- Conditions
- Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- Not listed
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1978
- U.S. locations
- 1
- States / cities
- San Diego, California
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 1249–1272
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Pompe Disease
- Interventions
- BMN 701
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 4
- States / cities
- San Diego, California • Gainesville, Florida • Tampa, Florida + 1 more
- Conditions
- Phenylketonurias
- Interventions
- Pegvaliase-Pqpz
- Drug
- Lead sponsor
- University of Missouri-Columbia
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Columbia, Missouri
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 17
- States / cities
- Gainesville, Florida • Tampa, Florida • Chicago, Illinois + 14 more
- Conditions
- Fabry's Disease
- Interventions
- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 2 Years to 65 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 5
- States / cities
- Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
- Conditions
- Gout
- Interventions
- D-0120, Allopurinol
- Drug
- Lead sponsor
- InventisBio Co., Ltd
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 19
- States / cities
- Birmingham, Alabama • Plantation, Florida • Evergreen Park, Illinois + 16 more
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
- Interventions
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Fed
- Interventions
- Desloratadine
- Drug
- Lead sponsor
- Dr. Reddy's Laboratories Limited
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 28 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2006
- U.S. locations
- 1
- States / cities
- Fort Myers, Florida
- Conditions
- Healthy Volunteers, Fasting, Fed
- Interventions
- Apixaban 5 mg Oral Dissolving Film (TAH3311) and Tablet (Eliquis®)
- Drug
- Lead sponsor
- TAHO Pharmaceuticals Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Las Vegas, Nevada
- Conditions
- Gaucher's Disease Type III
- Interventions
- Venglustat, imiglucerase
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 4
- States / cities
- New Haven, Connecticut • Iowa City, Iowa • Dallas, Texas + 1 more
- Conditions
- Gout
- Interventions
- DYV700, Placebo, Colchicine 0.6 mg
- Drug
- Lead sponsor
- Dyve Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 284 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Poway, California
- Conditions
- Gouty Arthritis
- Interventions
- Canakinumab
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 341 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Primary Hyperoxaluria Type 3
- Interventions
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Rochester, Minnesota • New York, New York
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 100 Years
- Enrollment
- 999 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2099
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Interventions
- Aldurazyme, placebo
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2005
- U.S. locations
- 8
- States / cities
- Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- Interventions
- RGX-111
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 4 Months and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2029
- U.S. locations
- 1
- States / cities
- Orange, California
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Fomivirsen sodium, Ganciclovir
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 194 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 18
- States / cities
- Los Angeles, California • Pasadena, California • San Diego, California + 14 more
- Conditions
- Blood Pressure, Gout
- Interventions
- febuxostat
- Drug
- Lead sponsor
- University of Mississippi Medical Center
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Jackson, Mississippi
- Conditions
- Wilson Disease
- Interventions
- ALXN1840, SoC Therapy
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 214 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 6
- States / cities
- Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more
- Conditions
- Bowel Incontinence
- Interventions
- Renew Insert
- Device
- Lead sponsor
- Renew Medical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 97 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 3
- States / cities
- Los Gatos, California • San Diego, California • Weston, Florida
- Conditions
- Glycogen Storage Disease Type II
- Interventions
- Alglucosidase alfa GZ419829
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 0 Days to 6 Months
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 5
- States / cities
- Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more
- Conditions
- Non-Neoplastic Hematologic and Lymphocytic Disorder
- Interventions
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
- Procedure · Biological · Drug + 2 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Up to 49 Years
- Enrollment
- 98 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 9,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2034
- U.S. locations
- 91
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 70 more
- Conditions
- Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
- Interventions
- Ammonia Study Device
- Diagnostic Test
- Lead sponsor
- Sequitur Health Corp.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Portland, Oregon