- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Minute to 5 Years
- Enrollment
- 69 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2016
- U.S. locations
- 3
- States / cities
- Oakland, California • Cincinnati, Ohio • Pittsburgh, Pennsylvania
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 481–504
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- arimoclomol, Placebo
- Drug
- Lead sponsor
- ZevraDenmark
- Industry
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2024
- U.S. locations
- 2
- States / cities
- Oakland, California • Rochester, Minnesota
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- Not listed
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 30 Years to 80 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
- Interventions
- Melphalan
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2021
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
- Interventions
- Not listed
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Industry
- Eligibility
- 0 Months and older
- Enrollment
- 226 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia
- Conditions
- Disaster, Vulnerable Populations
- Interventions
- Nurses Taking On Readiness Measures (N-TORM)
- Behavioral
- Lead sponsor
- Hunter College of City University of New York
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 31 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Porphyria Cutanea Tarda
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2000
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Crinecerfont
- Drug
- Lead sponsor
- Neurocrine Switzerland GmbH
- Industry
- Eligibility
- 3 Months to 47 Months
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 11
- States / cities
- San Francisco, California • Aurora, Colorado • Hartford, Connecticut + 8 more
- Conditions
- Phenylketonurias, PAH Deficiency
- Interventions
- HMI-102
- Genetic
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 13
- States / cities
- Los Angeles, California • Orange, California • Tampa, Florida + 10 more
- Conditions
- Fabry Disease
- Interventions
- This is a non-interventional study
- Other
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Eagan, Minnesota
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Valganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- X-Linked Hypophosphatemia
- Interventions
- Burosumab
- Biological
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- 1 Year to 4 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 3
- States / cities
- New Haven, Connecticut • Indianapolis, Indiana • St Louis, Missouri
- Conditions
- Hypophosphatasia
- Interventions
- ALXN1850, Placebo
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 3
- States / cities
- Baltimore, Maryland • Kansas City, Missouri • Durham, North Carolina
- Conditions
- Jaundice, Neonatal, Hyperbilirubinemia, Neonatal
- Interventions
- Phototherapy, Stannsoporfin, Placebo
- Procedure · Drug
- Lead sponsor
- InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company
- Industry
- Eligibility
- 1 Hour to 72 Hours
- Enrollment
- 91 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 18
- States / cities
- Colton, California • San Diego, California • San Francisco, California + 14 more
- Conditions
- Gaucher Disease, Type 3
- Interventions
- No intervention
- Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 5 participants
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Porphyria
- Interventions
- heme arginate, tin mesoporphyrin
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1997
- U.S. locations
- 2
- States / cities
- New York, New York • Galveston, Texas
- 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
- Gout, Hyperuricemia
- Interventions
- [14C] LC350189
- Drug
- Lead sponsor
- LG Chem
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 12 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 14
- States / cities
- Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more
- Conditions
- MELAS Syndrome
- Interventions
- Idebenone, Placebo
- Drug · Other
- Lead sponsor
- Michio Hirano
- Other
- Eligibility
- 8 Years to 65 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Hyperuricemia, Gout
- Interventions
- Vitamin C
- Dietary Supplement
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 135 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Acute Gouty Arthritis Flares
- Interventions
- ACZ885, Triamcinolone acetonide 40 mg
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 136 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 33
- States / cities
- Anniston, Alabama • Mobile, Alabama • Norwalk, California + 28 more
- Conditions
- Gaucher Disease
- Interventions
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 14
- States / cities
- La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Lu AG13909
- Drug
- Lead sponsor
- H. Lundbeck A/S
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan