The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia

Diabetes Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02858843
• Other study ID #: 2016P000959
• Status: Terminated
• Phase: N/A
• Start date: August 1, 2016
• Est. completion date: May 1, 2018

Study information

• Verified date: May 2018
• Source: Massachusetts General Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: May 1, 2018
• Est. primary completion date: May 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Diabetes
• Fibrosis
• Hyperglycemia

Intervention

Drug:
• lumacaftor-ivacaftor
Drug given for cystic fibrosis

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Massachusetts General Hospital

References & Publications (2)

Bellin MD, Laguna T, Leschyshyn J, Regelmann W, Dunitz J, Billings J, Moran A. Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study. Pediatr Diabetes. 2013 Sep;14(6):417-21. doi: 10.1111/pedi.12026. Epub 2013 Mar 13. — View Citation

Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in glycated hemoglobin (hemoglobin A1C)	A blood test will be used to determine the hemoglobin A1c change while on the medication.	1 year
Primary	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	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
Secondary	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