A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

• Status: Completed
• Trial end date: 2018-12-21
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Key Inclusion Criteria:

• Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).

• Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.

• Participants with a screening LCI2.5 result ≥7.5.

• Participants who are able to swallow tablets.

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension.

• Colonization with organisms associated with a more rapid decline in pulmonary status.

• Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.