- Conditions
- Gout
- Interventions
- Canakinumab, Colchicine, Allopurinol, Placebo Matching Canakinumab, Placebo Matching Colchicine
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 432 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 19
- States / cities
- Huntington Beach, California • San Diego, California • Jupiter, Florida + 16 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 577–600
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- Alglucosidase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003
- U.S. locations
- 1
- States / cities
- New Brunswick, New Jersey
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
- Interventions
- Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
- Behavioral
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Phenylketonuria (PKU)
- Interventions
- MZE782, Placebo
- Drug · Other
- Lead sponsor
- Maze Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 7
- States / cities
- Oakland, California • Palo Alto, California • Chicago, Illinois + 4 more
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 74
- States / cities
- Sheffield, Alabama • Anchorage, Alaska • Phoenix, Arizona + 71 more
- Conditions
- Pompe Disease
- Interventions
- rAAV1-CMV-GAA (study agent) Administration, RMST
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2015
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
- Interventions
- acoramidis, Placebo Oral Tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 632 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 40
- States / cities
- Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
- Conditions
- Iron Overload
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Mucopolysaccharidosis IIIB
- Interventions
- SBC-103
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 12 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Pittsburgh, Pennsylvania
- Conditions
- Generalized Lipodystrophy
- Interventions
- Placebo, Low-Dose REGN4461, High-dose REGN4461
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 3
- States / cities
- Bethesda, Maryland • Ann Arbor, Michigan • Dallas, Texas
- Conditions
- Pompe Disease
- Interventions
- Pyridostigmine Bromide
- Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 8 Years to 60 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
- Interventions
- Abatacept
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Classic Galactosemia, Speech Disorders in Children, Language Disorders in Children
- Interventions
- Babble Boot Camp
- Behavioral
- Lead sponsor
- Arizona State University
- Other
- Eligibility
- 2 Months to 54 Months
- Enrollment
- 285 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 1
- States / cities
- Tempe, Arizona
- Conditions
- Familial Hypercholesterolemia
- Interventions
- atorvastatin
- Drug
- Lead sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
- Industry
- Eligibility
- 6 Years to 15 Years
- Enrollment
- 272 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Washington D.C., District of Columbia • Gainesville, Florida + 3 more
- Conditions
- Arthritis, Gout, Fibromyalgia, Rheumatoid Arthritis, Osteoarthritis, Lupus Erythematosus, Systemic
- Interventions
- Phone-Based Walk With Ease
- Behavioral
- Lead sponsor
- University of South Carolina
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 267 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Columbia, South Carolina
- Conditions
- Menkes Disease, Occipital Horn Syndrome, Unexplained Copper Deficiency
- Interventions
- Copper Histidine
- Drug
- Lead sponsor
- Cyprium Therapeutics, Inc.
- Industry
- Eligibility
- Up to 80 Years
- Enrollment
- 93 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
- Interventions
- CLN2 Treatment
- Other
- Lead sponsor
- Jessica Scherr
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin (Kuvan)
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2018
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Apparent Mineralocorticoid Excess (AME)
- Interventions
- Glycyrrhetic Acid, Placebo
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2002 – 2009
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Congenital Adrenal Hyperplasia, Healthy
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 9 Years to 40 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Krabbe Disease
- Interventions
- FBX-101
- Biological
- Lead sponsor
- Forge Biologics, Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Fabry Disease
- Interventions
- ST-920
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 9
- States / cities
- Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
- Interventions
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2024
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Smith-Lemli-Opitz Syndrome, Pregnancy
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- Female only
- Enrollment
- 1,800 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2005
- U.S. locations
- 1
- States / cities
- Scarborough, Maine