- Conditions
- Fabry Disease
- Interventions
- Fluorescein angiography
- Other
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
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 73–96
of 594
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Parts A/B: Adrabetadex, Parts A/B: Sham Control
- Drug · Other
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 11 more
- 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
- 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
- Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
- Interventions
- enzyme analysis
- Other
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Danon Disease
- Interventions
- RP-A501
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older · Male only
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 3
- States / cities
- La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
- Conditions
- Fabry Disease
- Interventions
- Lucerastat, Placebo
- Drug
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 118 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 17
- States / cities
- Birmingham, Alabama • Irvine, California • Oakland, California + 14 more
- Conditions
- Lysosomal Storage Diseases, Inborn Errors of Metabolism
- Interventions
- ALD-601
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 18 Years and older · Female only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Durham, North Carolina
- Conditions
- Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome
- Interventions
- Laronidase
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2018
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- G6PD Deficiency
- Interventions
- Sodium Chromate Cr51
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years to 70 Years · Male only
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2017
- U.S. locations
- 2
- States / cities
- New York, New York
- 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 Disease
- Interventions
- eliglustat
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015
- U.S. locations
- 1
- States / cities
- Evansville, Indiana
- Conditions
- Gaucher Disease
- Interventions
- Investigational procedure
- Procedure
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- Not listed
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • New York, New York • Milwaukee, Wisconsin
- Conditions
- Sanfilippo Syndrome Type A
- Interventions
- GC1130A
- Drug
- Lead sponsor
- GC Biopharma Corp
- Industry
- Eligibility
- 12 Months to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 2
- States / cities
- Oakland, California • Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis I
- Interventions
- AGT-181 (HIRMAb-IDUA)
- Drug
- Lead sponsor
- ArmaGen, Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 6
- States / cities
- Oakland, California • Orange, California • Decatur, Georgia + 3 more
- Conditions
- Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
- Interventions
- Olipudase alfa
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- Up to 2 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Chicago, Illinois • Cincinnati, Ohio • Columbus, Ohio + 3 more
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl 150 mg
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 7
- States / cities
- Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 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
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 2
- States / cities
- Rockville, Maryland • Fairfax, Virginia
- Conditions
- Enchondromatosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 2 Years to 100 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Sanfilippo Syndrome Type C
- Interventions
- Not listed
- Lead sponsor
- Phoenix Nest
- Industry
- Eligibility
- 12 Months and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- 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
- Conditions
- Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
- Interventions
- There are no interventions in this study.
- Other
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- Not listed
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Sanfilippo Syndrome Type C, Mucopolysaccharidosis (MPS) IIIC
- Interventions
- Not listed
- Lead sponsor
- Phoenix Nest
- Industry
- Eligibility
- 1 Year to 25 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Dallas, Texas