- Conditions
- Tay Sachs Disease, Sandhoff Disease, Late Onset Tay Sachs Disease
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 2
- States / cities
- Tampa, Florida • Minneapolis, Minnesota
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 121–144
of 595
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
- Fabry Disease
- Interventions
- agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2005
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
- Conditions
- Mucopolysaccharidosis II
- Interventions
- iduronate-2-sulfatase enzyme replacement therapy, Placebo
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years to 25 Years · Male only
- Enrollment
- 96 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 4
- States / cities
- Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
- Conditions
- Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
- Interventions
- Pirfenidone, Placebo
- Drug
- Lead sponsor
- William Gahl, M.D.
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
- Interventions
- Venipuncture
- Procedure
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2024
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Mucopolysaccharidoses, Carotid Disease, Cardiac Disease, Inflammation
- Interventions
- Carotid ultrasonography, Echocardiography, transthoracic, Venipuncture
- Diagnostic Test · Procedure
- Lead sponsor
- Children's Hospital of Orange County
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 1
- States / cities
- Orange, California
- Conditions
- Gas, Bloating, Distension, Constipation, Diarrhea
- Interventions
- APP Diary, Paper Diary
- Other
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- Augusta, Georgia
- 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
- Age-related Macular Degeneration
- Interventions
- Ranibizumab 0.5mg
- Drug
- Lead sponsor
- Southern California Desert Retina Consultants, MC
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 108 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 4
- States / cities
- Mountain View, California • Palm Desert, California • Baltimore, Maryland + 1 more
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2024
- U.S. locations
- 7
- States / cities
- Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
- Conditions
- Gaucher's Disease
- Interventions
- CEREDASE™
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Gaucher Disease Type 1
- Interventions
- FLT201
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 16
- States / cities
- Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
- Interventions
- rivogenlecleucel, rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 3 Months to 21 Years
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Palo Alto, California
- Conditions
- Mucolipidosis Type IV
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Timeline
- 2023 – 2028
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Fabry Disease
- Interventions
- Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Interventions
- agalsidase beta
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2024
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II
- Interventions
- Observation
- Other
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 4
- States / cities
- Gainesville, Florida • St Louis, Missouri • Lake Success, New York + 1 more
- Conditions
- Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
- Interventions
- Observational study
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 73 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Transfusional Iron Overload
- Interventions
- ICL670
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2003
- U.S. locations
- 8
- States / cities
- Oakland, California • Orange, California • Boston, Massachusetts + 4 more
- Conditions
- Cystinosis
- Interventions
- Cystagon®, RP103
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- Not listed
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Hunter Syndrome
- Interventions
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
- Other · Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota