- Conditions
- Gaucher Disease, Type 1
- Interventions
- velaglucerase alfa, imiglucerase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- Durham, 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 1033–1056
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- MGTA-456
- Drug
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Cincinnati, Ohio
- Conditions
- Batten Disease, CLN6
- Interventions
- Natural history
- Other
- Lead sponsor
- Emily de los Reyes
- Other
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Oculocutaneous Albinism
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Year to 80 Years
- Enrollment
- 206 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Multiple Acyl-CoA Dehydrogenase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • New York, New York
- Conditions
- Gaucher Disease
- Interventions
- no intervention
- Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 30 participants
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Cardiac Amyloidosis
- Interventions
- EchoNet-LVH screening for cardiac amyloidosis
- Other
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy
- Interventions
- Lorenzo's oil
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 240 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Phenylketonurias, PKU
- Interventions
- Not listed
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Orange, California • Tampa, Florida + 5 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl Cysteine
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 12 Months and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Gout
- Interventions
- Probenecid, Allopurinol
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 294,878 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
- Interventions
- Not listed
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2033
- U.S. locations
- 13
- States / cities
- La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2033
- U.S. locations
- 12
- States / cities
- Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 9 more
- Conditions
- Gout, Hyperuricemia
- Interventions
- Tigulixostat, Theophylline
- Drug
- Lead sponsor
- LG Chem
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Propionic Acidemia
- Interventions
- Not listed
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- 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
- Autism, Asperger Disorder, PDD-NOS
- Interventions
- Cholesterol
- Other
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Sevirumab
- Drug
- Lead sponsor
- Sandoz
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Galveston, Texas
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
- Interventions
- triheptanoin
- Drug
- Lead sponsor
- Jerry Vockley, MD, PhD
- Other
- Eligibility
- 1 Month and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Pompe Disease
- Interventions
- Alglucosidase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 113 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 51
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more
- Conditions
- Hyperuricemia, Gout
- Interventions
- Allopurinol
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Phenylketonurias
- Interventions
- sapropterin dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 14
- States / cities
- Los Angeles, California • Oakland, California • Sacramento, California + 11 more
- Conditions
- Acute Gout
- Interventions
- apremilast, indomethacin SR
- Drug
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Other
- Eligibility
- 18 Years and older · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010
- U.S. locations
- 1
- States / cities
- Lebanon, New Hampshire
- Conditions
- Fabry Disease
- Interventions
- This is a non-interventional study
- Other
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Eagan, Minnesota