BEACON-CF: Investigational mRNA Therapy for Cystic Fibrosis Request More Information Trial Objective For many people living with cystic fibrosis, there are treatments available called cystic fibrosis transmembrane conductance regulator (CFTR) modulators that can help treat the underlying cause of cystic fibrosis. However, more than 5,000 people living with cystic fibrosis have a change in both of their CFTR genes that makes them not expected to benefit from CFTR modulators. The BEACON-CF clinical research study is exploring an investigational study drug called VX-522, a type of messenger RNA (mRNA) therapy, to see if it is safe and well tolerated for those who are not expected to benefit from CFTR modulators. mRNA therapy is designed to send a message that tells cells (the building blocks of the body) to produce normal proteins that can do the job of proteins that are faulty or missing. If the investigational study drug is effective, it will tell cells in the lungs to create normal CFTR protein, which may improve the way the lungs work in people with cystic fibrosis. VX-522 will be given as a single dose using a modified PARI nebulizer, an investigational medical device that turns liquid medications into a mist, which is then inhaled. Enrollment Active Clinical Trials Currently Recruiting Who Can Participate Adults aged 18-65 with cystic fibrosis who are not expected to benefit from CFTR modulators. Participants must have a forced expiratory volume in 1 second (FEV1) value of more than or equal to 40%. Participants also cannot have a history of solid organ or hematological transplantation. Additional eligibility criteria may apply. Age: 18-65 Gender: Any Gender Estimated Time Commitment 28 weeks with 8 in-person visits and 4 phone calls. × Payments are intended to compensate the participant for their time and effort during the study. Payments are typically given to the participant at the end of each completed study visit. Reimbursements are intended to compensate the participant for travel-related expenses. They must be approved by the study staff and require you to turn in receipts. Travel reimbursement will vary from study to study. Payment & Reimbursement Payment: Provided Travel Reimbursement: Available Trial Contact For more information, contact: Samantha Johnson 303-270-2598 Request More Information Trial Location National Jewish Main Campus, Denver, CO Trial Sponsors Vertex Pharmaceuticals Inc. Principal Investigators Jennifer Taylor-Cousar, MD, MSCS + × Jennifer Taylor-Cousar, MD, MSCS Medical Director, Clinical Research Services Co-Director, Adult Cystic Fibrosis Program Director, Cystic Fibrosis Therapeutics Development Center Adult Program Professor Division of Pulmonary, Critical Care & Sleep Medicine Department of Medicine Division of Pediatric Pulmonary Medicine Department of Pediatrics President, Medical Staff View Full Profile Patient Rating 4.8 out of 5 stars Make an Appoinment Request More Information By completing this form, you agree to learn more about this study and see if you qualify. First Name:* Last Name:* Email Address:* Phone:* I would also like to receive periodic emails with health information, news and clinical research updates from National Jewish Health: YesNo
Trial Objective For many people living with cystic fibrosis, there are treatments available called cystic fibrosis transmembrane conductance regulator (CFTR) modulators that can help treat the underlying cause of cystic fibrosis. However, more than 5,000 people living with cystic fibrosis have a change in both of their CFTR genes that makes them not expected to benefit from CFTR modulators. The BEACON-CF clinical research study is exploring an investigational study drug called VX-522, a type of messenger RNA (mRNA) therapy, to see if it is safe and well tolerated for those who are not expected to benefit from CFTR modulators. mRNA therapy is designed to send a message that tells cells (the building blocks of the body) to produce normal proteins that can do the job of proteins that are faulty or missing. If the investigational study drug is effective, it will tell cells in the lungs to create normal CFTR protein, which may improve the way the lungs work in people with cystic fibrosis. VX-522 will be given as a single dose using a modified PARI nebulizer, an investigational medical device that turns liquid medications into a mist, which is then inhaled.