Evaluation of the Pharmacokinetics and Safety of VX-548 in Participants With Mild or Moderate Hepatic Impairment

March 18, 2024 updated by: Vertex Pharmaceuticals Incorporated

A Phase 1, Open-label Study to Evaluate the Pharmacokinetics and Safety of Multiple Doses of VX-548 in Subjects With Mild or Moderate Hepatic Impairment and in Matched Healthy Subjects

The purpose of this study is to evaluate the pharmacokinetics and safety of multiple doses of VX-584 in participants with mild or moderate hepatic impairment as compared to matched healthy controls.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
36

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Division of Clinical Pharmacology, University of Miami
    • Oklahoma
      • Orlando, Oklahoma, United States, 32809
        • Orlando Clinical Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Description

Key Inclusion Criteria:

  • Cohorts 1 and 3: Participants with Mild or Moderate Hepatic Impairment

    • Participants will satisfy the criteria for mild (Cohort 1) and moderate hepatic impairment (Cohort 3) defined as a Child-Pugh total score of 5 to 6 and 7 to 9 points, respectively at the screening visit
    • Participants will have chronic (greater than or equal to (≥) 6 months) documented liver disease

  • Cohorts 2 and 4: Matched Healthy Participants

    • Participants will be matched (cohort 2 matched to cohort 1; and cohort 4 matched to cohort 3) during screening to participants with hepatic impairment for age, sex, and weight

Key Exclusion Criteria:

  • Cohorts 1 and 3: Participants with Mild or Moderate Hepatic Impairment

    • History of febrile illness or other acute illness that has not fully resolved by 14 days before the first dose of study drug
    • Severe portal hypertension
    • History or presence of severe hepatic encephalopathy (Grade >2)
    • Any condition possibly affecting drug absorption
    • Significant renal dysfunction (creatinine clearance <60 milliliter per minute [mL/min] ) estimated according to the method of Cockcroft and Gault at the screening Visit or Day-1
    • History of solid organ or bone marrow transplantation

  • Cohorts 2 and 4: Matched Healthy Participants

    • History of febrile illness or other acute illness that has not fully resolved by 14 days before the first dose of study drug
    • Any condition possibly affecting drug absorption

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort 2: Matched Healthy Participants
Healthy participants matched to cohort 1 will receive multiple doses of VX-548 q12h from Day 1 through Day 14.
Drug: VX-548
Tablets for oral administration.
Other Names:
  • Suzetrigine
Experimental: Cohort 1: Mild Hepatic Impairment
Participants will receive multiple doses of VX-548 every 12 hours (q12h) from Day 1 through Day 14.
Drug: VX-548
Tablets for oral administration.
Other Names:
  • Suzetrigine
Experimental: Cohort 3: Moderate Hepatic Impairment
Participants will receive multiple doses of VX-548 q12h from Day 1 through Day 14.
Drug: VX-548
Tablets for oral administration.
Other Names:
  • Suzetrigine
Experimental: Cohort 4: Matched Healthy Participants
Healthy participants matched to cohort 3 will receive multiple doses of VX-548 q12h from Day 1 through Day 14.
Drug: VX-548
Tablets for oral administration.
Other Names:
  • Suzetrigine

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Maximum Observed Plasma Concentration (Cmax) of VX-548
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Area Under the Plasma Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-548
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Time Taken for VX-548 to Reach Maximum Concentration (tmax)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Time Required for Plasma Concentration of VX-548 to Reduce to Half (t1/2)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Apparent Volume of Distribution of VX-548 (Vz/F)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Apparent Clearance of VX-548 (CL/F)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to the Last Measured Concentration (AUC0-last) of VX-548
Time Frame: Day 1 to Day 31
Day 1 to Day 31

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Maximum Observed Plasma Concentration (Cmax) of VX-548 Metabolite
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Area Under the Plasma Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-548 Metabolite
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Time Taken for VX-548 metabolite to Reach Maximum Concentration (tmax)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Time Required for Plasma Concentration of VX-548 Metabolite to Reduce to Half (t1/2)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Apparent Volume of Distribution of VX-548 Metabolite (Vz/F)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Apparent Clearance of VX-548 Metabolite (CL/F)
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to the Last Measured Concentration (AUC0-last) of VX-548 Metabolite
Time Frame: Day 1 to Day 31
Day 1 to Day 31
Fraction Unbound (fu) for VX-548 and its Metabolite in Plasma
Time Frame: Day 14: Up to 24 hours Post-dose
Day 14: Up to 24 hours Post-dose
Unbound Area Under the Concentration Versus Time Curve of VX-548 and its Metabolite
Time Frame: Day 14: Up to 24 hours Post-dose
Day 14: Up to 24 hours Post-dose
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 42
From Day 1 up to Day 42

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 14, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 24, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 24, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 26, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 26, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 29, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 20, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 18, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2024

More Information

Terms related to this study

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • VX21-548-007

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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