A Phase 1/2 Study of VX-522 in Subjects with Cystic Fibrosis
Research type
Research Study
Full title
A Phase 1/2 Dose-escalating Study Evaluating the Safety, Tolerability, and Efficacy of VX-522 in Subjects 18 Years of Age and Older with Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
IRAS ID
1005878
Contact name
Kari Forsaith
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2022-000726-25
Clinicaltrials.gov Identifier
Research summary
The BEACON-CF clinical research study will help to find out whether a single dose of an investigational study drug, called VX-522, is safe and well tolerated as a potential treatment for people living with cystic fibrosis who are not expected to benefit from a type of existing treatment called CFTR modulators. The investigational study drug, VX-522, is a messenger RNA (mRNA) therapy which is inhaled into the lungs. mRNA therapy works by sending a message that tells cells (the building blocks of the body) to produce normal proteins to do the job of proteins that do not work properly or are missing.
There are three parts to this study:
The first part will evaluate the safety and tolerability of single ascending doses (SAD) of VX-522, and participants are planned to be in the study for 28 weeks. The second part will evaluate safety and tolerability of multiple ascending doses (MAD) of VX-522 , and participants are planned to be in the study for up to 12 weeks. The third part will evaluate the safety and tolerability of multiple doses of VX-522 co-administered with ivacaftor (IVA) treatment, and participants are planned to be in the study for up to 16 weeks.The study will include male and female participants of 18 years old to 65 years old.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0198
Date of REC Opinion
9 May 2023
REC opinion
Further Information Favourable Opinion