Clinical trial NCT02858843: The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia
Official title: A Study of the Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia in Persons With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Cystic Fibrosis
Diabetes
Keywords: genetics
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
First posted: 2016-08-08
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SCREENING

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in glycated hemoglobin (hemoglobin A1C)
timeFrame: 1 year
measure: Change in units of insulin used over a period of 6 months to 1 year.
timeFrame: 1 year
Secondary outcomes:
measure: Change in glycemia contingent on genetic risk score
timeFrame: 1 year
measure: Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements
timeFrame: 1 year
Other outcomes:
Locations:
Study officials:
References:
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.
citation: 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.