NCT05437120

Brief Summary

The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with moderate hepatic impairment and in matched healthy participants.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jul 2022

Shorter than P25 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 23, 2022

Completed
6 days until next milestone

First Posted

Study publicly available on registry

June 29, 2022

Completed
23 days until next milestone

Study Start

First participant enrolled

July 22, 2022

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 16, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 16, 2023

Completed
Last Updated

March 30, 2023

Status Verified

March 1, 2023

Enrollment Period

8 months

First QC Date

June 23, 2022

Last Update Submit

March 29, 2023

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (5)

  • Maximum Observed Plasma Concentration (Cmax) of VX-121, TEZ, D-IVA, and Relevant Metabolites

    Cohort 1: Pre-dose up to Day 23; Cohort 2: Pre-dose up to Day 13

  • Area Under the Concentration Versus Time Curve (AUC) of VX-121,TEZ, D-IVA, and Relevant Metabolites

    Cohort 1: Pre-dose up to Day 23; Cohort 2: Pre-Dose up to Day 13

  • Fraction Unbound (fu) for VX-121 and D-IVA in Plasma

    Cohorts 1 and 2: Pre-dose up to Day 2

  • Unbound Maximum Observed Concentration (Cmax ub) for VX-121 and D-IVA

    Cohorts 1 and 2: Pre-dose up to Day 2

  • Unbound Area Under the Concentration Versus Time Curve (AUC ub) of VX-121 and D-IVA

    Cohorts 1 and 2: Pre-dose up to Day 2

Secondary Outcomes (1)

  • Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Cohort 1: Day 1 up to Day 32; Cohort 2: Day 1 up to Day 17

Study Arms (2)

Cohort 1: Moderate Hepatic Impairment

EXPERIMENTAL

Participants with moderate hepatic impairment will receive single dose of VX-121/TEZ/D-IVA .

Drug: VX-121/TEZ/D-IVA

Cohort 2: Matched Healthy Participants

EXPERIMENTAL

Healthy participants will receive single dose of VX-121/TEZ/D-IVA.

Drug: VX-121/TEZ/D-IVA

Interventions

VX-121/TEZ/D-IVADRUG

Fixed-dose combination tablets for oral administration.

Also known as: VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, VX-121/tezacaftor/deutivacaftor
Cohort 1: Moderate Hepatic ImpairmentCohort 2: Matched Healthy Participants

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Cohort 1: Participants with Moderate Hepatic Impairment
  • Participants will satisfy the criteria for moderate hepatic impairment defined as a Child-Pugh total score of 7 to 9 points at the screening visit
  • Participants will have chronic (≥6 months) documented liver disease
  • Cohort 2: Matched Healthy Participants
  • Participants will be matched during screening to participants with hepatic impairment for cigarette smoking habit, age, sex, and weight

You may not qualify if:

  • Cohort 1: Participants with Moderate Hepatic Impairment
  • History of febrile illness or other acute illness
  • History of solid organ or bone marrow transplantation
  • History or presence of severe hepatic encephalopathy (Grade \>2)
  • Any condition possibly affecting drug absorption
  • Severe portal hypertension
  • Significant renal dysfunction (creatinine clearance \<50 milliliter per minute \[mL/min\] ) estimated according to the method of Cockcroft and Gault at the screening Visit or Day-1
  • Cohort 2: Matched Healthy Participants
  • History of febrile illness or other acute illness
  • Any condition possibly affecting drug absorption

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Clinical Pharmacology of Miami, LLC

Miami, Florida, 33014-3616, United States

Location

GCP Research

St. Petersburg, Florida, 33705, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 23, 2022

First Posted

June 29, 2022

Study Start

July 22, 2022

Primary Completion

March 16, 2023

Study Completion

March 16, 2023

Last Updated

March 30, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

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

Locations

US(2)