Evaluation of the Pharmacokinetics and Safety of Inaxaplin in Participants With Mild or Moderate Hepatic Impairment

A Phase 1, Open-label Study to Assess the Pharmacokinetics and Safety of a Single Dose of Inaxaplin in Subjects With Mild or Moderate Hepatic Impairment and in Matched Healthy Subjects

The purpose of this study is to evaluate the pharmacokinetics, safety and tolerability of Inaxaplin (IXP) in participants with mild or moderate hepatic impairment and in matched healthy participants.

Study Overview

• Status: Completed
• Conditions: Focal Segmental Glomerulosclerosis (FSGS)
• Intervention / Treatment: Drug: IXP

Detailed Description

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • Division of Clinical Pharmacology, University of Miami
    • Saint Petersburg, Florida, United States, 33705
      • GCP Research
  • Texas
    • San Antonio, Texas, United States, 78215
      • Texas Liver Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Key Inclusion Criteria:

• Cohorts 1 and 3: Participants with Hepatic Impairment

  • Cohort 1 (mild impairment): defined as a Child-Pugh total score of 5 to 6 (Child-Pugh Class A)
  • Cohort 3 (moderate impairment): defined as a Child-Pugh total score of 7 to 9 (Child-Pugh Class B)

• Cohorts 2 and 4: Healthy Participants

  • Participants will be matched during screening to participants with mild (Cohort 1) or moderate (Cohort 3) hepatic impairment according to each of the following parameters: age (± 10 years); sex; and BMI (± 15%)

Key Exclusion Criteria:

• Cohorts 1 and 3: Participants with Hepatic Impairment

  • Acute febrile illness within 14 days and/or acute illness within 5 days before first dose of study drug
  • Participants who smoke

• Cohorts 2 and 4: Healthy Participants

  • Acute febrile illness within 14 days and/or acute illness within 5 days before first dose of study drug
  • Any condition possibly affecting drug absorption

Other protocol defined Inclusion/Exclusion criteria may apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: Mild Hepatic Impairment; Participants will receive a single dose of IXP on Day 1.	Drug: IXP; Tablet for oral administration.; Other Names: VX-147; Inaxaplin
Experimental: Cohort 2: Matched Healthy Participants; Healthy participants matched to Cohort 1 will receive a single dose of IXP on Day 1.	Drug: IXP; Tablet for oral administration.; Other Names: VX-147; Inaxaplin
Experimental: Cohort 3: Moderate Hepatic Impairment; Participants will receive a single dose of IXP on Day 1.	Drug: IXP; Tablet for oral administration.; Other Names: VX-147; Inaxaplin
Experimental: Cohort 4: Matched Healthy Participants; Healthy participants matched to Cohort 3 will receive a single dose of IXP on Day 1.	Drug: IXP; Tablet for oral administration.; Other Names: VX-147; Inaxaplin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Maximum Observed Plasma Concentration (Cmax) of IXP	From Day 1 up to Day 10
Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity (AUC0-inf) of IXP	From Day 1 up to Day 10

Secondary Outcome Measures

Outcome Measure	Time Frame
Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)	From Day 1 up to Day 20

Collaborators and Investigators

• Sponsor: Vertex Pharmaceuticals Incorporated

Study record dates

Study Major Dates

• Study Start (Actual): August 2, 2024
• Primary Completion (Actual): December 20, 2024
• Study Completion (Actual): December 20, 2024

Study Registration Dates

• First Submitted: July 26, 2024
• First Submitted That Met QC Criteria: July 26, 2024
• First Posted (Actual): July 31, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 17, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Glomerulonephritis; Nephritis; Glomerulosclerosis, Focal Segmental
• Other Study ID Numbers: VX20-147-006

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No