Vx-147 For Adults With Apol1-Mediated Proteinuric Kidney Disease
Posted Date: Jun 13, 2024
- Investigator: Prakash Gudsoorkar
- Specialties: Kidney Disease, Nephrology
- Type of Study: Drug
Apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease (AMKD) is a type of chronic kidney disease (CKD) caused by mutations in the APOL1 gene. AMKD occurs in people who have two copies of either APOL1 risk variants, G1 and G2. People who inherit two mutations of the APOL1 gene have a substantially more aggressive form of CKD compared to those who do not inherit two mutations. The APOL1 risk variants cause kidney disease by injuring the cells of the kidney. This disrupts the kidneys’ ability to filter blood, which can lead to protein in the urine (termed “proteinuria”) and an overall decline in kidney function. VX-147 is a small molecule inhibitor of APOL1 that blocks the APOL1-mediated toxic effects on the cells of the kidney, which may decrease proteinuria and lead to improved kidney function. VX-147 is the first investigational drug aimed at treating the underlying cause of AMKD. This study will evaluate the efficacy, safety, and tolerability of oral VX-147 in adults with APOL1-mediated kidney disease. In this study patients will be randomized to receive either VX-147 or placebo tablets taken orally every day. The study will last 2 to 4 years. Study visits will be more frequent in the beginning but then every 4-8 weeks.
Criteria:
Patients Aged 18 To 65 Years, With Two Apol1 Mutations (G1/G1, G2/G2, Or G1/G2), Urine Protein To Creatinine Ratio (Upcr) =0.7 G/G To <10 G/G, Egfr =25 To <75 Ml/Min/1.73M2 And On Stable Doses Of Standard Of Care Medications Are Eligible To Enroll. Subjects With Diabetes Mellitus, Uncontrolled Hypertension, Or A History Of Organ Or Bone Marrow Transplant Are Ineligible For This Study. Men And Women Must Avoid Pregnancy And Use Contraceptive Methods The Entire Period Of The Study. Other Criteria Apply.
Keywords:
Kidney, Apol1, Protein
For More Information:
Leksi Travitz
7174487597
travitli@ucmail.uc.edu