A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Purpose
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
Condition
- Cystic Fibrosis
Eligibility
- Eligible Ages
- Over 12 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Heterozygous for F508del and an MF mutation (as defined in the protocol) - Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height
Exclusion Criteria
- Clinically significant cirrhosis with or without portal hypertension - Lung infection with organisms associated with a more rapid decline in pulmonary status - Solid organ or hematological transplantation Other protocol defined Inclusion/Exclusion criteria may apply
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator Placebo |
Participants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period. |
|
|
Experimental VX-659/TEZ/IVA TC |
Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period. |
|
More Details
- Status
- Completed
- Sponsor
- Vertex Pharmaceuticals Incorporated