NCT02653027

Brief Summary

The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor effects how people with cystic fibrosis respond to an oral glucose tolerance test, a test for diabetes.

Trial Health

15
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Timeline
Completed

Started Jan 2018

Shorter than P25 for not_applicable diabetes

Status
withdrawn

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

January 7, 2016

Completed
5 days until next milestone

First Posted

Study publicly available on registry

January 12, 2016

Completed
2 years until next milestone

Study Start

First participant enrolled

January 1, 2018

Completed
1 month until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2018

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2018

Completed
Last Updated

July 31, 2019

Status Verified

July 1, 2019

Enrollment Period

1 month

First QC Date

January 7, 2016

Last Update Submit

July 29, 2019

Conditions

DiabetesCystic Fibrosis

Keywords

Genetics

Outcome Measures

Primary Outcomes (2)

  • Change in Fasting Glucose

    This will be compared from baseline to 3 months after starting the medication

    3 months

  • Change in Fasting Glucose

    This will compare baseline to 6 months after starting the medication

    6 months

Secondary Outcomes (4)

  • Genetic risk score

    6 months

  • Pulmonary function test (PFT) FEV1 measurements

    6 months

  • Change in Fasting Insulin

    3 months

  • Change in Fasting Insulin

    6 months

Study Arms (1)

Lumacaftor Ivacaftor

EXPERIMENTAL

Subjects will get an OGTT before and after starting the combination therapy lumacaftor-ivacaftor.

Drug: Lumacaftor-ivacaftorOther: OGTT

Interventions

Lumacaftor-ivacaftorDRUG

Subjects who are planning on starting the combination therapy (lumacaftor-ivacaftor) will participate in OGTTs before and after starting the medication.

Also known as: Orkambi
Lumacaftor Ivacaftor
OGTTOTHER

A subject is given an oral glucose load and insulin and glucose measurements are taken at specified time periods.

Also known as: Oral Glucose tolerance test
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 CF, genotype homozygous PheDel508
  • Subject is planning on starting lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug\* OR subject is taking combination drug and had an OGTT done at a partners facility in the 6 months prior to initiating the drug.
  • Contraindications for taking drug include abnormal liver enzyme tests, renal dysfunction, pregnancy or nursing mothers

You may not qualify if:

  • Currently taking any medications for diabetes (including oral or injectable antihyperglycemic agents and/or insulin).
  • Had an admission for CF exacerbation less than 2 weeks prior to staring the medication. This will be defined as requiring new IV or PO antibiotics different than those used in maintenance therapy.
  • Is currently taking oral glucocorticoids or has been on oral or IV glucocorticoids in the past 2 weeks.

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

Diabetes MellitusCystic Fibrosis

Interventions

lumacaftor, ivacaftor drug combinationGlucose Tolerance Test

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Blood Chemical AnalysisClinical Chemistry TestsClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisDiagnostic Techniques, EndocrineInvestigative Techniques

Study Officials

  • Jose Florez, MD, PhD

    Massachusetts General Hospital

    PRINCIPAL INVESTIGATOR
0

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

January 7, 2016

First Posted

January 12, 2016

Study Start

January 1, 2018

Primary Completion

February 1, 2018

Study Completion

May 1, 2018

Last Updated

July 31, 2019

Record last verified: 2019-07