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ClinicalTrials.gov public records Last synced Nov 7, 2026, 12:15 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 1417–1440 of 10,356 matching trials from the live ClinicalTrials.gov search.

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

Conditions: Sickle Cell Anemia (HbSS), Sickle-β0-thalassemia (HbSβ0)
Interventions: PK-optimized oral hydroxyurea at MTD until 15 years of age.; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 6 Months and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Retinopathy of Prematurity (ROP)
Interventions: Ranibizumab; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 6 Months to 5 Years
Enrollment: 180 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 9
States / cities: Sacramento, California • Aurora, Colorado • Chicago, Illinois + 6 more

Conditions: Infertility, Hereditary Diseases
Interventions: Preimplantation Genetic Testing; Other
Lead sponsor: Genomic Prediction Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2025
U.S. locations: 1
States / cities: North Brunswick, New Jersey

Conditions: Atopic Dermatitis
Interventions: CD2027 Ointment 3 mcg/g, twice daily, Vehicle Ointment, twice daily; Drug
Lead sponsor: Galderma R&D; Industry
Eligibility: 18 Years and older
Enrollment: 102 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009
U.S. locations: 14
States / cities: Hot Springs, Arkansas • San Diego, California • Denver, Colorado + 10 more

Conditions: Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
Interventions: Not listed
Lead sponsor: University of Chicago; Other
Eligibility: 1 Day to 18 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 3
States / cities: Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York

Conditions: Neurofibromatosis 1, Social Skills
Interventions: PEERS; Behavioral
Lead sponsor: University of Wisconsin, Milwaukee; Other
Eligibility: 12 Years to 17 Years
Enrollment: 8 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: Cystic Fibrosis, Diabetes Mellitus
Interventions: Insulin Asparte, Repaglinide; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: 16 Years and older
Enrollment: 108 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2001 – 2007
U.S. locations: 6
States / cities: Palo Alto, California • Springfield, Massachusetts • Minneapolis, Minnesota + 3 more

Conditions: IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
Interventions: FODMAP-targeting digestive enzyme blend, Placebo; Dietary Supplement · Other
Lead sponsor: Kiwi Health Inc; Industry
Eligibility: 18 Years and older
Enrollment: 150 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Alpha-1 Antitrypsin Deficiency
Interventions: No Intervention; Genetic
Lead sponsor: Adverum Biotechnologies, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 2
States / cities: Gainesville, Florida • Charleston, South Carolina

Conditions: Wilson Disease
Interventions: Not listed
Lead sponsor: Yale University; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Healthy Volunteers
Interventions: Iberdomide; Drug
Lead sponsor: Bristol-Myers Squibb; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023
U.S. locations: 1
States / cities: Daytona Beach, Florida

Conditions: Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Secondary Acute Myeloid Leukemia
Interventions: eltrombopag olamine; Drug
Lead sponsor: Case Comprehensive Cancer Center; Other
Eligibility: 60 Years and older
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Amniotic Band Syndrome, Upper Extremity Deformities, Congenital
Interventions: 3D Printed Upper-limb Prosthesis, Home Intervention; Device · Behavioral
Lead sponsor: University of Nebraska; Other
Eligibility: 3 Years to 18 Years
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Omaha, Nebraska

Conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions: alglucosidase alfa, Placebo; Biological · Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 5
States / cities: Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more

Conditions: Cleft Palate
Interventions: Intensive Feedback, Routine Feedback; Behavioral
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: Not listed
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 5
States / cities: Phoenix, Arizona • Durham, North Carolina • Cincinnati, Ohio + 2 more

Conditions: Sickle Cell Anemia
Interventions: beet juice (Unbeetable); Drug
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 19 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Winston-Salem, North Carolina

Conditions: Epidermolysis Bullosa
Interventions: SD-101 dermal cream (3%), SD-101 Dermal Cream (6%), Vehicle (SD-101 0%); Drug
Lead sponsor: Scioderm, Inc.; Industry
Eligibility: 6 Months and older
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014
U.S. locations: 7
States / cities: Palo Alto, California • Chicago, Illinois • St Louis, Missouri + 4 more

Conditions: Sickle Cell Disease, Obesity, Childhood, Hypertension, Hyperlipidemia in Children, Hyperglycemia
Interventions: Not listed
Lead sponsor: University of Mississippi Medical Center; Other
Eligibility: 10 Years to 19 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Jackson, Mississippi

Conditions: Sickle Cell Disease
Interventions: ICL670; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 185 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 31
States / cities: Mobile, Alabama • Loma Linda, California • Los Angeles, California + 25 more

Conditions: Down Syndrome, Speech and Language Disorder, Speech Disorders in Children, Speech Intelligibility, Speech Sound Disorder
Interventions: AAC Generative Language Intervention, Standard of Care / Business as Usual; Behavioral
Lead sponsor: University of Central Florida; Other
Eligibility: 3 Years to 4 Years
Enrollment: 70 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2023
U.S. locations: 2
States / cities: Orlando, Florida • Albuquerque, New Mexico

Conditions: Duchenne Muscular Dystrophy
Interventions: Prednisolone; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 1 Month to 30 Months · Male only
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 6
States / cities: Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more

Conditions: Congenital Heart Disease
Interventions: Angiotensin Converting Enzyme Inhibitor, No ACE Inhibitor; Drug · Other
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: Up to 17 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Hemophilia A
Interventions: BAY94-9027; Biological
Lead sponsor: Bayer; Industry
Eligibility: Up to 12 Years · Male only
Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 7
States / cities: Sacramento, California • Pensacola, Florida • Cincinnati, Ohio + 4 more

Conditions: Sickle Cell Disease
Interventions: GBT440; Drug
Lead sponsor: Global Blood Therapeutics; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: San Antonio, Texas

Search by objective public record fields.

Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)

Rainbow Extension Study

Embryo Health Study

Efficacy and Safety Study of CD2027 Ointment 3 Microgram Per Gram (mcg/g) Twice Daily Treatment for Adults With at Least Moderate Atopic Dermatitis

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

Diabetes Therapy to Improve BMI and Lung Function in CF

A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating

Long-Term Follow-up Study of ADVM-043

Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

A Study to Compare the Relative Bioavailability of Two Iberdomide (CC-220) Formulations and to Assess The Effect Of Food on the Drug Levels of Iberdomide in Healthy Adult Participants

Eltrombopag Olamine in Improving Platelet Recovery in Older Patients With Acute Myeloid Leukemia Undergoing Chemotherapy

The Influence of 3D Printed Prostheses on Neural Activation Patterns

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Improving Outcomes in Cleft Palate Surgery

Study of Beet Juice for Patients With Sickle Cell Anemia

Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

Obesity in Pediatric Sickle Cell Disease: A New Phenomenon

Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).

Building Sentences With Preschoolers Who Use AAC

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

Angiotensin Converting Enzyme Inhibitors During Cardiopulmonary Bypass in Infants and Children

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects