VX20-121-102 - Phase 3, Randomized, Double-blind, Controlled Study
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)
IRAS ID
1003950
Contact name
Stacey Lindmark
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2021-000712-31
Research summary
Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR). Changes in this gene can affect the amount of the protein made from this gene or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky and causes problems in the lungs and other organs.
This study will evaluate the effectiveness and and safety of VX-121/TEZ/D-IVA in CF subjects who are homozygous (having two identical alleles of a particular gene or genes) for F508del (F/F), heterozygous (having two different alleles of a particular gene or genes) for F508del and a gating (F/G) or a (F/RF) mutation, or have at least 1 triple combination responsive CFTR mutation
The total study duration is approximately 64 weeks (4 weeks for the Screening Period, 4 weeks for the Run-in Period, 52 weeks for the Treatment Period, and 4 weeks for the Safety Follow-up Period).REC name
North West - Haydock Research Ethics Committee
REC reference
21/NW/0294
Date of REC Opinion
24 Nov 2021
REC opinion
Further Information Favourable Opinion