- Conditions
- Fabry Disease
- Interventions
- Replagal (agalsidase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
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 1225–1248
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
- Interventions
- zagociguat 15mg
- Drug
- Lead sponsor
- Tisento Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 9
- States / cities
- La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
- Conditions
- Gout, Hyperuricemia, Arthritis, Joint Disease
- Interventions
- ulodesine, Placebo
- Drug
- Lead sponsor
- BioCryst Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 69 Years
- Enrollment
- 279 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 54
- States / cities
- Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 51 more
- Conditions
- Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
- Interventions
- Phenoptin
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Chicago, Illinois • Minneapolis, Minnesota + 6 more
- Conditions
- Fabry Disease
- Interventions
- Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
- Interventions
- Vatiquinone, Placebo
- Drug · Other
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Up to 20 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 12
- States / cities
- San Diego, California • Stanford, California • New Haven, Connecticut + 7 more
- Conditions
- Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
- Interventions
- Antioxidants, Cholesterol
- Drug
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- Up to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2025
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- MPS IVA, MPS VI
- Interventions
- Vosoritide Injection [Voxzogo]
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 5 Years to 10 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Oakland, California
- 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
- Lactose Intolerance
- Interventions
- RP-G28, Placebo
- Drug · Other
- Lead sponsor
- Ritter Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 377 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016
- U.S. locations
- 1
- States / cities
- Princeton, New Jersey
- Conditions
- Amyloidosis Transthyretin, Athlete Heart
- Interventions
- Not listed
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Timeline
- 2021
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Fabry Disease
- Interventions
- Replagal agalsidase alfa, Replagal
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 39 Years to 45 Years · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Shwachman-Diamond Syndrome
- Interventions
- Oral Glucose Tolerance Test, Modified Oral Glucose Tolerance Test, Modified Mixed Meal Tolerance Test, Continuous Glucose Monitor, Food Diary, Medical History Questionnaires
- Diagnostic Test · Other · Device
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 3 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Mucopolysaccharidosis II
- Interventions
- lymphocyte gene therapy
- Genetic
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1996
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Indolent Systemic Mastocytosis
- Interventions
- Masitinib, Placebo, Best Supportive Care
- Drug · Other
- Lead sponsor
- AB Science
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 2
- States / cities
- Sacramento, California • Houston, Texas
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Implitapide
- Drug
- Lead sponsor
- Medical Research Laboratories International
- Industry
- Eligibility
- 8 Years to 70 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Phenylketonuria
- Interventions
- SYNB1618, SYNB1934
- Drug
- Lead sponsor
- Synlogic
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 11
- States / cities
- Aurora, Colorado • Boca Raton, Florida • Gainesville, Florida + 7 more
- Conditions
- Neimann-Pick Disease
- Interventions
- Vorinostat
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
- Interventions
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
- Drug · Radiation · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 50 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome
- Interventions
- Stem Cell Transplant, Laronidase ERT
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 7 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2012
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Glycogen Storage Disease Type II, Glycogenosis 2
- Interventions
- Myozyme
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2006
- U.S. locations
- 8
- States / cities
- Aurora, Colorado • Wichita, Kansas • Cambridge, Massachusetts + 5 more
- Conditions
- Leigh Syndrome
- Interventions
- Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
- Interventions
- SPK-3006
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2032
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more
- Conditions
- Hypertriglyceridemia
- Interventions
- Omega-3-Acid Ethyl Esters
- Drug
- Lead sponsor
- Jewish Hospital, Cincinnati, Ohio
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio