Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated115 enrolled

Overview

The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis (CF).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

115

Conditions

Cystic Fibrosis

Interventions

Placebo (matched to VX-121 suspension)DRUG

Placebo matched to VX-121 suspension for oral administration.

VX-121 (Suspension)DRUG

Suspension for oral administration.

TEZ/IVADRUG

Fixed-dose combination tablet for oral administration.

Eligibility

Sex: ALLMin age: 18 YearsHealthy volunteers:

Medical Language ↔ Plain English

Key Inclusion Criteria: * Part A, B, and C: Healthy Volunteers * Female subjects must be of non-childbearing potential * Between the ages of 18 and 55 years, inclusive * Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight \>50 kg * Part D: Subjects with CF * Heterozygous for F508del and an MF mutation (F/MF) * FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height * Body weight ≥35 kg Key Exclusion Criteria: * Part A, B and C: Healthy Volunteers * Any condition possibly affecting drug absorption * History of febrile illness or other acute illness within 5 days before the first study drug dose * Part D: Subjects with CF * History of clinically significant cirrhosis with or without portal hypertension * History of solid organ or hematological transplantation * Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply

Locations (7)

Academic Medical Center

Amsterdam, Netherlands

PRA Health Sciences Onderzoekscentrum UMCG

Groningen, Netherlands

UMC St. Radboud

Nijmegen, Netherlands

Erasmus Medical Center

Rotterdam, Netherlands

Outcomes

Primary Outcomes

Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time frame: From Day 1 Through Safety Follow-up (up to Day 15 for Part A [except Cohorts A3 and A9], up to Day 26 for Cohort A3, up to Day 34 for Cohort A9, up to Day 20 for Part B, up to Day 24 for Part C and up to Week 9 for Part D)

Secondary Outcomes

Part A: Maximum Observed Concentration (Cmax) of VX-121

Time frame: Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose

Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121

Time frame: Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose

Part B: Maximum Observed Concentration (Cmax) of VX-121

Time frame: Day 1, Day 5, and Day 10

Part B: Area Under the Concentration Versus Time Curve During the Dosing Interval (AUCtau) of VX-121

Time frame: Day 1, Day 5, and Day 10

Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121

Time frame: Pre-dose at Day 5 and Day 10

Part C: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and, IVA and Its Metabolites (M1-IVA and M6-IVA)

Time frame: Day 1, Day 7, and Day 14

Part C: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA)

Data from ClinicalTrials.gov

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