Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

Vertex Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Glomerulosclerosis, Focal Segmental

Treatments: Drug: VX-147

Study type: Interventional

Funder types: Industry

Identifiers

NCT04340362

2020-000185-42 (EudraCT Number)

VX19-147-101

Details and patient eligibility

About

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).

Enrollment: 16 patients

Sex: All

Ages: 18 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
• FSGS diagnosed by kidney biopsy

Key Exclusion Criteria:

• Evidence of non-APOL1-mediated FSGS
• Participants with known sickle cell disease
• Solid organ or Bone marrow transplant

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

VX-147

Experimental group

Description:

All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (\<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and \<2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.

Treatment:

Drug: VX-147