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Completed

NCT Number: NCT04340362

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

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

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Key information

Conditions

Glomerulosclerosis, Focal Segmental Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Glomerulonephritis Kidney Diseases Male Urogenital Diseases Nephritis Urogenital Diseases Urologic Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hopital Henri Mondor, Créteil, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

VX-147

Drug

Tablets for oral administration.

Other names: IXP

Primary outcomes

  1. Percent Change From Baseline in UPCR

    Time frame: From Baseline up to Week 13

Secondary outcomes

  1. Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: From Baseline up to Week 17

  2. Maximum Observed Concentration (Cmax) of VX-147

    Time frame: Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5

  3. Observed Pre-dose Concentration (Ctrough) of VX-147

    Time frame: Pre-dose on Day 8, 15, Week 3, 5, 9 and 13

  4. Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147

    Time frame: Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Apr 9, 2020
Registry last updated
Sep 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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