- 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
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 385–408
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Classic Galactosemia
- Interventions
- AT-007
- Drug
- Lead sponsor
- Applied Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Berlin, New Jersey
- Conditions
- Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
- Interventions
- 15 O Water
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 123 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1994 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Neuronal Ceroid Lipofuscinosis, Batten's Disease
- Interventions
- Not listed
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
- Interventions
- Neutralizing Antibody to SPK-3006 capsid
- Diagnostic Test
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more
- Conditions
- Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Acid Lipase Deficiency, LIPA Deficiency, LAL-Deficiency
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2029
- U.S. locations
- 19
- States / cities
- Phoenix, Arizona • Stanford, California • Miramar, Florida + 14 more
- Conditions
- Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2018
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Gaucher's Disease
- Interventions
- Eliglustat
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2021
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • Decatur, Georgia • Detroit, Michigan
- Conditions
- Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
- Interventions
- Vamifeport, Placebo
- Drug
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 20
- States / cities
- Gilbert, Arizona • San Diego, California • Jacksonville, Florida + 16 more
- Conditions
- Generalised Lipodystrophy, Partial Lipodystrophy
- Interventions
- Metreleptin
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2031
- U.S. locations
- 16
- States / cities
- Birmingham, Alabama • Duarte, California • Atlanta, Georgia + 11 more
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Interventions
- Natural History Observation
- Other
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 4
- States / cities
- Stanford, California • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Homocystinuria Due to CBS Deficiency
- Interventions
- Not listed
- Lead sponsor
- Travere Therapeutics, Inc.
- Industry
- Eligibility
- 1 Year to 65 Years
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 7
- States / cities
- Aurora, Colorado • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Lu AG13909
- Drug
- Lead sponsor
- H. Lundbeck A/S
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
- Interventions
- Evuzamitide
- Drug
- Lead sponsor
- Lorena Saelices
- Other
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • New York, New York • Dallas, Texas
- Conditions
- Glycogen Storage Disease Type I
- Interventions
- Triheptanoin
- Drug
- Lead sponsor
- Areeg El-Gharbawy
- Other
- Eligibility
- 1 Month to 65 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Hypercholesterolemia, Familial
- Interventions
- Torcetrapib/atorvastatin
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
- Interventions
- Inotersen, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 82 Years
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 9
- States / cities
- Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
- Conditions
- PolG, Primary Mitochondrial Myopathy
- Interventions
- Not listed
- Lead sponsor
- The Emmes Company, LLC
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Kingston, Massachusetts
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
- Interventions
- GZ402666
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- evinacumab
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 116 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Boca Raton, Florida • Boston, Massachusetts + 5 more
- Conditions
- Wilson Disease
- Interventions
- zinc acetate
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1993
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis
- Interventions
- Mycophenolate mofetil, Liquid Placebo
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 6 Years to 25 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Congenital Disorder of Glycosylation
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota