Inaxaplin for Kidney Disease

(AMPLIFIED Trial)

Recruiting at 33 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: FSGS, Uncontrolled hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 13 weeks

45 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Inaxaplin to determine its effectiveness for a specific type of kidney disease linked to the APOL1 gene. Researchers aim to assess the safety, effectiveness, and tolerability of Inaxaplin. They seek participants with kidney disease related to the APOL1 gene who do not have uncontrolled high blood pressure. Individuals experiencing protein in their urine due to this gene may be suitable for the study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that Inaxaplin is likely to be safe for humans?

A previous study found Inaxaplin to be safe and well tolerated. Participants took single doses up to 165 mg and multiple doses up to 120 mg daily for two weeks without significant problems. These results suggest that the treatment is generally well tolerated. No major side effects were reported at these dosage levels, indicating a promising safety profile for Inaxaplin. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Inaxaplin is unique because it targets proteinuric APOL1-mediated kidney disease (AMKD) directly, which is different from current treatments like ACE inhibitors or ARBs that mainly focus on controlling blood pressure and reducing proteinuria. This means Inaxaplin has the potential to address the root cause of kidney damage in patients with AMKD. Additionally, Inaxaplin is administered as a single daily dose, which could improve patient adherence compared to more complex treatment regimens. Researchers are excited about its potential to offer a more targeted and convenient approach to treating this specific kind of kidney disease.

What evidence suggests that Inaxaplin might be an effective treatment for kidney disease?

Research has shown that Inaxaplin holds promise for treating APOL1-mediated kidney disease (AMKD). In earlier studies, blocking the APOL1 channel with Inaxaplin led to a noticeable decrease in proteinuria, a condition where excess protein in the urine indicates kidney issues. Currently, no specific treatment exists for AMKD, but these early results offer hope. This trial will evaluate Inaxaplin as a potential new treatment option, aiming to address the genetic cause of the disease for those affected. ^{2 4 5 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with APOL1-mediated kidney disease, specifically those who have certain genetic markers (G1/G1, G2/G2, or G1/G2) and a minimum kidney function level (eGFR ≥ 25 mL/min/1.73m^2).

Inclusion Criteria

My APOL1 genotype is G1/G1, G2/G2, or G1/G2.

My kidney function test shows an eGFR of 25 mL/min or higher.

Exclusion Criteria

My blood pressure is not well-controlled.

My kidney condition is diagnosed as FSGS not caused by APOL1 gene changes.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of Inaxaplin (IXP) once daily for 13 weeks

13 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Inaxaplin

Trial Overview The study tests the effectiveness and safety of a drug called Inaxaplin in patients with proteinuric APOL1-mediated kidney disease to see how well it works and what side effects it might have.

How Is the Trial Designed?

Group I: IXP (Inaxaplin) Experimental Treatment 1 Intervention

Participants with proteinuric APOL1-mediated kidney disease (AMKD) with or without the comorbidity of Type 2 diabetes mellitus (T2DM) will receive a single dose of IXP once daily, for 13 weeks.

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials

267

Recruited

36,100+

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology

Citations

1. nejm.org nejm.org/doi/full/10.1056/NEJMoa2202396

Inaxaplin for Proteinuric Kidney Disease in Persons with ... Targeted inhibition of APOL1 channel function with inaxaplin reduced proteinuria in participants with two APOL1 variants and focal segmental glomerulosclerosis.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10461697/

Inaxaplin for the treatment of APOL1-associated kidney ... While research efforts has been impressive, there is currently no effective targeted therapy for APOL1 mediated kidney disease (AMKD).

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06794996

Study Details | NCT06794996 | Inaxaplin in Participants ... The purpose of the study is to evaluate the efficacy, safety, and tolerability of Inaxaplin (IXP) in participants with proteinuric APOL1- mediated kidney ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2590059525000986

APOL1 Mediated Kidney Disease: A Review and Look ... A strong association between APOL1 and numerous kidney diseases has become evident. Some of the strongest associations has been between HIV-associated ...

5. investors.vrtx.com investors.vrtx.com/news-releases/news-release-details/vertex-advances-inaxaplin-vx-147-phase-3-portion-adaptive-phase

Vertex Advances Inaxaplin (VX-147) into Phase 3 Portion ... Inaxaplin (VX-147) has advanced into the Phase 3 portion of the global Phase 2/3 pivotal clinical trial in APOL1-mediated kidney disease (AMKD).

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11006409/

Safety and Tolerability of the APOL1 Inhibitor, Inaxaplin ... Inaxaplin is safe and well tolerated at single doses up to 165 mg and multiple doses up to 120 mg daily for 14 days.

7. vrtx.com vrtx.com/our-science/pipeline/apol1-mediated-kidney-disease/

R&D Pipeline | APOL1-Mediated Kidney Disease AMKD is a genetic kidney disease caused by having two APOL1 risk variants. The disease can lead to kidney cell injury, cell death and damage to the glomeruli.