- Conditions
- Niemann-Pick Type C Disease
- Interventions
- Nizubaglustat, Placebo
- Drug
- Lead sponsor
- Azafaros B.V.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 72 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 5
- States / cities
- Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 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 217–240
of 595
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Fabry Disease
- Interventions
- GZ/SAR402671
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 3
- States / cities
- Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 9,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2034
- U.S. locations
- 92
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 72 more
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 1 Day to 90 Years
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
- Conditions
- Neimann-Pick Disease
- Interventions
- Vorinostat
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2021
- U.S. locations
- 1
- States / cities
- Lexington, Massachusetts
- Conditions
- Krabbe Disease
- Interventions
- Not listed
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Pompe Disease, Muscle Weakness
- Interventions
- Resistance Exercise Training
- Other
- Lead sponsor
- University of California, Irvine
- Other
- Eligibility
- 16 Years to 75 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Gaucher Disease, Type 1
- Interventions
- VPRIV®
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 95 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Oakland, California • Atlanta, Georgia + 7 more
- Conditions
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Dallas, Texas
- Conditions
- Gaucher Disease
- Interventions
- eliglustat
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 79 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2017
- U.S. locations
- 3
- States / cities
- Miami, Florida • Saint Paul, Minnesota • Knoxville, Tennessee
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Week to 100 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2050
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 11
- States / cities
- Atlanta, Georgia • Chicago, Illinois • Kansas City, Kansas + 8 more
- Conditions
- Gaucher Disease
- Interventions
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 3
- States / cities
- Coral Springs, Florida • Decatur, Georgia • New York, New York
- Conditions
- Type I Gaucher Disease
- Interventions
- Ambroxol
- Drug
- Lead sponsor
- Exsar Corporation
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Monmouth Junction, New Jersey
- 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
- Hermansky Pudlak Syndrome
- Interventions
- Pulmonary function test, Chest CT, Sample collection
- Other
- Lead sponsor
- Vanderbilt University
- Other
- Eligibility
- 12 Years to 90 Years
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 5
- States / cities
- Maywood, Illinois • Boston, Massachusetts • New York, New York + 2 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
- Hurler Syndrome
- Interventions
- IRT Laronidase
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 6 Months to 3 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2018
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 6 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 3
- States / cities
- Oakland, California • Torrance, California • Minneapolis, Minnesota
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
- Interventions
- Idursulfase
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2008
- U.S. locations
- 16
- States / cities
- Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more
- Conditions
- Lysosomal Storage Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Metachromatic Leukodystrophy
- Interventions
- Warfarin
- Drug
- Lead sponsor
- The Cooper Health System
- Other
- Eligibility
- 1 Year to 10 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Camden, New Jersey