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Showing 1–24 of 97 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 1Phase 2 Interventional

A Fabry Disease Gene Therapy Study

NCT04040049
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
12 Years to 17 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
5
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, agalsidase
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
2
States / cities
Bethesda, Maryland • Cambridge, Massachusetts
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Conditions
Fabry Disease
Interventions
4D-310
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
4
States / cities
La Jolla, California • Atlanta, Georgia • Pittsburgh, Pennsylvania + 1 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
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
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
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
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
7 Years to 15 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
1
States / cities
Tucson, Arizona
Conditions
Fabry's Disease
Interventions
Smartpill, Endoscopic Mucosal Resection
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Completed Phase 1 Interventional Accepts healthy volunteers

Migalastat Food Effect Study

NCT01489995
Conditions
Fabry Disease
Interventions
A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011
U.S. locations
1
States / cities
Austin, Texas
Completed Phase 1Phase 2 Interventional Results available

Extension Study of PRX-102 for up to 60 Months

NCT01981720
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa)
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
5
States / cities
Gainesville, Florida • Iowa City, Iowa • Baltimore, Maryland + 2 more
Completed No phase listed Observational Accepts healthy volunteers

Analysis of the Nervous System in Patients With Fabry's Disease

NCT00001491
Conditions
Fabry's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
325 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Fabry Disease
Interventions
Migalastat HCl 20 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
2 Years to 11 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
6
States / cities
Atlanta, Georgia • Minneapolis, Minnesota • Charlotte, North Carolina + 3 more
Completed No phase listed Observational Accepts healthy volunteers

Vasodilation in Patients With Fabry's Disease

NCT00001774
Conditions
Cerebrovascular Accident, Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1997 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Fabry Disease
Interventions
DRX005B
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
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
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
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
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
3
States / cities
Decatur, Georgia • New York, New York • Dallas, Texas