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Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax, VA

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Miglustat
Venglustat
GZ402671
GZ419828
Montelukast
Diphenhydramine
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Acetaminophen
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Active trials

7 of 8 total trials

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (SHORTEN)

This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced in...

Enrolling
Fabry's Disease
Drug: AGALSIDASE BETA (GZ419828)
Drug: Acetaminophen

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe...

Enrolling
Fabry Disease
Drug: migalastat HCl 150 mg

This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat ve...

Enrolling
Fabry Disease
Drug: Venglustat (GZ402671)
Drug: Agalsidase alfa

This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropath...

Enrolling
Fabry Disease
Drug: Placebo
Drug: Venglustat (GZ402671)

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study e...

Active, not recruiting
Fabry Disease
Drug: Lucerastat

This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to ev...

Active, not recruiting
Fabry Disease
Biological: ST-920

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treat...

Enrolling
Pompe Disease (Late-onset)
Biological: Cipaglucosidase Alfa
Drug: Miglustat

Trial sponsors

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