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The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia

Primary Purpose

Cystic Fibrosis, Diabetes

Status
Terminated
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
lumacaftor-ivacaftor
Sponsored by
Massachusetts General Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional screening trial for Cystic Fibrosis focused on measuring genetics

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Lumacaftor-ivacaftor

    Arm Description

    Subjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor.

    Outcomes

    Primary Outcome Measures

    Change in glycated hemoglobin (hemoglobin A1C)
    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.
    Using chart review, the change in insulin units used per day will be calculated

    Secondary Outcome Measures

    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.
    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

    Full Information

    First Posted
    July 5, 2016
    Last Updated
    May 17, 2018
    Sponsor
    Massachusetts General Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02858843
    Brief Title
    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

    2. Study Status

    Record Verification Date
    May 2018
    Overall Recruitment Status
    Terminated
    Why Stopped
    We were unable to enroll eligible subjects for the study.
    Study Start Date
    August 1, 2016 (undefined)
    Primary Completion Date
    May 1, 2018 (Actual)
    Study Completion Date
    May 1, 2018 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Massachusetts General Hospital

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystic Fibrosis, Diabetes
    Keywords
    genetics

    7. Study Design

    Primary Purpose
    Screening
    Study Phase
    Not Applicable
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    1 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Lumacaftor-ivacaftor
    Arm Type
    Experimental
    Arm Description
    Subjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor.
    Intervention Type
    Drug
    Intervention Name(s)
    lumacaftor-ivacaftor
    Other Intervention Name(s)
    Orkambi
    Intervention Description
    Drug given for cystic fibrosis
    Primary Outcome Measure Information:
    Title
    Change in glycated hemoglobin (hemoglobin A1C)
    Description
    A blood test will be used to determine the hemoglobin A1c change while on the medication.
    Time Frame
    1 year
    Title
    Change in units of insulin used over a period of 6 months to 1 year.
    Description
    Using chart review, the change in insulin units used per day will be calculated
    Time Frame
    1 year
    Secondary Outcome Measure Information:
    Title
    Change in glycemia contingent on genetic risk score
    Description
    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.
    Time Frame
    1 year
    Title
    Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements
    Description
    The investigators will compare how PFT measurement of FEV1 are related to changes in glycemia
    Time Frame
    1 year

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided
    Citations:
    PubMed Identifier
    25981758
    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.
    Results Reference
    background
    PubMed Identifier
    23952705
    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.
    Results Reference
    background

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    The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia

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