VX-840 for Kidney Disease

MI
Overseen ByMedical Information
Age: 18 - 65
Sex: Any
Trial Phase: Phase 1
Sponsor: Vertex Pharmaceuticals Incorporated

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on understanding the behavior and safety of a new treatment, VX-840, in the body. Researchers test different doses to assess tolerance levels. Participants will receive either the actual treatment or a placebo (a harmless pill resembling the treatment) in different study phases. The trial seeks healthy individuals with a body mass index (BMI) between 18 and 32 and a body weight over 50 kg. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

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 VX-840 is likely to be safe for humans?

Research has shown that VX-840 is currently being tested for safety in humans. In earlier studies, researchers administered VX-840 to healthy individuals to assess its safety and tolerability. The treatment was generally well-tolerated, with most participants experiencing no serious side effects. Some common side effects were mild and resolved on their own.

As this study is in its early stages, the primary goal is to ensure VX-840 does not cause harm. The drug is still undergoing careful testing before advancing to more advanced stages. Results so far suggest that VX-840 is safe enough to continue testing, but more information is needed to fully understand its safety profile.12345

Why do researchers think this study treatment might be promising?

VX-840 is unique because it represents a novel approach by focusing on safety and pharmacokinetics, which means it looks closely at how the drug moves through the body and at what dose levels it's safe. Unlike current treatments for similar conditions, which might rely on established mechanisms like blocking certain enzymes or receptors, VX-840 could offer a fresh mechanism or method of action. Researchers are excited because new insights into its pharmacokinetics might lead to more precise dosing, potentially improving effectiveness and reducing side effects compared to some existing options.

What evidence suggests that VX-840 could be effective?

Earlier studies have shown that VX-147, a molecule similar to VX-840, reduces proteinuria, a condition where excess protein is present in the urine, in patients with APOL1-related kidney disease. VX-147 blocks the APOL1 protein channel, which is believed to cause kidney damage. In this trial, participants will receive either VX-840 or a placebo. Although researchers are still testing VX-840 for safety and efficacy, it is designed based on VX-147's success. Researchers hope VX-840 may also improve kidney health by targeting the same process. Early findings from similar compounds suggest effectiveness, but further research is needed to confirm this.23456

Are You a Good Fit for This Trial?

Inclusion Criteria

Body mass index (BMI) of 18.0 to 32.0 kilograms per meter square (kg/m^2)
A total body weight greater than (>) 50 kg
Female participants must be of non-childbearing potential

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Participants receive a single dose of VX-840 or placebo at different dose levels

1 week

Treatment Part B

Participants receive multiple doses of VX-840 or placebo at different dose levels, determined based on Part A data

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Placebo
  • VX-840

How Is the Trial Designed?

4

Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B: VX-840Experimental Treatment1 Intervention
Group II: Part A: VX-840Experimental Treatment1 Intervention
Group III: Part A: PlaceboPlacebo Group1 Intervention
Group IV: Part B: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials
267
Recruited
36,100+
Dr. David Altshuler profile image

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani profile image

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology

Published Research Related to This Trial

VX-787 is a promising new oral medication for treating both pandemic and seasonal influenza, showing strong potential in its class.
Research on carboxylic acid isosteres of VX-787 revealed that maintaining a negative charge is crucial for the drug's effectiveness and selectivity against influenza A, as neutral replacements significantly reduced potency.
Isosteric replacements of the carboxylic acid of drug candidate VX-787: Effect of charge on antiviral potency and kinase activity of azaindole-based influenza PB2 inhibitors.Boyd, MJ., Bandarage, UK., Bennett, H., et al.[2019]
VX-128, a selective Nav 1.8 inhibitor, was found to be well-tolerated in single doses up to 300 mg, but showed a higher incidence of adverse effects compared to placebo, with 41.7% of subjects experiencing side effects.
In a multiple-ascending dose study, skin rashes and one serious case of angioedema were reported, leading to early termination of the study, although VX-128 demonstrated a dose-dependent trend in increasing pain tolerance in certain pain tests.
A phase I, randomized, double-blind, placebo-controlled, single- and multiple dose escalation study evaluating the safety, pharmacokinetics and pharmacodynamics of VX-128, a highly selective Nav 1.8 inhibitor, in healthy adults.Hijma, HJ., van Brummelen, EMJ., Siebenga, PS., et al.[2022]

Citations

APOL1 DIAGNOSTICS AND THERAPEUTICS

Pharmacokinetics (PK) of VX-840 in Healthy Participants. 1. Completed ? Compound inhibitor. NCT05351047. A Study to Assess Safety, Tolerability, PK and PD of.

Novel Therapies in APOL1-Mediated Kidney Disease

A novel small molecule inhibitor on APOL1 channel called VX-147 or inaxaplin demonstrated promising results in reducing proteinuria in patients with APOL1- ...

APOL1 Mediated Kidney Disease: A Review and Look ...

Zhang et al reported that the number of recipient risk variant alleles was associated with an increased risk of death, censored allograft loss, and risk of ...

A Phase 1 Dose Escalation Study to Evaluate Safety and ...

This trial tests a new drug called VX-840 to see if it is safe and well-tolerated in healthy people. The study focuses on safety before using the drug in ...

Real-World Outcomes with Chimeric Antigen Receptor T ...

Effectiveness outcomes of interest were OS, progression-free survival (PFS), overall response rate (ORR), and complete response (CR) rate.

VX-840 - Drug Targets, Indications, Patents

A Phase I, Randomized, Double-blind, Placebo-controlled Study Evaluating the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of VX-840 in ...