Overview

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.