Focal Segmental Glomerulosclerosis (FSGS)Phase 1NCT06529796
Evaluation of the Pharmacokinetics and Safety of Inaxaplin in Participants With Mild or Moderate Hepatic Impairment
A Phase 1 study testing IXP in people with Focal Segmental Glomerulosclerosis (FSGS), run by Vertex Pharmaceuticals Incorporated at 3 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Focal Segmental Glomerulosclerosis (FSGS)
- What is being tested?
- IXP
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 3 sitesUnited States
- Study dates
- 2 Aug 2024to 20 Dec 2024
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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%)
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Division of Clinical Pharmacology, University of Miami
Miami, Florida, 33136
Texas Liver Institute
San Antonio, Texas, 78215
GCP Research
St. Petersburg, Florida, 33705
About this study
Official title: 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.
Read the 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.).
- Being tested
- IXP
- Medical subject headings
- Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IXPDrug
Also called VX-147, Inaxaplin
Tablet for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Mild Hepatic Impairment | Experimental | Participants will receive a single dose of IXP on Day 1. Receives: Drug: IXP |
| Cohort 2: Matched Healthy Participants | Experimental | Healthy participants matched to Cohort 1 will receive a single dose of IXP on Day 1. Receives: Drug: IXP |
| Cohort 3: Moderate Hepatic Impairment | Experimental | Participants will receive a single dose of IXP on Day 1. Receives: Drug: IXP |
| Cohort 4: Matched Healthy Participants | Experimental | Healthy participants matched to Cohort 3 will receive a single dose of IXP on Day 1. Receives: Drug: IXP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Observed Plasma Concentration (Cmax) of IXP
Measured: 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
Measured: From Day 1 up to Day 10
Secondary outcomes (1)
Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 up to Day 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2024
- Study start2 Aug 2024
- Primary completion20 Dec 2024
- Study completion20 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Focal Segmental Glomerulosclerosis (FSGS)Studies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06529796
- Sponsor's protocol ID
- VX20-147-006
- Status last verified
- Jan 2025
- First submitted
- 26 Jul 2024
- Last updated
- 20 Jan 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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