Markers of Osteoporosis in Cystic Fibrosis

Study Overview
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Brief Summary
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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 Approximately 10 adult subjects with CF who participated in the main study and have results indicating bone disease will receive treatment with Denosumab for up to 5 years. They will be asked to return annually for repeat DEXA scans, micro CT, and blood collection.

Official Title
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Markers of Osteoporosis in Cystic Fibrosis

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: Denosumab


Participation Criteria
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Eligibility Criteria
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Cystic Fibrosis Main Study Inclusion Criteria: Must have CF diagnosis confirmed by sweat test or genotype analysis Subjects (and parents/legal guardians as applicable) must have the ability to read and write in English Sub-study Exclusion Criteria: No CF diagnosis Men or women without osteoporosis Less than 18 years of age Unwilling to return annually for study visits for up to 5 years Unwilling and/or medically unable to take denosumab

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 64 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| No Intervention: Main Study<br>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 (if not completed as standard of care within 6 months prior to research visit), micro CT, and blood collection. | |
| Experimental: Denosomab Sub-study<br>Approximately 10 subjects with CF that have completed the main study and have results which indicate bone disease are eligible to participate in the sub study. Subjects who consent will receive treatment with denosumab (60 mg/ml via subcutaneous injection in the upper arm, upper thigh, or abdomen every 6 months) for up to 5 years. These subjects will be asked to return every 6 months for injections and annually (+/- 6 months) for up to 5 years for a DEXA scan, micro CT, and blood collection. | Drug: Denosumab<br>* treatment with denosumab every 6 months for up to 5 years<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| DEXA results | Z and/or T scores | Looking at single timepoints and well as changes for up to 5 years for those in the sub-study | 
| Micro CT results | Trabecular bone volume to tissue volume (BV/TV), cortical and trabecular thickness (Tb.Th, microns), trabecular spacing (Tb.Sp; microns), and structure model index (SMI; index of trabecular rod vs plate-like shape/ stoutness) will be assessed. Change in BV/TV and cortical thickness will be taken to indicate increased/decreased bone mass accrual by microCT. | Looking at single timepoints and well as changes for up to 5 years for those in the sub-study |