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
- Are there results?
- None posted in the registry
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
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06529796
- Acronym / sponsor ID
- VX20-147-006
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 2 Aug 2024 (actual)
- Primary completion
- 20 Dec 2024 (actual)
- Study completion
- 20 Dec 2024 (actual)
- First posted
- 31 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Mild Hepatic Impairment | Experimental | Drug: IXPParticipants will receive a single dose of IXP on Day 1. |
| Cohort 2: Matched Healthy Participants | Experimental | Drug: IXPHealthy participants matched to Cohort 1 will receive a single dose of IXP on Day 1. |
| Cohort 3: Moderate Hepatic Impairment | Experimental | Drug: IXPParticipants will receive a single dose of IXP on Day 1. |
| Cohort 4: Matched Healthy Participants | Experimental | Drug: IXPHealthy participants matched to Cohort 3 will receive a single dose of IXP on Day 1. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Cohorts 1 and 3: Participants with Hepatic Impairment
- Cohorts 2 and 4: Healthy Participants
Key exclusion criteria (7 listed)
- Cohorts 1 and 3: Participants with Hepatic Impairment
- Cohorts 2 and 4: Healthy Participants
- Other protocol defined Inclusion/Exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IXPAlso: VX-147, InaxaplinTablet for oral administration. | Drug | Cohort 1: Mild Hepatic Impairment, Cohort 2: Matched Healthy Participants, Cohort 3: Moderate Hepatic Impairment, Cohort 4: Matched Healthy Participants |
Primary outcome
Maximum Observed Plasma Concentration (Cmax) of IXP
Time frame: 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
Time frame: From Day 1 up to Day 10
1 secondary outcome
Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Day 20
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2025.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IXP in people with Focal Segmental Glomerulosclerosis (FSGS), run by Vertex Pharmaceuticals Incorporated at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Observed Plasma Concentration (Cmax) of IXP (time frame: From Day 1 up to Day 10). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2025.