Triple Combination Therapy for Cystic Fibrosis

Recruiting in Palo Alto (17 mi)

+31 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Vertex Pharmaceuticals Incorporated

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing a combination of three medications to help people with cystic fibrosis. The goal is to see if these drugs are safe and effective over time. These medications work by improving the function of a faulty protein in cystic fibrosis patients, which can enhance their lung function and overall health. Trikafta is a combination of three drugs (elexacaftor, tezacaftor, and ivacaftor) that target the F508del mutation of the CFTR gene.

Eligibility Criteria

This trial is for people with cystic fibrosis who finished treatment in a previous study (VX21-121-105). It's not open to those who've had organ transplants, cancer, drug intolerance during the prior study, or serious liver issues like cirrhosis with portal hypertension.

Inclusion Criteria

Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Exclusion Criteria

You had a bad reaction to a drug in the previous study.

I have a history of organ transplant or cancer.

I have severe liver problems that could make taking new medications risky.

Participant Groups

The trial tests the long-term safety and effectiveness of a combination medication called vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) for individuals with cystic fibrosis.

1Treatment groups

Experimental Treatment

Group I: VNZ/TEZ/D-IVAExperimental Treatment1 Intervention

Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study.