VX20-121-103 - Phase 3, Randomized, Double-blind, Controlled Study
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 Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation
Vertex Pharmaceuticals Incorporated
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 of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in cystic fibrosis (CF) subjects who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other triple combination responsive (TCR) CFTR mutation and no F508del 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).
North West - Greater Manchester South Research Ethics Committee
Date of REC Opinion
31 Dec 2021
Further Information Favourable Opinion