- Conditions
- Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
- Interventions
- Enzyme testing
- Other
- Lead sponsor
- Children's Hospitals and Clinics of Minnesota
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Saint Paul, Minnesota
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 409–432
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Barth Syndrome
- Interventions
- Elamipretide, Placebo
- Drug
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Industry
- Eligibility
- 12 Years and older · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2026
- U.S. locations
- 16
- States / cities
- San Diego, California • Stanford, California • Aurora, Colorado + 12 more
- Conditions
- Cancers
- Interventions
- Laparoscopic surgery
- Procedure
- Lead sponsor
- Erin Rowell
- Other
- Eligibility
- Up to 11 Years · Female only
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2035
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 21
- States / cities
- Long Beach, California • Los Angeles, California • Stanford, California + 18 more
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- GZ402665
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
- Interventions
- Hematopoietic stem cell transplantation
- Procedure
- Lead sponsor
- Children's Hospital Los Angeles
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Gaucher's Disease
- Interventions
- Eliglustat
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- Evansville, Indiana
- Conditions
- Systemic Lupus Erythematosus, Sjogren's Syndrome, Inflammatory Myositis, Psoriatic Arthritis, Gout, Ankylosing Spondylitis, Arthritis of Multiple Sites Associated With Inflammatory Bowel Disease (Diagnosis), Osteoarthritis
- Interventions
- mRNA COVD19 vaccine
- Biological
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
- Interventions
- Not listed
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- 1 Day to 120 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2054
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
- Interventions
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 13
- States / cities
- La Mesa, California • Orange, California • Denver, Colorado + 9 more
- Conditions
- Familial Chylomicronemia, High Risk Severe Hypertriglyceridemia (SHTG)
- Interventions
- Plozasiran
- Drug
- Lead sponsor
- Arrowhead Pharmaceuticals
- Industry
- Eligibility
- 15 Years and older
- U.S. locations
- 1
- States / cities
- Pasadena, California
- Conditions
- Gout
- Interventions
- DECT scan
- Procedure
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 223 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 9
- States / cities
- Irvine, California • Englewood, Colorado • Wheat Ridge, Colorado + 6 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 3 Months and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Foscarnet sodium
- Drug
- Lead sponsor
- Astra USA
- Industry
- Eligibility
- 18 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- virtual educational based transition intervention self-paced
- Other
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 16 Years to 22 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- Cholesterol supplementation
- Dietary Supplement
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- Up to 85 Years
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 5
- States / cities
- Bethesda, Maryland • Omaha, Nebraska • Cincinnati, Ohio + 2 more
- Conditions
- Mucopolysaccharidosis II, MPS II
- Interventions
- SB-913
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 5 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 5
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Dallas, Texas
- Conditions
- Urea Cycle Disorders
- Interventions
- Not listed
- Lead sponsor
- Mark Batshaw
- Other
- Eligibility
- Not listed
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more
- Conditions
- Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
- Interventions
- Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 1 Month to 25 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- X-linked Adrenoleukodystrophy
- Interventions
- vitamin D3
- Dietary Supplement
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Months to 25 Years · Male only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST)
- Interventions
- Imatinib mesylate
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 148 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2013
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Portland, Oregon • Philadelphia, Pennsylvania