Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1- Mediated Proteinuric Kidney Disease (AMPLITUDE)

Vertex Pharmaceuticals

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Proteinuric Kidney Disease

Treatments: Drug: Placebo; Drug: VX-147

Study type: Interventional

Funder types: Industry

Identifiers

NCT05312879

VX21-147-301

2021-004762-35 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.

Enrollment: 466 estimated patients

Sex: All

Ages: 10 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• APOL1 genotype of G1/G1, G2/G2, or G1/G2
• Proteinuric kidney disease

Key Exclusion Criteria:

• Solid organ or bone marrow transplant
• Uncontrolled hypertension
• History of diabetes mellitus
• Known underlying cause of kidney disease including but not limited to sickle cell disease

Other protocol defined Inclusion/Exclusion criteria apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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466 participants in 4 patient groups, including a placebo group

Phase 2: VX-147

Experimental group

Description:

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

Treatment:

Drug: VX-147

Phase 2: Placebo

Placebo Comparator group

Description:

Participants will receive placebo matched to VX-147.

Treatment:

Drug: Placebo

Phase 3: VX-147

Experimental group

Description:

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

Treatment:

Drug: VX-147

Phase 3: Placebo

Placebo Comparator group

Description:

Participants will receive placebo matched to VX-147.

Treatment:

Drug: Placebo