Evaluation of ELX/TEZ/IVA in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
Research type
Research Study
Full title
A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
IRAS ID
1007349
Contact name
Rajesh Thangapazham
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Research summary
VX22-445-122 (Study 122) is a phase 3, 2-part (part A and part B), multicentre, open-label study evaluating the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD) and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than 24 months of age. Approximately 14 subjects will be administered ELX/TEZ/IVA for 15 days in Part A and 50 subjects will be administered ELX/TEZ/IVA for 24 weeks of treatment in Part B.
• Part A - Safety, tolerability, and available PK data will be reviewed by Vertex after Part A to confirm or revise the dosage levels chosen for Part B. Based on emerging PK data, additional subjects may be enrolled as needed in Part A in order to provide sufficient information to confirm or revise the dosage levels for Part B.
• Part B - Part B will initiate after available data from Part A have been reviewed by Vertex and the Part B dosage levels have been confirmed or revised. Subjects will be enrolled sequentially in Cohort B1 (18 to <24 months of age) followed by Cohort B2 (12 to <18 months of age). Initiation of Cohort B2 will be based on a review by Vertex of all Cohort B1 PK data through Week 4 and all safety data through Week 8.At present this application supports a request for approval of Part A of Study 122 only. Following completion of Part A of Study 122, updated documentation will be submitted via a substantial amendment for approval prior to the initiation of Part B of the study.
REC name
London - Riverside Research Ethics Committee
REC reference
23/LO/0194
Date of REC Opinion
13 Sep 2023
REC opinion
Further Information Favourable Opinion