CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis

NCT04206436 | Active Not Recruiting DMC

• 1 other identifier: 1908566174
• Study Type: observational
• Target: 63
• Locations: 1 country
• Sites: 1

Brief Summary

Study is looking at the effects of cystic fibrosis treatment on bone muscle.

Conditions

Cystic Fibrosis; Bone Loss; Muscle Loss

Outcome Measures

Primary Outcomes (2)

• Identify the effects of CFTR modulators on bone mineral density

  Changes from baseline to 12 month in total volumetric BMD and in estimated failure load as measured by HRpQCT at the distal radius and tibia

  12 months

• establish the effect of CFTR modulators on sarcopenia.

  changes from baseline to 12 months in whole body lean mass using body composition software on DXA

  12 months

Secondary Outcomes (2)

• Identify the effect of CFTR modulators on bone mineral density

  6, 12 and 24 month

• establish the effect of CFTR modulators on sarcopenia.

  6, 12 and 24 month

Study Arms (2)

on CFTR

For patients on or near time of initiation CFTR modulator therapy

Drug: Cftr Modulators

Controls

controls will be patients not eligible for available treatment

Interventions

Cftr Modulators DRUG

CFTR modulators are drugs used to treat cystic fibrosis

on CFTR

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Enrollment will target groups that are frequency matched for age (decade), race, sex, pancreatic insufficiency and glucocorticoid usage (inhaled or systemic as both have skeletal effects in some studies) to control for these factors influencing relevant bone and muscle outcomes. (see statistical description). The study will enroll up to 30 subjects in each group, expecting up to 25% dropout.

You may qualify if:

• documented, confirmed diagnosis of CF
• Age ≥18 years old
• \>21 days since the start of their last pulmonary exacerbation at the baseline visit
• Provide signed written informed consent to participate

You may not qualify if:

• Estimated glomerular filtration rate (eGFR) \<30 ml/min/m2 using the CKD-EPI equation,
• Treatment with any osteoporosis medication within 6 months for oral agents or 1 year for intravenous or injectable agents (Subjects may participate if therapy stopped earlier than these time periods).
• Current treatment with growth hormone or IGF-1
• Currently pregnant or lactating or planning plan on becoming pregnant during the duration of the study.
• Life expectancy less than 12 months
• History of lung transplantation
• Conditions that in the opinion of the investigators would interfere with the ability to collect or interpret the data, or put the patient at higher safety risk from study procedures.

Sponsors & Collaborators

• Indiana University: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (1)

Indiana University

Indianapolis, Indiana, 46202-5149, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Samples kept for batch processing at end of study. Samples may also be kept and de-identified and kept for additional testing

MeSH Terms

Conditions

Cystic Fibrosis; Bone Diseases, Metabolic; Muscular Atrophy

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Atrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Study Officials

• Erik A Imel, MD

  Indiana University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: December 18, 2019
• First Posted: December 20, 2019
• Study Start: December 18, 2019
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: October 2, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)