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Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease

NCT03228940 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2023-11-18

No results posted yet for this study

Summary

Fabry Disease (FD) is a rare X-linked lysosomal storage disorder (LSD) caused by mutations in the GLA gene which translates into decreased activity or lack of function of the enzyme alpha-galactosidase A (α-GAL A) and accumulation of the enzymes substrate, i.e., Gb3, throughout the body. Cardiovascular and renal complications are among the leading causes of death in FD patients. RVX000222 is a BET inhibitor which modulates the expression of a variety of genes and, due to its effects on pathways downstream of substrate accumulation and reduction of major cardiac events, holds promise as a potential add-on therapy to accompany enzyme replacement therapy (ERT) in FD patients.

Conditions

Interventions

DRUG

RVX000222

oral, BID

Sponsors & Collaborators

  • Resverlogix Corp

    lead INDUSTRY

Principal Investigators

  • Michael West, MD · Queen Elizabeth II Health Sciences Centre, Victoria General Site

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-22
Primary Completion
2022-11-22
Completion
2022-11-22
FDA Drug
Yes

Countries

  • Canada

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03228940 on ClinicalTrials.gov