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

Vertex Pharmaceuticals Incorporated
NCT IDNCT05422222ClinicalTrials.gov data as of Apr 2026
Phase

Phase 3

Target enrollment

210

Study length

about 8 years

Ages

1–11

Locations

19 sites in CA, CO, GA +12

What this study is about

This trial is testing a treatment called VX-121/TEZ/D-IVA for children with cystic fibrosis who have at least one mutation that responds to triple combination therapy. The goal is to see if this treatment is safe, well-tolerated, and effective in these individuals.

Simplified from trial records by PatientMatch.

What you may be asked to do

  • 1.Take VX-121/TEZ/D-IVA

Participation Burden

What's physically and logistically required of participants.

Logistics & Travel
In-person visits

Requires travel to a study site

Physical Intervention
Standard

How treatment is administered

Treatment Assignment
All receive treatment

Everyone gets the investigational treatment.

Extracted study details

Pulled from the trial record to show what is being tested and what the study is measuring.

Endpoints

Primary: 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: Part B: Absolute Change in BMI-for-age Z-score, Part B: Absolute Change in Body Mass Index (BMI), Part B: Absolute Change in Weight, Part B: Absolute Change in Weight-for-age Z-score, Part B: Absolute Change in Weight-for-length, Part B: Absolute Change in Weight-for-length Z-score

Body systems

Respiratory