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treatment for Fabry Disease

Phase 1 & 2
Waitlist Available
Led By Michael West, MD
Research Sponsored by Resverlogix Corp
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16 weeks
Awards & highlights
No Placebo-Only Group

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.

Eligible Conditions
  • Fabry Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~16 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 16 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Adverse events
Changes in clinical laboratory parameters
Secondary study objectives
Change in Alkaline Phosphatase
Changes in key markers of Chronic Kidney Disease-Bone Mineral Disease (CKD-BMD)
Changes in key markers of inflammation
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: treatmentExperimental Treatment1 Intervention
RVX000222 (apabetalone) 100 mg to be administered orally BID 12 hours apart.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Apabetalone
Not yet FDA approved

Find a Location

Who is running the clinical trial?

Resverlogix CorpLead Sponsor
12 Previous Clinical Trials
3,653 Total Patients Enrolled
Michael West, MDPrincipal InvestigatorQueen Elizabeth II Health Sciences Centre, Victoria General Site
1 Previous Clinical Trials
167 Total Patients Enrolled
~0 spots leftby Nov 2025
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