VX20-121-104 - Phase 3, Open-Label Study Evaluating long term safety and effectiveness of VX-121
Research type
Research Study
Full title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
IRAS ID
1005889
Contact name
N/A N/A
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2021-000713-17
Clinicaltrials.gov Identifier
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.
Subjects who complete the last Treatment Period visit in a parent study and meet eligibility criteria are eligible to enroll. Parent studies are Phase 3 Vertex studies investigating VX-121/TEZ/D-IVA and include, but are not limited to, Study VX20-121-102 (Study 102) and Study VX20-121-103 (Study 103). This study will provide data on the long-term safety, efficacy, and durability of VX-121/TEZ/D-IVA in CF subjects who are homozygous or heterozygous for the F508del mutation, or who have at least 1 other triple combination responsive CFTR mutation and no F508del mutation (TCR/non-F).
The total study duration is approximately 100 weeks (96 weeks for Treatment Period and 4 weeks for the Safety Follow-up Period).REC name
South Central - Oxford A Research Ethics Committee
REC reference
23/SC/0111
Date of REC Opinion
28 Sep 2022
REC opinion
Further Information Favourable Opinion