Markers of Osteoporosis in Cystic Fibrosis

• ClinicalTrials.gov Identifier: NCT03921060
• Recruitment Status: Recruiting
• First Posted: April 19, 2019
• Last Update Posted: April 14, 2022
• Sponsor: University of Texas Southwestern Medical Center
• Information provided by (Responsible Party): Raksha Jain, University of Texas Southwestern Medical Center

Tracking Information

• First Submitted Date: March 26, 2019
• First Posted Date: April 19, 2019
• Last Update Posted Date: April 14, 2022
• Actual Study Start Date: September 2, 2021
• Estimated Primary Completion Date: July 2027 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 16, 2019)

• DEXA results [ Time Frame: Looking at single timepoints and well as changes for up to 5 years for those in the sub-study ]
  Z and/or T scores
• Micro CT results [ Time Frame: Looking at single timepoints and well as changes for up to 5 years for those in the sub-study ]
  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.

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Markers of Osteoporosis in Cystic Fibrosis
• Official Title: Markers of Osteoporosis in Cystic Fibrosis

Brief Summary

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Diagnostic
• Condition: Cystic Fibrosis

Intervention

Drug: Denosumab

treatment with denosumab every 6 months for up to 5 years

Study Arms

• No Intervention: 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 (if not completed as standard of care within 6 months prior to research visit), micro CT, and blood collection.
• Experimental: Denosomab Sub-study
  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.
  Intervention: Drug: Denosumab

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: April 16, 2019): 100
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: July 2027
• Estimated Primary Completion Date: July 2027 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 64 Years (Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: Ashley Keller; 2146482817; ashley.keller@utsouthwestern.edu

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03921060
• Other Study ID Numbers: STU 052018-007
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Raksha Jain, University of Texas Southwestern Medical Center
• Original Responsible Party: University of Texas Southwestern Medical Center
• Current Study Sponsor: University of Texas Southwestern Medical Center
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: University of Texas Southwestern Medical Center
• Verification Date: April 2022