The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Study of the Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia in Persons With Cystic Fibrosis

Conditions
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Cystic Fibrosis, Diabetes

Intervention / Treatment
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* Drug: lumacaftor-ivacaftor


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 18 years old or greater Patients diagnosed with cystic fibrosis (CF), genotype homozygous Phe508del 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 Does not have a HgbA1c within 1 year prior to starting medication. Has not been on the combination therapy for at least 2 months

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Screening
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Lumacaftor-ivacaftor<br>Subjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor. | Drug: lumacaftor-ivacaftor<br>* Drug given for cystic fibrosis<br>* Other names: Orkambi;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in glycated hemoglobin (hemoglobin A1C) | A blood test will be used to determine the hemoglobin A1c change while on the medication. | 1 year | 
| Change in units of insulin used over a period of 6 months to 1 year. | Using chart review, the change in insulin units used per day will be calculated | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in glycemia contingent on genetic risk score | 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. | 1 year | 
| Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements | The investigators will compare how PFT measurement of FEV1 are related to changes in glycemia | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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genetics