- Conditions
- Homocystinuria
- Interventions
- SYNB1353
- Drug
- Lead sponsor
- Synlogic
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 31 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022
- U.S. locations
- 1
- States / cities
- High Point, North Carolina
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 721–744
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hunter Syndrome
- Interventions
- No treatment
- Other
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 70 Years · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more
- Conditions
- Metabolic Syndrome
- Interventions
- ELM Group, ELM Classes, ELM Individual
- Behavioral · Other
- Lead sponsor
- Rush University Medical Center
- Other
- Eligibility
- 18 Years to 72 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2021
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Urea Cycle Disorder
- Interventions
- RAVICTI
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 1 Day to 2 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 16
- States / cities
- Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Hydrocortisone
- Drug
- Lead sponsor
- Neurocrine UK Limited
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 91 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Blood Coagulation Disorders
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Years to 80 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
- Interventions
- zagociguat 15mg, zagociguat 30mg, Placebo
- Drug
- Lead sponsor
- Tisento Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 10
- States / cities
- La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
- Conditions
- Wilson Disease
- Interventions
- Standard of Care Medications
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
- Conditions
- Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3, Neuronal Ceroid Lipofuscinosis CLN5, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN7, Neuronal Ceroid Lipofuscinosis CLN8, Neuronal Ceroid Lipofuscinosis CLN10, Batten Disease
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2035
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Sanfilippo Syndrome (MPS III)
- Interventions
- rAAV9-CMV-hNAGLUop
- Biological
- Lead sponsor
- NeuroGT
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2027 – 2029
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Gaucher's Disease
- Interventions
- Lysodase
- Drug
- Lead sponsor
- National Institute of Mental Health (NIMH)
- NIH
- Eligibility
- Not listed
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Wilson Disease, Wilson's Disease, Wilsons Disease
- Interventions
- PM577a
- Drug
- Lead sponsor
- Prime Medicine, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- New Haven, Connecticut • Chicago, Illinois • San Antonio, Texas
- Conditions
- MPS III B, Mucopolysaccharidosis Type IIIB
- Interventions
- AX 250
- Drug
- Lead sponsor
- Allievex Corporation
- Industry
- Eligibility
- 1 Year to 10 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Pompe Disease
- Interventions
- Clenbuterol, Placebo
- Drug
- Lead sponsor
- Dwight Koeberl, M.D., Ph.D.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- MELAS
- Interventions
- IW-6463 Tablets
- Drug
- Lead sponsor
- Tisento Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 5
- States / cities
- Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
- Conditions
- Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism, Pseudohypoparathyroidism Type Ia, Albright Hereditary Osteodystrophy
- Interventions
- Not listed
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 14 participants
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Fabry Disease
- Interventions
- Iohexol
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 5 Years to 25 Years · Male only
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 5
- States / cities
- La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
- Conditions
- Cystinosis
- Interventions
- HIIT Exercise
- Other
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Long-term Follow-Up
- Other
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 3
- States / cities
- San Francisco, California • New Brunswich, New Jersey • Philadelphia, Pennsylvania
- Conditions
- Gout, Hyperuricemia, Arthritis, Joint Disease, Renal Insufficiency
- Interventions
- Placebo, Ulodesine (BCX4208) 5 mg, Ulodesine (BCX4208) 10 mg
- Drug
- Lead sponsor
- BioCryst Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 69 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 10
- States / cities
- Mobile, Alabama • Peoria, Arizona • Irvine, California + 7 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 13 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Diabetes Mellitus, Sarcoidosis, Dental Caries, Sjorgren's Syndrome, Cystinosis, Head and Neck Neoplasms
- Interventions
- Not listed
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- Not listed
- Enrollment
- 264 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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