Cystic Fibrosis, Diabetes
Conditions
Keywords
genetics
Brief summary
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.
Detailed description
This is a single center, open label study. Patients will have 1 visits at the Diabetes Research Center (DRC), or Clinical Research Center (CRC). The participants will have been previously screened to make sure they are candidates for the study. These patients will be contacted prior to their first visit to discuss enrollment in the study. At the study visit the participant will come to the CRC or DRC for a research visit. The following will occur at this study visit: informed consent; brief medical history; weight and height; vital signs and blood pressure; blood draw for DNA extraction, A1c and an extra research tube for storage. This will be scheduled at a time that is convenient to the patient.
Interventions
Drug given for cystic fibrosis
Sponsors
Massachusetts General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SCREENING
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Age 18 years old or greater 2. Patients diagnosed with cystic fibrosis (CF), genotype homozygous Phe508del 3. Subject has been started on lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug\* \* Contraindications for taking drug include abnormal liver enzyme tests, renal dysfunction, pregnancy or nursing mothers
Exclusion criteria
1. Does not have a HgbA1c within 1 year prior to starting medication. 2. Has not been on the combination therapy for at least 2 months
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in glycated hemoglobin (hemoglobin A1C) | 1 year | A blood test will be used to determine the hemoglobin A1c change while on the medication. |
| Change in units of insulin used over a period of 6 months to 1 year. | 1 year | Using chart review, the change in insulin units used per day will be calculated |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in glycemia contingent on genetic risk score | 1 year | The investigators will examine how change in glycemia is dependent on genotype at variants associated with type 2 diabetes and insulin secretion using genetic risk scores. |
| Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements | 1 year | The investigators will compare how PFT measurement of FEV1 are related to changes in glycemia |
Outcome results
None listed