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

NCT02776098 | Completed

• 2 other identifiers: 15-0122791K23DK107937-01
• Study Type: observational
• Target: 54
• Locations: 1 country
• Sites: 2

Brief 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 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.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; Glucose Intolerance; Muscle Accrual; Cystic Fibrosis-related Diabetes; Healthy Controls; Healthy Participants

Outcome Measures

Primary Outcomes (1)

• Compare change in mean lean body mass (LBM) from baseline to end of study

  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

  24 months (CF without CFRD subjects & healthy controls); 6 months for CF subjects

Study Arms (3)

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).

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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)
• 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.
• 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.
• 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
• Males or females age 12 years or above.
• Confirmed diagnosis of CF per CF Foundation guidelines.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• National Institutes of Health (NIH): collaborator
• University of Pennsylvania: collaborator
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (2)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis; Glucose Intolerance

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; Hyperglycemia; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Clement L Ren, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2016
• First Posted: May 18, 2016
• Study Start: May 1, 2016
• Primary Completion: March 4, 2024
• Study Completion: March 4, 2024
• Last Updated: March 7, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)