NCT02858843

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Aug 2016

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 5, 2016

Completed
27 days until next milestone

Study Start

First participant enrolled

August 1, 2016

Completed
7 days until next milestone

First Posted

Study publicly available on registry

August 8, 2016

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2018

Completed
Last Updated

May 21, 2018

Status Verified

May 1, 2018

Enrollment Period

1.7 years

First QC Date

July 5, 2016

Last Update Submit

May 17, 2018

Conditions

Keywords

genetics

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (2)

  • Change in glycemia contingent on genetic risk score

    1 year

  • Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements

    1 year

Study Arms (1)

Lumacaftor-ivacaftor

EXPERIMENTAL

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

Drug: lumacaftor-ivacaftor

Interventions

Drug given for cystic fibrosis

Also known as: Orkambi
Lumacaftor-ivacaftor

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Does not have a HgbA1c within 1 year prior to starting medication.
  • Has not been on the combination therapy for at least 2 months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (2)

  • 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.

    PMID: 25981758BACKGROUND
  • 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.

    PMID: 23952705BACKGROUND

MeSH Terms

Conditions

Cystic FibrosisDiabetes Mellitus

Interventions

lumacaftor, ivacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesGlucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System Diseases

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
SCREENING
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Chief of the Diabetes Unit, Associate Professor Harvard Medical School

Study Record Dates

First Submitted

July 5, 2016

First Posted

August 8, 2016

Study Start

August 1, 2016

Primary Completion

May 1, 2018

Study Completion

May 1, 2018

Last Updated

May 21, 2018

Record last verified: 2018-05