- Conditions
- Gout, Hyperuricemia
- Interventions
- Arhalofenate, Febuxostat, Colchicine
- Drug
- Lead sponsor
- Gilead Sciences
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 1,956
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 6 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 3
- States / cities
- Oakland, California • Torrance, California • Minneapolis, Minnesota
- 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
- Mitochondrial Disorders, Li-Fraumeni Syndrome, Carriers of p53 Mutation
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 82 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2007 – 2021
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Prader-Willi Syndrome
- Interventions
- CSTI-500
- Drug
- Lead sponsor
- ConSynance Therapeutics
- Industry
- Eligibility
- 13 Years to 50 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Interventions
- No Intervention
- Other
- Lead sponsor
- iECURE, Inc.
- Industry
- Eligibility
- 7 Months to 15 Months
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2041
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • New York, New York
- Conditions
- Hypercholesterolemia, Hyperlipidemia, Familial Combined
- Interventions
- colesevelam HCl tablets and fenofibrate tablets, fenofibrate tablets and Welchol placebo tablets
- Drug
- Lead sponsor
- Daiichi Sankyo
- Industry
- Eligibility
- 30 Years to 70 Years
- Enrollment
- 129 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2004
- U.S. locations
- 10
- States / cities
- Longwood, Florida • Ocala, Florida • Winterpark, Florida + 7 more
- Conditions
- Gaucher's Disease
- Interventions
- glucocerebrosidase
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Osteoporosis, Cystic Fibrosis
- Interventions
- alendronate sodium, calcium carbonate, cholecalciferol
- Drug
- Lead sponsor
- University of North Carolina
- Other
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2002
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Interventions
- PANCRECARB® (pancrelipase), Placebo
- Drug
- Lead sponsor
- Digestive Care, Inc.
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Pompe Disease
- Interventions
- ACTUS-101
- Genetic
- Lead sponsor
- AskBio Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Sanfilippo Syndrome Type A
- Interventions
- GC1130A
- Drug
- Lead sponsor
- GC Biopharma Corp
- Industry
- Eligibility
- 12 Months to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Anemia, Sickle Cell, Transfusion Hemosiderosis
- Interventions
- deferasirox
- Drug
- Lead sponsor
- Children's Hospital Los Angeles
- Other
- Eligibility
- 14 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Hypercholesterolemia
- Interventions
- Evolocumab, Low-density Lipoprotein Cholesterol (LDL-C) Apheresis
- Biological · Procedure
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Boca Raton, Florida • Kansas City, Kansas • Grandville, Michigan + 1 more
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- AEGR-733
- Drug
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Philadelphia, Pennsylvania
- Conditions
- Leigh Syndrome
- Interventions
- EPI-743
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2023
- U.S. locations
- 4
- States / cities
- Stanford University, California • Akron, Ohio • Houston, Texas + 1 more
- Conditions
- Hereditary Amyloidosis, Transthyretin-Related
- Interventions
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Diagnostic Test
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 30 Years to 90 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Friedreich's Ataxia
- Interventions
- Interferon γ-1b
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 10 Years to 26 Years
- Enrollment
- 86 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
- Conditions
- Phenylketonuria
- Interventions
- JNT-517 Tablet, Placebo Tablet: BID
- Drug
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 7 more
- Conditions
- Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2027
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Lowe Syndrome
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- Male only
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2009
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Cystic Fibrosis, Anemia, Iron-Deficiency
- Interventions
- Ferrous sulfate 325mg, Placebo
- Drug
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 2
- States / cities
- Portland, Maine • Lebanon, New Hampshire
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- CER-001
- Drug
- Lead sponsor
- Cerenis Therapeutics, SA
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 2
- States / cities
- Hartford, Connecticut • North Massapequa, New York
- 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