- 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
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 265–288
of 2,421
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- Palm Harbor, Florida
- Conditions
- Chronic Kidney Disease
- Interventions
- grain (soy) protein diet, casein (meat) protein diet
- Other
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- X-linked Adrenoleukodystrophy
- Interventions
- Lorenzo's Oil
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Months to 18 Years · Male only
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Hyperinsulinemia Hypoglycemia
- Interventions
- Exendin (9-39)
- Drug
- Lead sponsor
- Tracey McLaughlin
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Congenital Hyperinsulinism
- Interventions
- Exendin (9-39), Vehicle
- Drug
- Lead sponsor
- Diva De Leon
- Other
- Eligibility
- Up to 12 Months
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Pompe Disease
- Interventions
- Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
- Biological · Drug · Other
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2034
- U.S. locations
- 15
- States / cities
- Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
- Conditions
- Pancreatic Cancer
- Interventions
- Synthetic Human Secretin
- Drug
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 35 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2012
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- 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
- Achondroplasia
- Interventions
- Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 17 Years
- Enrollment
- 114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 7
- States / cities
- San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
- Conditions
- Intercritical Gout
- Interventions
- Placebo, Rilonacept 80 mg, Rilonacept 160 mg
- Other · Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 241 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 67
- States / cities
- Birmingham, Alabama • Gilbert, Arizona • Peoria, Arizona + 61 more
- Conditions
- Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- alglucosidase alfa, Placebo
- Biological · Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 5
- States / cities
- Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
- Conditions
- Pompe Disease
- Interventions
- Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test
- Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 20 Years to 65 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Prader-Willi Syndrome
- Interventions
- DCCR, Placebo
- Drug
- Lead sponsor
- Essentialis, Inc.
- Industry
- Eligibility
- 10 Years to 22 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- Orange, California
- Conditions
- Gout, Hyperuricemia
- Interventions
- LC350189 formulated capsule, Placebo
- Drug
- Lead sponsor
- LG Chem
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 156 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 34
- States / cities
- Birmingham, Alabama • Chandler, Arizona • Mesa, Arizona + 28 more
- Conditions
- Generalized Lipodystrophy
- Interventions
- mibavademab
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Ann Arbor, Michigan
- 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
- Conditions
- Vitamin D Deficiency, Cystic Fibrosis
- Interventions
- Vitamin D3
- Dietary Supplement
- Lead sponsor
- University of Southern California
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
- Interventions
- Aldurazyme (laronidase)
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years to 50 Years · Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Lipodystrophy, HIV Infection
- Interventions
- Metformin, Pioglitazone
- Drug
- Lead sponsor
- Tufts Medical Center
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Lesch-Nyhan Disease
- Interventions
- Ecopipam
- Drug
- Lead sponsor
- Psyadon Pharma
- Industry
- Eligibility
- 6 Years to 65 Years · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 2
- States / cities
- San Diego, California • Atlanta, Georgia
- Conditions
- Lynch Syndrome
- Interventions
- Atorvastatin 20mg, Atorvastatin 20mg AND Aspirin 325 mg
- Drug
- Lead sponsor
- Fox Chase Cancer Center
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- AI Toolkit for ATTR-CM Diagnosis
- Diagnostic Test
- Lead sponsor
- Yale University
- Other
- Eligibility
- 50 Years to 95 Years
- Enrollment
- 1,500,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 12
- States / cities
- San Francisco, California • New Haven, Connecticut • Chicago, Illinois + 9 more
- Conditions
- Endometrial Adenocarcinoma, Endometrial Carcinoma, Lynch Syndrome, Relatives
- Interventions
- Genetic Counseling, Genetic Testing, Laboratory Biomarker Analysis, Mutation Carrier Screening
- Other · Procedure
- Lead sponsor
- Ohio State University Comprehensive Cancer Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,001 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2025
- U.S. locations
- 9
- States / cities
- Akron, Ohio • Canton, Ohio • Cincinnati, Ohio + 3 more