Muscle Accrual and Function in Cystic Fibrosis-Impact of Glucose Intolerance

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

54 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-05-31

Study Completion Date

2024-03-04

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Muscle Function and Physical Activity in the Modern Era of Cystic Fibrosis

NCT06251622

Cystic Fibrosis

RECRUITING

Nutritional, Metabolic and Respiratory Status in Cystic Fibrosis

NCT00476281

Cystic Fibrosis Diabetes

COMPLETED NA

Study of Metabolic Effects of Pregnancy in Women With Cystic Fibrosis

NCT00014768

Cystic Fibrosis

TERMINATED

BioEnergetics and Metabolomics in Cystic Fibrosis

NCT02225899

Cystic Fibrosis

COMPLETED

Strength and Muscle Related Outcomes for Nutrition and Lung Function in CF

NCT05639556

Cystic Fibrosis

RECRUITING

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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).

No interventions assigned to this group

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).

No interventions assigned to this group

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).

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Males or females age 16 to 22 years.
2. Confirmed diagnosis of CF per CF Foundation guidelines
3. Parental/guardian permission (informed consent) and if appropriate, child assent.

1. Age-, sex-, ethnicity-, and BMI-matched at time of enrollment to CF subjects without CFRD
2. Parental/guardian permission (informed consent) and if appropriate, child assent.

1. Males or females age 12 years or above.
2. Confirmed diagnosis of CF per CF Foundation guidelines.
3. 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.
4. Parental/guardian permission (informed consent) and if appropriate, child assent.

Exclusion Criteria

1. Chronic systemic glucocorticoid use e.g. for allergic bronchopulmonary aspergillosis
2. Organ transplantation
3. Forced Expiratory Volume (FFEV) 1%-predicted \< 40%
4. Established diagnosis of CFRD and treatment with insulin or hypoglycemic agent
5. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
6. Pregnancy or breastfeeding (if female)
7. 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.

1. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
2. Pregnancy or breastfeeding (if female)
3. Pre-existing neurological or neuromuscular disease

1. Chronic systemic glucocorticoid use e.g. for allergic bronchopulmonary aspergillosis
2. Organ transplantation
3. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
4. Pregnancy or breastfeeding (if female)
5. Pre-existing neurological or neuromuscular disease

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes