Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
Study of VX19-147-101 in Adults of African or Caribbean Descent with FSGS
A Phase 2, Open-Label, Single-arm, 2-part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults with APOL1-mediated Focal Segmental Glomerulosclerosis
Brief Description
This clinical research study is investigating a potential treatment option for adults of African or Caribbean descent with biopsy-confirmed focal segmental glomerulosclerosis (FSGS). Testing for the APOL1 gene is included in this clinical study.
Trial Physician / Study Coordinator
Lauren Warsocki
EmailSite Name
University Hospitals of Leicester NHS Trust - Leicester General Hospital (LGH)
Gwendolen Road, Leicester, Leicestershire, LE5 4PW
Sponsor
Vertex Pharmaceuticals
Study Drug
VX-147
Estimated enrollment
10
Estimated end date
December, 2022
Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
Study of VX19-147-101 in Adults of African or Caribbean Descent with FSGS
A Phase 2, Open-Label, Single-arm, 2-part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults with APOL1-mediated Focal Segmental Glomerulosclerosis
Brief Description
This clinical research study is investigating a potential treatment option for adults of African or Caribbean descent with biopsy-confirmed focal segmental glomerulosclerosis (FSGS). Testing for the APOL1 gene is included in this clinical study.
Trial is for people with
Primary focal segmental glomerulosclerosis (FSGS) diagnosed by kidney biopsy with specific APOL1 gene variations (APOL1 genetic testing is including in this study)
Study Goal
This clinical trial will determine if VX-147 can reduce proteinuria in patients with APOL1-mediated FSGS.
What is involved for the Patient?
Interested individuals will be evaluated during a screening period to determine their eligibility to participate, including genetic testing for the apolipoprotein L1 (APOL1) gene variants. Participation could last approximately 17 weeks followed by an optional off-treatment observation period lasting up to 12 weeks.
If permitted by the research facility and your study doctor, you might be permitted to have some of the study visits at your home with a home health nurse followed by a telemedicine video visit or phone call with the study doctor.
About the drug or intervention
VX-147 is a tablet taken by mouth once daily.