- Conditions
- Late-onset Pompe Disease
- Interventions
- DNL952
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 4
- States / cities
- Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 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 25–48
of 1,597
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl-L-Leucine, Placebo
- Drug · Other
- Lead sponsor
- IntraBio Inc
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- Cholic Acid
- Drug
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • Omaha, Nebraska
- Conditions
- X-linked Adrenoleukodystrophy
- Interventions
- vitamin D3
- Dietary Supplement
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Months to 25 Years · Male only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Soft Tissue Injuries, Gouty Arthritis, Fractures, Ureteral Colic, Dysmenorrhea
- Interventions
- Not listed
- Lead sponsor
- Radnor Registry Research, Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 843 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • New Orleans, Louisiana • Jackson, Mississippi + 5 more
- Conditions
- Glycogen Storage Disease
- Interventions
- Diabetes Sentry
- Device
- Lead sponsor
- University of Florida
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Congenital Disorders of Glycosylation
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 80 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
- Interventions
- Tauroursodeoxycholic Acid and Doxycycline
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Hutchinson-Gilford Progeria Syndrome, Werner Syndrome
- Interventions
- Progerinin, Placebo
- Drug
- Lead sponsor
- PRG Science & Technology Co., Ltd.
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 64 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Glendale, California
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Interventions
- ABO-101
- Drug
- Lead sponsor
- Arbor Biotechnologies
- Industry
- Eligibility
- 6 Years to 64 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2043
- U.S. locations
- 2
- States / cities
- Rochester, Minnesota • Saint Paul, Minnesota
- Conditions
- Gaucher Disease Type 1, Gaucher Disease Type 3
- Interventions
- Velaglucerase alfa
- Drug
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 4 Years to 14 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 338 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Interventions
- Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 3
- States / cities
- Stanford, California • New York, New York • Pittsburgh, Pennsylvania
- Conditions
- Gout Chronic, Gout, DDI (Drug-Drug Interaction)
- Interventions
- ABP-671 plus allopurinol
- Drug
- Lead sponsor
- Atom Therapeutics Co., Ltd
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- Cary, North Carolina
- 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
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2004
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 more
- Conditions
- Fabry Disease
- Interventions
- Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
- Diagnostic Test
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 8 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 26
- States / cities
- Anchorage, Alaska • Phoenix, Arizona • Beverly Hills, California + 23 more
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Myozyme® (alglucosidase alfa)
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 1 Month and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2020
- U.S. locations
- 4
- States / cities
- Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
- Conditions
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- Interventions
- NTLA-2001, Placebo
- Biological · Drug
- Lead sponsor
- Intellia Therapeutics
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 1,200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 38
- States / cities
- Tucson, Arizona • Los Angeles, California • Palo Alto, California + 29 more
- Conditions
- Hyperuricemia, Gout
- Interventions
- Arhalofenate, Febuxostat, Colchicine
- Drug
- Lead sponsor
- Gilead Sciences
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
- Conditions
- Gout
- Interventions
- lesinurad 400 mg, naproxen 250 mg, indomethacin 25 mg
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013
- U.S. locations
- 1
- States / cities
- Kalamazoo, Michigan
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease
- Interventions
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 5
- States / cities
- San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
- Conditions
- Progeria
- Interventions
- Lonafarnib, Zoledronic Acid, and Pravastatin
- Drug
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2023
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts