Targeting the RANK/RANKL/OPG pathway for treatment of osteoporosis in Cystic Fibrosis
Study ID: STU 052018-007
Main Study up to 100 subjects, both non-CF volunteers and Cystic Fibrosis (CF) patients, will participate in a single study visit that will include a DeXa scan, micro CT, and blood collection. Denosumab (Prolia) Sub study This is a single center open label pilot study to determine safety and benefit of use of denosumab (Prolia) in individuals with CF who have osteoporosis. The aim is to determine if bone density as measured by DeXa improves over 1 year period of time after treatment. approximately 10 adult subjects with CF who participated in the main study and have results indicating bone disease, who will be prescribed denosumab (Prolia) either through standard of care or research will participate in the sub study. Denosumab (Prolia) will be administered subcutaneously 1 time every 6 months for a total of 2 doses.
Criteria for Inclusion of Subjects: Main Study Healthy Volunteer Criteria * Males and females * Must be greater than or equal to 12 years of age. * All subjects must understand and sign the informed consent/assent. * Subjects (and parents/legal guardians as applicable) must have the ability to read and write in English. Main Study Cystic Fibrosis Patient Criteria * Males and females * Must be greater than or equal to 12 years of age. * Must have CF diagnosis confirmed by sweat test or genotype analysis * All subjects must understand and sign the informed consent/assent. * Subjects (and parents/legal guardians as applicable) must have the ability to read and write in English. Denosumab Sub study Criteria Must have CF diagnosis Men or women with osteoporosis and a bone density (DXA) with any Z or T score of < - 2.0 Must be greater than or equal to 18 years of age Participated in main study and received results that indicate bone disease. Willing to return annually for study visits and phone calls Willing to take denosumab (Prolia) per protocol. Willing to adhere to a highly effective method of contraception Patients eligible for Trikafta must agree to not start this drug while on this study or be stable on this medication for a minimum of 90 days before enrollment in this study
The research objectives of this pilot study are to determine: * what markers best identify bone disease * to determine safety and efficacy of use of denosumab (Prolia) in treating osteoporosis in patients with cystic fibrosis