MedDataX Logo

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

NCT ID: NCT03768089

Last Updated: 2022-07-14

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

115 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-03-20

Study Completion Date

2019-05-03

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

NCT03912233

Cystic Fibrosis
COMPLETED PHASE2

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

NCT05076149

Cystic Fibrosis
COMPLETED PHASE3

A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

NCT05033080

Cystic Fibrosis
COMPLETED PHASE3

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

NCT05444257

Cystic Fibrosis
ACTIVE_NOT_RECRUITING PHASE3

A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

NCT03911713

Cystic Fibrosis
COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Part A: Pooled Placebo (Cohorts A1-5; Except A3)

Participants received single dose of placebo matched to VX-121.

Group Type PLACEBO_COMPARATOR

Placebo (matched to VX-121 suspension)

Intervention Type DRUG

Placebo matched to VX-121 suspension for oral administration.

Part A: VX-121 (Cohort A1)

Participants received single dose of VX-121 10 milligrams (mg).

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part A: VX-121 (Cohort A2)

Participants received single dose of VX-121 20 mg.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part A: VX-121 (Cohort A3)

Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk.

Group Type EXPERIMENTAL

Placebo (matched to VX-121 suspension)

Intervention Type DRUG

Placebo matched to VX-121 suspension for oral administration.

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part A: VX-121 (Cohort A4)

Participants received single dose of VX-121 40 mg.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part A: VX-121 (Cohort A5)

Participants received single dose of VX-121 60 mg.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part A: VX-121 (Cohort A9)

Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

VX-121 (Tablet)

Intervention Type DRUG

Tablet for oral administration.

Part B: Pooled Placebo (Cohorts B1-4)

Participants received placebo matched to VX-121 for 10 days.

Group Type PLACEBO_COMPARATOR

Placebo (matched to VX-121 suspension)

Intervention Type DRUG

Placebo matched to VX-121 suspension for oral administration.

Part B: VX-121 (Cohort B1)

Participants received VX-121 10 mg once daily (qd) for 10 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part B: VX-121 (Cohort B2)

Participants received VX-121 20 mg qd for 10 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part B: VX-121 (Cohort B3)

Participants received VX-121 40 mg qd for 10 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part B: VX-121 (Cohort B4)

Participants received VX-121 60 mg qd for 10 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

Part C: Pooled Placebo (Cohorts C1-3)

Participants received placebo matched to VX-121/TEZ/IVA for 14 days.

Group Type PLACEBO_COMPARATOR

Placebo (matched to VX-121 suspension)

Intervention Type DRUG

Placebo matched to VX-121 suspension for oral administration.

Placebo (matched to TEZ/IVA)

Intervention Type DRUG

Placebo matched to TEZ/IVA for oral administration.

Placebo (matched to IVA)

Intervention Type DRUG

Placebo matched to IVA for oral administration.

Part C: VX-121 (Cohort C1)

Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 14 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

TEZ/IVA

Intervention Type DRUG

Fixed-dose combination tablet for oral administration.

IVA

Intervention Type DRUG

Tablet for oral administration.

Part C: VX-121 (Cohort C2)

Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

TEZ/IVA

Intervention Type DRUG

Fixed-dose combination tablet for oral administration.

IVA

Intervention Type DRUG

Tablet for oral administration.

Part C: VX-121 (Cohort C3)

Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days.

Group Type EXPERIMENTAL

VX-121 (Suspension)

Intervention Type DRUG

Suspension for oral administration.

TEZ/IVA

Intervention Type DRUG

Fixed-dose combination tablet for oral administration.

IVA

Intervention Type DRUG

Tablet for oral administration.

Part D: Placebo

Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks.

Group Type PLACEBO_COMPARATOR

Placebo (matched to TEZ/IVA)

Intervention Type DRUG

Placebo matched to TEZ/IVA for oral administration.

Placebo (matched to IVA)

Intervention Type DRUG

Placebo matched to IVA for oral administration.

Placebo (matched to VX-121 tablet)

Intervention Type DRUG

Placebo matched to VX-121 tablet for oral administration.

Part D: VX-121/TEZ/IVA

Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks.

Group Type EXPERIMENTAL

TEZ/IVA

Intervention Type DRUG

Fixed-dose combination tablet for oral administration.

IVA

Intervention Type DRUG

Tablet for oral administration.

VX-121 (Tablet)

Intervention Type DRUG

Tablet for oral administration.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Placebo (matched to VX-121 suspension)

Placebo matched to VX-121 suspension for oral administration.

Intervention Type DRUG

VX-121 (Suspension)

Suspension for oral administration.

Intervention Type DRUG

TEZ/IVA

Fixed-dose combination tablet for oral administration.

Intervention Type DRUG

IVA

Tablet for oral administration.

Intervention Type DRUG

Placebo (matched to TEZ/IVA)

Placebo matched to TEZ/IVA for oral administration.

Intervention Type DRUG

Placebo (matched to IVA)

Placebo matched to IVA for oral administration.

Intervention Type DRUG

VX-121 (Tablet)

Tablet for oral administration.

Intervention Type DRUG

Placebo (matched to VX-121 tablet)

Placebo matched to VX-121 tablet for oral administration.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

VX-661/VX-770 tezacaftor/ivacaftor VX-770 ivacaftor

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Academic Medical Center

Amsterdam, , Netherlands

Site Status

PRA Health Sciences Onderzoekscentrum UMCG

Groningen, , Netherlands

Site Status

UMC St. Radboud

Nijmegen, , Netherlands

Site Status

Erasmus Medical Center

Rotterdam, , Netherlands

Site Status

HagaZiekenhuis van den Haag

The Hague, , Netherlands

Site Status

Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital

Birmingham, , United Kingdom

Site Status

The Medicines Evaluation Unit

Manchester, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Netherlands United Kingdom

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2018-000126-55

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX17-121-001

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
NCT05422222 RECRUITING PHASE3
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
NCT02709109 COMPLETED PHASE2
Pharmacokinetics and Safety Assessment of VX-121/Tezacaftor/Deutivacaftor in Participants With Moderate Hepatic Impairment
NCT05437120 COMPLETED PHASE1
A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
NCT03224351 COMPLETED PHASE2
A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
NCT03227471 COMPLETED PHASE1/PHASE2
A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
NCT03029455 COMPLETED PHASE1
Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
NCT00865904 COMPLETED PHASE2
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT05668741 RECRUITING PHASE1/PHASE2
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
NCT02070744 COMPLETED PHASE2
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
NCT01531673 COMPLETED PHASE2
A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis
NCT02951182 COMPLETED PHASE2
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
NCT04043806 COMPLETED PHASE3
Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
NCT03691779 COMPLETED PHASE3
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
NCT04537793 COMPLETED PHASE3
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
NCT02347657 COMPLETED PHASE3
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants
NCT07283770 RECRUITING PHASE1
A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
NCT02508207 COMPLETED PHASE2
A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
NCT04105972 COMPLETED PHASE3
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
NCT04353817 COMPLETED PHASE3
A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)
NCT05111145 COMPLETED PHASE3
A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult Subjects
NCT03486236 COMPLETED PHASE1
Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
NCT04058366 COMPLETED PHASE3
Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy Subjects
NCT01216046 COMPLETED PHASE1
A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis
NCT02951195 COMPLETED PHASE2
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
NCT02516410 COMPLETED PHASE3