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CFTR Modulator

VX-121 + Tezacaftor + Deutivacaftor for Cystic Fibrosis

Phase 3

Waitlist Available

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from day 1 up to day 50

Awards & highlights

Study Summary

This trial is to study VX-121/tezacaftor/deutivacaftor, a drug combo for treating cystic fibrosis, in patients with a certain mutation.

Who is the study for?

This trial is for children aged 1-11 with stable cystic fibrosis who have at least one mutation responsive to the treatment. It's not suitable for those with a history of organ transplant, cancer, significant liver disease, or certain lung infections that worsen breathing.Check my eligibility

What is being tested?

The study tests VX-121/tezacaftor/deutivacaftor in young CF patients to see how their bodies handle it and if it's safe and effective. The medication aims to improve lung function by targeting the defective protein causing CF.See study design

What are the potential side effects?

While specific side effects aren't listed here, common ones for CF treatments include chest discomfort, coughing, digestive changes, and potential liver issues. Monitoring during the trial helps manage any adverse reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My cystic fibrosis is stable and I have at least one CFTR gene mutation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from day 1 up to day 50

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from day 1 up to day 50

This trial's timeline: 3 weeks for screening, Varies for treatment, and from day 1 up to day 50 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites

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

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

Secondary outcome measures

Part B: Absolute Change in BMI-for-age Z-score

Part B: Absolute Change in Body Mass Index (BMI)

Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

+16 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Part B: VX-121/TEZ/D-IVAExperimental Treatment1 Intervention

Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.

Group II: Part A: VX-121/TEZ/D-IVAExperimental Treatment1 Intervention

Participants will receive VX-121/TEZ/D-IVA in the morning.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

VX-121/TEZ/D-IVA

2022

Completed Phase 3

~1070

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

243 Previous Clinical Trials

32,177 Total Patients Enrolled

126 Trials studying Cystic Fibrosis

17,539 Patients Enrolled for Cystic Fibrosis

Media Library

VX-121/TEZ/D-IVA (CFTR Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT05422222 — Phase 3

Cystic Fibrosis Research Study Groups: Part A: VX-121/TEZ/D-IVA, Part B: VX-121/TEZ/D-IVA

Cystic Fibrosis Clinical Trial 2023: VX-121/TEZ/D-IVA Highlights & Side Effects. Trial Name: NCT05422222 — Phase 3

VX-121/TEZ/D-IVA (CFTR Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05422222 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are investigators still looking for participants for this research project?

"Yes, an inspection of clinicaltrials.gov discloses that this trial is currently looking for participants. This specific study was posted on June 21st, 2022 and updated as recently as August 30th, 8/30/2022. 210 individuals are needed across 8 locations."

Answered by AI

Does this research project consider applicants who are adults?

"The age bracket for patients that this study can accept begins at 1 year old and ends at 10 years old."

Answered by AI

Could you give me an estimate of how many individuals are involved in this research project?

"In order to start this clinical trial, 210 eligible patients must enroll. These individuals can be recruited from several locations, such as Boston Children's Hospital in Boston, Massachusetts and UPMC Children's Hospital of Pittsburgh in Pittsburgh, Pennsylvania."

Answered by AI

How many different hospitals are participating in this clinical trial?

"To make things more convenient for participants, this clinical trial is taking place at 8 locations. Those who live near Boston, Pittsburgh or Aurora will have a shorter distance to travel. However, there are also 8 other sites located in different cities."

Answered by AI

Who does this study aim to target as potential participants?

"The ideal candidate for this study would be somebody with cystic fibrosis who is between 1 and 11 years old. They should also have at least one TCR mutation in the CFTR gene."

Answered by AI

Are there any reported dangers of VX-121/TEZ/D-IVA to human beings?

"VX-121/TEZ/D-IVA is a phase 3 trial drug, which means that while there is some evidence of efficacy, mostly there is data backing up its safety. Power rates it as a 3 on their safety scale."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

2022. "Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05422222.

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