VX-147 for Kidney Disease

(AMPLITUDE Trial)

Recruiting in Palo Alto (17 mi)

+269 other locations

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Transplant, Uncontrolled hypertension, Diabetes, others

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing VX-147, a new drug, in adults and children with a genetic form of kidney disease. The drug aims to reduce the harmful effects of a specific protein in the kidneys.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What makes the drug VX-147 unique for treating kidney disease?

VX-147 (Inaxaplin) is unique because it is specifically designed to target the underlying causes of kidney disease, potentially offering a novel approach compared to existing treatments that often focus on managing symptoms rather than addressing the root cause.

^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults and kids with a genetic form of kidney disease linked to APOL1 genes. Participants should have protein in their urine, indicating kidney issues, but can't have diabetes, other known causes of kidney disease like sickle cell, uncontrolled high blood pressure, or a history of organ or bone marrow transplants.

Inclusion Criteria

Have you been told to have levels of protein in your urine (this is referred as proteinuria)?

Have you been diagnosed with chronic kidney disease (CKD) or focal segmental glomerulosclerosis (FSGS) syndrome?

Has a doctor told you that you have kidney problem?

Exclusion Criteria

Do you have a history of diabetes?

Are you currently undergoing dialysis treatment?

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 2 Treatment

Participants receive different dose levels of VX-147 or placebo

8-12 weeks

Phase 3 Treatment

Participants receive VX-147 or placebo based on Phase 2 outcomes

12-16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Participant Groups

The study is testing VX-147's effectiveness and safety compared to a placebo (a treatment with no active drug) in treating APOL1-mediated proteinuric kidney disease. It will also look at how the body processes the drug.

Group I: Phase 3: VX-147 Experimental Treatment 1 Intervention

Participants will receive VX-147 with the dose to be based on the outcome of Phase 2.

Group II: Phase 2: VX-147 Experimental Treatment 1 Intervention

Participants will be randomized to receive different dose levels of VX-147.

Group III: Phase 2: Placebo Placebo Group 1 Intervention

Participants will receive placebo matched to VX-147.

Group IV: Phase 3: Placebo Placebo Group 1 Intervention

Participants will receive placebo matched to VX-147.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

EmVenio Research - Chicago, IL Chicago, IL

Northwest Louisiana Nephrology, LLC - Shreveport Shreveport, LA

Henry Ford Hospital Detroit, MI

Minneapolis VA Healthcare System Minneapolis, MN

More Trial Locations

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated Lead Sponsor

References

1. United States pubmed.ncbi.nlm.nih.gov

Effect of Low-Dose Methotrexate on eGFR and Kidney Adverse Events: A Randomized Clinical Trial. [2023] Low-dose methotrexate (LD-MTX) is contraindicated in advanced CKD, but kidney safety in normal kidney function or mild-to-moderate CKD is less clear.

2. Spain pubmed.ncbi.nlm.nih.gov

[Anuria in HIV+ patients treated with indinavir]. [2013] Two cases of oliguric acute renal failure in HIV+ patients treated with indinavir are described.

3. England pubmed.ncbi.nlm.nih.gov

Effective elimination of high-dose methotrexate by repeated hemodiafiltration and high-flux hemodialysis in patients with acute kidney injury. [2022] Acute kidney injury (AKI) after high dose methotrexate (HD-MTX) is associated with delayed MTX-excretion and life-threatening toxicity. Glucapridase, the recommended therapy, is expensive and not always available.

4. Germany pubmed.ncbi.nlm.nih.gov

Coadministration of vindesine with high-dose methotrexate therapy increases acute kidney injury via BCRP, MRP2, and OAT1/OAT3. [2022] To investigate whether coadministration of vindesine is a risk factor for acute kidney injury caused by high-dose methotrexate in patients with hematologic malignancies and identify its mechanism.

5. Switzerland pubmed.ncbi.nlm.nih.gov

[Use of oral glucose-lowering agents in patients with renal impairment]. [2012] People with chronic kidney disease (CKD) of stages 3-4 (creatinine clearance