FSGS Treatment with VX-147 – Phase IIa

A 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX‐147 in Adults with APOL1-mediated Focal Segmental Glomerulosclerosis

Protocol Description

In this study, researchers are evaluating the drug VX-147 to see if it is effective in lowering the amount of protein in urine caused by focal segmental glomerulosclerosis (FSGS) in patients with an apolipoprotein L1 (APOL1) gene mutation. FSGS is a rare kidney disease that causes scarring in the parts of the kidneys that filter waste materials from blood, allowing protein to leak into the urine, a condition called proteinuria. If the protein leaks for too long, it can lead to kidney failure.

Study Level: Phase IIa

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting participants, ages 18 and older, who may have APOL1-dependent FSGS.
Males and Females: Ages 18 through 60


Participants will take the study drug orally every morning for 13 weeks. For the first 2 weeks, participants will take a single 15mg tablet, and for the remaining 11 weeks, participants will take 3 tablets. Visits for health checks and blood draws will be done at specified intervals during the dosing period, with a follow up visit 4 weeks after the last dose. For convenience, some visits may be done at the participants’ homes rather than at the study center.
Visits: 10
Duration: 17 weeks

Status: Open to Enrollment

Source(s) of Support

Vertex Pharmaceuticals

Primary Investigator

Michael Moritz, MD

Contact Information

For more information about the study or enrollment, please contact:
Matt Butoryak, RN, CCRC