Vertex Pharmaceuticals, Inc. is investigating potential therapy for the ~5000 people living with CF, also known as the final 10%, who are not expected to benefit from CFTR modulators.
Vertex recently announced it has launched a new clinical research study – BEACON CF.
While most people living with cystic fibrosis (CF) can be treated with CFTR modulators, there are approximately 5000 patients with CF who have mutations in both of their CFTR genes that makes them unresponsive to CFTR modulators. This group of patients is often referred to as the final 10%*. There is an unmet medical need for therapies in this group of people living with CF.
About the BEACON-CF clinical research study
This is a phase 1 clinical research study exploring the safety and tolerability of VX-522, an investigational messenger RNA (mRNA) therapy, for people living with CF who have genotypes not expected to benefit from CFTR modulators.
Who is this study for?
1. Have been diagnosed with CF and are not expected to respond to CFTR modulators
2. Have forced expiratory volume 1 second (FEV1) of 40% or more
3. Do not have a history of solid organ or hematological transplantation
4. Are between 18-65 years old
Please note this is not a full list of eligibility criteria.
If you qualify for the research study and choose to participate you will receive all study-related medications and procedures at no cost. In addition, you will be compensated for your study related time and reimbursed for expenses needed to participate in the clinical research study.
New medicines are made possible by the volunteers who participate in clinical research studies. Vertex encourages people with CF to speak first with their doctor about their potential eligibility to enroll in a clinical research study.
For more information, please visit https://beacon-cf.com/bef
*Some members of the CF community commonly identify as the final 10%.
Protocol Title: A Phase 1 Single Dose Escalation Study Evaluating the Safety and Tolerability of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy. https://clinicaltrials.gov/ct2/show/NCT05668741