With AIDS and Cytomegalovirus (CMV) Infection

Infant Survival and Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia, A Phase III Trial

Natural History of Cherubism Observational Study

Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)

Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)

Donor Peripheral Stem Cell Transplant and Donor Natural Killer Cell Transplant After Total-Body Irradiation, Thiotepa, Fludarabine, and Muromonab-CD3 in Treating Patients With Leukemia or Other Blood Diseases

Natural History Study of Smith-Magenis Syndrome

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

Sex Differences in Early Brain Development; Brain Development in Turner Syndrome

Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Cardiopulmonary Rehabilitation for Adolescents and Adults With Congenital Heart Disease

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Adherence to Hydroxyurea in Children With Sickle Cell Anemia

Observe the Effects of the Serkel Cranial Remolding Orthosis on Infants With Deformational Plagiocephaly

Turmeric as Treatment in Epilepsy