Currently Enrolling
Interventional

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

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.

Currently Enrolling
Interventional

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

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.

Eligibility Criteria
Patient Population

Primary focal segmental glomerulosclerosis (FSGS) diagnosed by kidney biopsy with specific APOL1 gene variations (APOL1 genetic testing is including in this study)

Age

18 — 26+

History of transplant allowed?

No

History of dialysis allowed?

No

eGFR

30 — > 60

UPCR

1.0 or Less — 3.0 or Above

Permitted medication history

Ace Inhibitors/ARB, None, Other, Rituximab (AKA Rituxan), Prograf (AKA Tacrolimus), Cytoxan (Cyclophosphamide), Cellcept (AKA Mycophenolate), Acthar, Abatacept, Prednisone (Steroids)

Patient should be

Treatment Resistant, Steroid Dependent

About the Drug
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.

Sponsor

Vertex Pharmaceuticals

Estimated End Date

December, 2022

About the Trial
Study Drug

VX-147

Study Goal

This clinical trial will determine if VX-147 can reduce proteinuria in patients with APOL1-mediated FSGS.

About the drug or intervention

VX-147 is a tablet taken by mouth once daily.

Atlanta, GA
Frequently Asked Questions

Nephrotic Syndrome is not a disease itself, but rather a group of signs and symptoms that result from damage in the part of the kidney that filters blood (glomeruli).

Common symptoms include:

  • Foamy urine (called proteinuria) caused by protein “spilling” into the urine
  • Severe swelling in parts of the body, most noticeably around the eyes, hands, feet, and abdomen (called edema)
  • Weight gain due to a buildup of extra fluid
  • Fatigue
  • Loss of appetite
  • Low levels of protein in the blood (hypoalbuminemia)
  • Higher than normal fat and cholesterol levels in the blood (hyperlipidemia)

Nephrotic Syndrome can typically be diagnosed with a urine test.

Nephrotic Syndrome can be “primary” or “secondary” in nature.

Diseases that affect only the kidneys are called primary causes of Nephrotic Syndrome. Doctors often call these diseases “idiopathic,” which means that they arise from an unknown cause. Some of these diseases include:

  • Minimal Change Disease (MCD) – most common in children
  • Focal Segmental Glomerulosclerosis (FSGS)
  • Membranous Nephropathy (MN) – most common in adults
  • IgA Nephropathy (IgAN)

Secondary Nephrotic Syndrome is caused by an underlying, systemic condition like diabetes, lupus, HIV, and others.

The Kidney Health Gateway is a website owned and operated by NephCure Kidney International. The purpose of this website is to help patients with rare forms of primary Nephrotic Syndrome get connected to expert care and cutting-edge treatment options. By answering a few questions about you or your loved one’s condition, we can provide you with a list of clinical trials and/or expert doctors in your area.

If you have additional questions, please visit NephCure.org or email Info@NephCure.org.

 

See other frequently asked questions