Cystic FibrosisNCT04206436
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
An observational study of Cystic Fibrosis and Bone Loss, run by Indiana University at 1 site in United States.
Trial summary
Study is looking at the effects of cystic fibrosis treatment on bone muscle. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, Bone Loss, Muscle Loss
- What is being tested?
- Cftr Modulators
- How many people
- 63 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2019to 31 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- • 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana University
Indianapolis, Indiana, 46202-5149
About this study
Study is looking at the effects of cystic fibrosis treatment on bone muscle.
Read the detailed description
Cystic fibrosis (CF) is a complex multisystem genetic disease, with pulmonary and gastrointestinal consequences dominating the clinical picture. The life-expectancy of CF patients has increased through several therapeutic advances. Although respiratory failure remains the major cause of mortality in CF, musculoskeletal impairments contribute to major morbidity. In the general population, musculoskeletal conditions are among the most common reasons for seeking medical care, and the risk of osteoporotic fracture increases with age. As the CF population ages, the morbidity related to musculoskeletal effects may increase.
The etiology of CF related bone disease is multifactorial and includes effects of pancreatic insufficiency, poor nutritional status, vitamin D deficiency, glucocorticoid treatment, inflammation, hypogonadism, and sarcopenia, collectively resulting in attenuated bone mineral accrual and low bone density The effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulating drugs on bone disease in CF has not been evaluated. Effects of CFTR modulators may help counter the bone and muscle consequences of CF either directly by effects on bone or muscle cells, or indirectly by improved lung disease, improved nutritional status, decreased systemic inflammation or glucocorticoid use, or subsequent increases in physical activity.
The rationale that underlies the proposed research is that better understanding of the bone and muscle effects of CFTR modulator therapies will help guide strategies to optimize bone accrual, prevent osteoporosis and fractures, and improve functional outcomes in the aging CF population. Set on the backbone of a longitudinal observational cohort study, the study will systematically and comprehensively evaluate changes in bone and muscle mass and strength from baseline to 12 month and 24 month time points among patients receiving CFTR modulator therapies and also among controls not receiving CFTR modulator therapies.
- Conditions
- Cystic Fibrosis, Bone Loss, Muscle Loss
- Being tested
- Cftr Modulators
- Medical subject headings
- Cystic Fibrosis, Bone Diseases, Metabolic, Muscular Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cftr ModulatorsDrug
CFTR modulators are drugs used to treat cystic fibrosis
| Study group | Role | What they receive |
|---|---|---|
| on CFTR | For patients on or near time of initiation CFTR modulator therapy Receives: Drug: Cftr Modulators | |
| Controls | controls will be patients not eligible for available treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 12 months
Secondary outcomes (2)
Identify the effect of CFTR modulators on bone mineral density
Changes from baseline to 24 month in total volumetric BMD and in estimated failure load as measured by HRpQCT at the distal radius and tibia.
Changes from baseline to 6, 12, and 24 month in HRpQCT measure of cortical vBMD and trabecular vBMD at both the distal radius and tibia and cortical vBMD and trabecular vBMD at diaphyseal site.
Changes from baseline to 6, 12 and 24 month in HRpQCT measure of cortical vBMC and trabecular vBMD at both the distal radius and tibia and cortical vBMD at a diaphyseal site Changes from baseline to 6,12, and 24 month in areal BMD using DXA at eh lumbar spine, total body, femur neck and total hip
Measured: 6, 12 and 24 month
establish the effect of CFTR modulators on sarcopenia.
changes from baseline to 6 and 24 months in whole body lean mass using body composition software on DXA Changes from baseline to 6, 12, 24 months in appendicular lean mass relative to height measured using body composition software on DXA
Measured: 6, 12 and 24 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2019
- Study start18 Dec 2019
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, Erik Imel, Indiana University
- Investigators
- Erik A Imel, MD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04206436
- Sponsor's protocol ID
- 1908566174
- Status last verified
- Nov 2026
- First submitted
- 18 Dec 2019
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT04206436
- Acronym / sponsor ID
- 1908566174
- Conditions
- Cystic Fibrosis, Bone Loss, Muscle Loss
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 18 Dec 2019 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 20 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study is looking at the effects of cystic fibrosis treatment on bone muscle.
Detailed description
Cystic fibrosis (CF) is a complex multisystem genetic disease, with pulmonary and gastrointestinal consequences dominating the clinical picture. The life-expectancy of CF patients has increased through several therapeutic advances. Although respiratory failure remains the major cause of mortality in CF, musculoskeletal impairments contribute to major morbidity. In the general population, musculoskeletal conditions are among the most common reasons for seeking medical care, and the risk of osteoporotic fracture increases with age. As the CF population ages, the morbidity related to musculoskeletal effects may increase.
The etiology of CF related bone disease is multifactorial and includes effects of pancreatic insufficiency, poor nutritional status, vitamin D deficiency, glucocorticoid treatment, inflammation, hypogonadism, and sarcopenia, collectively resulting in attenuated bone mineral accrual and low bone density The effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulating drugs on bone disease in CF has not been evaluated. Effects of CFTR modulators may help counter the bone and muscle consequences of CF either directly by effects on bone or muscle cells, or indirectly by improved lung disease, improved nutritional status, decreased systemic inflammation or glucocorticoid use, or subsequent increases in physical activity.
The rationale that underlies the proposed research is that better understanding of the bone and muscle effects of CFTR modulator therapies will help guide strategies to optimize bone accrual, prevent osteoporosis and fractures, and improve functional outcomes in the aging CF population. Set on the backbone of a longitudinal observational cohort study, the study will systematically and comprehensively evaluate changes in bone and muscle mass and strength from baseline to 12 month and 24 month time points among patients receiving CFTR modulator therapies and also among controls not receiving CFTR modulator therapies.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| on CFTR | – | Drug: Cftr ModulatorsFor patients on or near time of initiation CFTR modulator therapy |
| Controls | – | –controls will be patients not eligible for available treatment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- documented, confirmed diagnosis of CF
Key exclusion criteria (7 listed)
- • Estimated glomerular filtration rate (eGFR) <30 ml/min/m2 using the CKD-EPI equation,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cftr ModulatorsCFTR modulators are drugs used to treat cystic fibrosis | Drug | on CFTR |
Primary outcome
Identify the effects of CFTR modulators on bone mineral density
Time frame: 12 months
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
establish the effect of CFTR modulators on sarcopenia.
Time frame: 12 months
changes from baseline to 12 months in whole body lean mass using body composition software on DXA
2 secondary outcomes
Identify the effect of CFTR modulators on bone mineral density
Time frame: 6, 12 and 24 month
establish the effect of CFTR modulators on sarcopenia.
Time frame: 6, 12 and 24 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Erik A Imel, MD principal investigator
Indiana University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis and Bone Loss, run by Indiana University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identify the effects of CFTR modulators on bone mineral density (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.