Cystic FibrosisNCT02776098
Muscle Accrual and Function in Cystic Fibrosis-Impact of Glucose Intolerance
An observational study of Cystic Fibrosis, run by Children's Hospital of Philadelphia at 2 sites in United States.
Trial summary
This study will investigate the link between glucose abnormalities and elements critical to muscle function including mass, composition and energy metabolism. the primary goal of the study is to determine whether Cystic Fibrosis (CF) disease is associated with muscle dysfunction, especially in the presence of glucose… Read more about this study
- Who is it for?
- Ages 12 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 54 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2016to 4 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 12 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: This is longitudinal cohort study of 3 main study groups: CF subjects without CFRD, Healthy matched controls and CF subjects with newly diagnosed CFRD started on insulin therapy. Healthy controls without CF will be recruited and will be matched for age, sex, ethnicity, and body mass index (BMI) to CF subjects. Subjects with newly diagnosed CFRD will also be enrolled. This study will be carried out over a 5-year period. CF subjects without CFRD (26) and healthy controls (26) will be followed annually for 2 years. CF subjects with newly diagnosed CFRD (10) will be followed for 6 months.
Who may be able to join
- Males or females age 16 to 22 years.
- Confirmed diagnosis of CF per CF Foundation guidelines
- Parental/guardian permission (informed consent) and if appropriate, child assent.
- Age-, sex-, ethnicity-, and BMI-matched at time of enrollment to CF subjects without CFRD
- Parental/guardian permission (informed consent) and if appropriate, child assent.
- Males or females age 12 years or above.
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- Confirmed diagnosis of CF per CF Foundation guidelines.
- New diagnosis of CFRD based on a) a clinically indicated Oral glucose tolerance test (OGTT) b) hyperglycemia (PG>200 mg/dL) persisting >48 hours and/or c) elevated HbA1C and within 4 weeks of starting insulin therapy.
- Parental/guardian permission (informed consent) and if appropriate, child assent.
Who cannot join
- Chronic systemic glucocorticoid use e.g. for allergic bronchopulmonary aspergillosis
- Organ transplantation
- Forced Expiratory Volume (FFEV) 1%-predicted < 40%
- Established diagnosis of CFRD and treatment with insulin or hypoglycemic agent
- Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
- Pregnancy or breastfeeding (if female)
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- Pre-existing neurological or neuromuscular disease
- All study visits for CF subjects will be scheduled during periods of baseline health. Visits will not be performed within 4 weeks of an acute respiratory illness or pulmonary exacerbation.
- Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
- Pregnancy or breastfeeding (if female)
- Pre-existing neurological or neuromuscular disease
- Chronic systemic glucocorticoid use e.g. for allergic bronchopulmonary aspergillosis
- Organ transplantation
- Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
- Pregnancy or breastfeeding (if female)
- Pre-existing neurological or neuromuscular disease
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
This study will investigate the link between glucose abnormalities and elements critical to muscle function including mass, composition and energy metabolism. the primary goal of the study is to determine whether Cystic Fibrosis (CF) disease is associated with muscle dysfunction, especially in the presence of glucose intolerance. This is a longitudinal cohort study of 3 main groups: CF subjects without Cystic Fibrosis-related diabetes (CFRD), healthy matched controls and CF subjects with newly diagnosed CFRD started on insulin therapy.
Read the detailed description
Cystic Fibrosis (CF) is a lethal inherited disease that primarily affects the lungs but also confers a high risk of diabetes, with up to 40-50% of adults experiencing Cystic Fibrosis-related diabetes (CFRD). CFRD is associated with an accelerated decline in lung function, nutritional status and survival and despite treatment mortality in patients with CFRD remains high. Airway inflammation and susceptibility to infections caused by hyperglycemia, and the catabolic effect of insulin deficiency are posited mechanisms of CFRD-associated morbidity. Respiratory failure caused by airway disease is well known but the contribution of respiratory muscle dysfunction may be critical. In Type 2 Diabetes Mellitus (T2DM) glucose and insulin defects are closely correlated with muscle function. The pulmonary muscles are crucial to respiration and airway clearance in CF. Muscle function is dependent on its mass, composition, and energy metabolism. Lean body mass (LBM) deficits are present in CF and improvement in LBM improves pulmonary function. Using T2DM as a model for muscle dysfunction, the study hypothesis is that glucose intolerance exacerbates LBM deficits, negatively affects muscle composition, and alters muscle metabolism leading to respiratory muscle dysfunction and a decline in pulmonary function.
CF subjects without CFRD and healthy controls will undergo 3 study visits (baseline then annually for 2 years) and CFRD subjects will undergo 2 study visits (baseline and 6 months after baseline). Evaluations will include neurologic exams, anthropometric assessments, 3-day dietary recall, MRI scans, oral glucose tolerance tests (CF subjects only), blood work, pulmonary function testing, muscle strength testing, exercise testing, bone density scans, and adverse event assessment.
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Glucose Intolerance, Muscle Accrual, Cystic Fibrosis-related Diabetes, Healthy Controls, Healthy Participants
- Medical subject headings
- Cystic Fibrosis, Glucose Intolerance
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cystic Fibrosis without Cystic Fibrosis-related Diabetes | Subjects with a confirmed diagnosis of Cystic Fibrosis (CF) without Cystic Fibrosis-related diabetes will be followed annually for 2 years for a total of four study visits over 2 years (screening, baseline, 12 and 24 month visits). | |
| Newly Diagnosed Cystic Fibrosis-Related Diabetes | Subjects with a confirmed diagnosis of Cystic Fibrosis (CF) and new diagnosis of Cystic Fibrosis-Related Diabetes (CFRD) will be followed for a total of 3 study visits over 6 months (screening, baseline and 6 months). | |
| Healthy Controls | Age, sex, ethnicity and body mass index matched (at time of enrollment to CF without CFRD subjects) healthy controls will be followed annually for 2 years for a total of four study visits (screening, baseline, 12 and 24 month visits). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare change in mean lean body mass (LBM) from baseline to end of study
Measured: 24 months (CF without CFRD subjects & healthy controls); 6 months for CF subjects
Secondary outcomes (1)
Compare Intramyocellular lipid (IMCL) accumulation from baseline to end of study
Proton magnetic resonance spectroscopy will be used to measure IMCL per previously published methods.
Measured: 24 months (CF without CFRD subjects & healthy controls); 6 months for CF subjects
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2016 (estimated)
- Study startMay 2016
- Primary completion4 Mar 2024
- Study completion4 Mar 2024
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 Children's Hospital of PhiladelphiaThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), University of Pennsylvania, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Responsible party
- Sponsor
- Investigators
- Clement L Ren, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02776098
- Sponsor's protocol ID
- 15-012279
- Other IDs
- 1K23DK107937-01
- Status last verified
- Mar 2024
- First submitted
- 16 May 2016
- Last updated
- 7 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Muscle Accrual and Function in Cystic Fibrosis-Impact of Glucose Intolerance
- ClinicalTrials.gov ID
- NCT02776098
- Acronym / sponsor ID
- 15-012279
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Glucose Intolerance, Muscle Accrual, Cystic Fibrosis-related Diabetes, Healthy Controls, Healthy Participants
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 54 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), University of Pennsylvania, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- May 2016
- Primary completion
- 4 Mar 2024 (actual)
- Study completion
- 4 Mar 2024 (actual)
- First posted
- 18 May 2016 (estimated)
- 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.
This study will investigate the link between glucose abnormalities and elements critical to muscle function including mass, composition and energy metabolism. the primary goal of the study is to determine whether Cystic Fibrosis (CF) disease is associated with muscle dysfunction, especially in the presence of glucose intolerance. This is a longitudinal cohort study of 3 main groups: CF subjects without Cystic Fibrosis-related diabetes (CFRD), healthy matched controls and CF subjects with newly diagnosed CFRD started on insulin therapy.
Detailed description
Cystic Fibrosis (CF) is a lethal inherited disease that primarily affects the lungs but also confers a high risk of diabetes, with up to 40-50% of adults experiencing Cystic Fibrosis-related diabetes (CFRD). CFRD is associated with an accelerated decline in lung function, nutritional status and survival and despite treatment mortality in patients with CFRD remains high. Airway inflammation and susceptibility to infections caused by hyperglycemia, and the catabolic effect of insulin deficiency are posited mechanisms of CFRD-associated morbidity. Respiratory failure caused by airway disease is well known but the contribution of respiratory muscle dysfunction may be critical. In Type 2 Diabetes Mellitus (T2DM) glucose and insulin defects are closely correlated with muscle function. The pulmonary muscles are crucial to respiration and airway clearance in CF. Muscle function is dependent on its mass, composition, and energy metabolism. Lean body mass (LBM) deficits are present in CF and improvement in LBM improves pulmonary function. Using T2DM as a model for muscle dysfunction, the study hypothesis is that glucose intolerance exacerbates LBM deficits, negatively affects muscle composition, and alters muscle metabolism leading to respiratory muscle dysfunction and a decline in pulmonary function.
CF subjects without CFRD and healthy controls will undergo 3 study visits (baseline then annually for 2 years) and CFRD subjects will undergo 2 study visits (baseline and 6 months after baseline). Evaluations will include neurologic exams, anthropometric assessments, 3-day dietary recall, MRI scans, oral glucose tolerance tests (CF subjects only), blood work, pulmonary function testing, muscle strength testing, exercise testing, bone density scans, and adverse event assessment.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cystic Fibrosis without Cystic Fibrosis-related Diabetes | – | –Subjects with a confirmed diagnosis of Cystic Fibrosis (CF) without Cystic Fibrosis-related diabetes will be followed annually for 2 years for a total of four study visits over 2 years (screening, baseline, 12 and 24 month visits). |
| Newly Diagnosed Cystic Fibrosis-Related Diabetes | – | –Subjects with a confirmed diagnosis of Cystic Fibrosis (CF) and new diagnosis of Cystic Fibrosis-Related Diabetes (CFRD) will be followed for a total of 3 study visits over 6 months (screening, baseline and 6 months). |
| Healthy Controls | – | –Age, sex, ethnicity and body mass index matched (at time of enrollment to CF without CFRD subjects) healthy controls will be followed annually for 2 years for a total of four study visits (screening, baseline, 12 and 24 month visits). |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This is longitudinal cohort study of 3 main study groups: CF subjects without CFRD, Healthy matched controls and CF subjects with newly diagnosed CFRD started on insulin therapy. Healthy controls without CF will be recruited and will be matched for age, sex, ethnicity, and body mass index (BMI) to CF subjects. Subjects with newly diagnosed CFRD will also be enrolled. This study will be carried out over a 5-year period. CF subjects without CFRD (26) and healthy controls (26) will be followed annually for 2 years. CF subjects with newly diagnosed CFRD (10) will be followed for 6 months.
Key inclusion criteria (9 listed)
- Males or females age 16 to 22 years.
- Confirmed diagnosis of CF per CF Foundation guidelines
- Parental/guardian permission (informed consent) and if appropriate, child assent.
- Age-, sex-, ethnicity-, and BMI-matched at time of enrollment to CF subjects without CFRD
- Parental/guardian permission (informed consent) and if appropriate, child assent.
Key exclusion criteria (16 listed)
- Chronic systemic glucocorticoid use e.g. for allergic bronchopulmonary aspergillosis
- Organ transplantation
- Forced Expiratory Volume (FFEV) 1%-predicted < 40%
- Established diagnosis of CFRD and treatment with insulin or hypoglycemic agent
- Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Compare change in mean lean body mass (LBM) from baseline to end of study
Time frame: 24 months (CF without CFRD subjects & healthy controls); 6 months for CF subjects
1 secondary outcome
Compare Intramyocellular lipid (IMCL) accumulation from baseline to end of study
Time frame: 24 months (CF without CFRD subjects & healthy controls); 6 months for CF subjects
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2024.
- 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.
Clement L Ren, MD principal investigator
Children's Hospital of Philadelphia
2 sites 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, run by Children's Hospital of Philadelphia at 2 sites 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 9 inclusion and 16 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: finished, as of the sponsor's last update on 7 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Compare change in mean lean body mass (LBM) from baseline to end of study (time frame: 24 months (CF without CFRD subjects & healthy controls); 6 months for CF subjects). 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 7 Mar 2024.