What side effects are associated with this type of intervention?

Common treatments for kidney disease, particularly those involving immunosuppressive therapies and monoclonal antibodies, can have a range of side effects. These may include infections, hypertension, gastrointestinal issues, fatigue, and hematologic abnormalities such as anemia and neutropenia. Specific treatments like cyclophosphamide can cause toxicities such as bladder irritation and increased risk of infections, while prednisone can lead to weight gain, mood changes, and increased blood sugar levels. Plasmapheresis, used to remove harmful antibodies, can result in low blood pressure and allergic reactions. These side effects highlight the need for careful monitoring and management during treatment.

What prior FDA approvals exist?

The provided context does not mention specific FDA-approved drugs for APOL1 modulation in kidney disease. However, broadly speaking, the FDA has approved various treatments for kidney disease, including immunosuppressive drugs like cyclosporine and everolimus for transplant patients, and medications like sirolimus and calcineurin inhibitors for managing renal dysfunction post-transplant. Additionally, lipid-lowering agents such as simvastatin have been used to manage hyperlipidemia in renal transplant recipients.

What other types of research exist?

Promising alternatives to VX-147 for kidney disease patients include antisense oligonucleotide therapies targeting apolipoprotein(a) (apo(a)), such as apo(a)-LRx, which has shown significant reduction in Lp(a) levels in clinical trials. Additionally, PCSK9 inhibitors like evolocumab and alirocumab, which are effective in lowering LDL-C and have potential benefits in cardiovascular and kidney health, are also noteworthy.

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Small Molecule

VX-147 for Kidney Disease (AMPLITUDE Trial)

Verified Trial

Phase 2 & 3

Recruiting

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Are you currently between the ages of 18 and 65?

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

Must not have

Do you have a history of diabetes?

Are you currently undergoing dialysis treatment?

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline through study completion (approximately 2 years after the last participant enrolls)

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, common ones may include reactions at the site where the drug is given, changes in blood tests that monitor kidney function, allergic reactions to ingredients in VX-147 or general medication-related symptoms such as nausea or headaches.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline through study completion (approximately 2 years after the last participant enrolls)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline through study completion (approximately 2 years after the last participant enrolls)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline through study completion (approximately 2 years after the last participant enrolls) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at the Week 48 Interim Analysis

Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)

eGFR Slope Assessed at Study Completion

Secondary study objectives

Acceptability Tablet Formulation of VX-147 in Pediatric Participants using the Convenience Domain of the Treatment Satisfaction Questionnaire for Medication (TSQM) Version 1.4

Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147

Maximum Plasma Concentration (Cmax) of VX-147

+3 more

Side effects data

From 2021 Phase 2 trial • 16 Patients • NCT04340362

23%

Headache

23%

Back pain

15%

Dyspepsia

15%

Blood bicarbonate decreased

15%

Fatigue

15%

Nausea

15%

Dizziness

Abdominal distension

Deep vein thrombosis

Palpitations

Tooth abscess

Vaccination complication

Blood creatine phosphokinase increased

Uterine leiomyoma

Peripheral swelling

Abdominal pain lower

Upper respiratory tract infection

Diarrhoea

Gastrooesophageal reflux disease

COVID-19

Urinary tract infection

Vulvovaginal mycotic infection

Gamma-glutamyltransferase increased

Muscle spasms

Musculoskeletal chest pain

Pain in extremity

Synovitis

Depressed mood

Thinking abnormal

Pollakiuria

Eczema

Dry skin

Transaminases increased

100%

80%

60%

40%

20%

Study treatment Arm

VX-147: Cohort 2

VX-147 : Cohort 1

VX-147 Total

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Phase 3: VX-147Experimental Treatment1 Intervention

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

Group II: Phase 2: VX-147Experimental Treatment1 Intervention

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

Group III: Phase 2: PlaceboPlacebo Group1 Intervention

Participants will receive placebo matched to VX-147.

Group IV: Phase 3: PlaceboPlacebo Group1 Intervention

Participants will receive placebo matched to VX-147.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

VX-147

2020

Completed Phase 2

~40

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for kidney disease include immunosuppressive therapy, plasmapheresis, and targeted therapies such as APOL1 modulation. Immunosuppressive agents like cyclophosphamide and prednisone reduce inflammation and prevent the formation of harmful antibodies, which is crucial in conditions like anti-GBM disease. Plasmapheresis helps remove circulating antibodies and inflammatory mediators, providing immediate relief from acute symptoms. APOL1 modulation, as studied in trials like VX-147, targets genetic factors that contribute to kidney disease, offering a more personalized treatment approach. These treatments are vital for kidney disease patients as they help preserve kidney function, prevent progression to end-stage kidney disease, and improve overall prognosis.

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