The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia

Not Applicable

Terminated

• Conditions: Diabetes; Cystic Fibrosis
• Interventions: Drug: lumacaftor-ivacaftor

• Registration Number: NCT02858843
• Lead Sponsor: Massachusetts General Hospital

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.

Study Timeline

• Study First Submitted: 2016-07-05
• Study Start: 2016-08-01
• Study First Submitted QC: 2016-08-03
• Study First Posted: 2016-08-08
• Status Verified: 2018-05
• Primary Completion: 2018-05-01
• Study Completion: 2018-05-01
• Last Update Submitted: 2018-05-17
• Last Update Posted: 2018-05-21

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 1

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

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Lumacaftor-ivacaftor	lumacaftor-ivacaftor	Subjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor.

Primary Outcome Measures

Name	Time	Method
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
Change in glycated hemoglobin (hemoglobin A1C)	1 year	A blood test will be used to determine the hemoglobin A1c change while on the medication.

Secondary Outcome Measures

Name	Time	Method
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
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.