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NCT Number: NCT03512119

Observational Study of Glucose Tolerance Abnormalities in Patient With Cystic Fibrosis Homozygous for Phe 508 Del CFTR Treated by Lumacaftor-Ivacaftor

Cystic Fibrosis related diabetes (CFRD), a major factor of morbid-mortality in CF, is characterized by a preclinical phase of glucose intolerance particularly long reaching up to 10 years.

At the physiopathology level, insulin secretion is determinant in the glucose tolerance abnormalities in CF. Indeed insulin secretion is dependent of the CFTR activity at the beta cell surface and inhibition of CFTR leads to a decrease in insulin secretion.

Recently, the combination of the lumacaftor, a CFTR corrector, with Ivacaftor, a CFTR potentiator, was studied in patient with CF homozygous for the Phe508 del CFTR mutation patients and showed an improvement of the respiratory state in comparison with the placebo group.

These data suggests that lumacaftor in combination with ivacaftor in targeting CFTR action may have an early impact on the insulin-secretion and consequently on the glucose tolerance.

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Key information

Conditions

Cystic Fibrosis Homozygous for Phe 508 Del CFTR Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Digestive System Diseases Genetic Diseases, Inborn Glucose Intolerance Glucose Intolerance or Newly Diagnosis Diabetes Glucose Metabolism Disorders Hyperglycemia Infant, Newborn, Diseases Lung Diseases Metabolic Diseases Nutritional and Metabolic Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpitaux Universitaires de Strasbourg, Strasbourg, Alsace, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • patients with CF homozygous for the Phe508del CFTR mutation aged 12 years and over
  • Combined Lumacaftor-Ivacaftor treatment scheduled or already started
  • glucose intolerance in OGTT (ADA criteria) or newly diabetes diagnosed at the OGTT (ADA criteria) or diabetic patients with insulin requirement ≤ 0.3 unit / kg / day or without insulin treatment
  • signed informed consent of patient and of one parent OR legal representative for minor subject

Exclusion criteria

  • hypersensitivity to the active substances or to any of the excipients of Lumactfor -Ivacaftor
  • lung and/or liver transplant patient
  • Known diabetes with insulin treatment > 0.3 unit / kg / day
  • patient pregnant or wishing to pregnancy

Treatment and study plan

Lumacaftor-Ivacaftor treatment

Drug

Lumacaftor-Ivacaftor treatment during one year

Primary outcomes

  1. Measure of 2 hours plasma glucose value (mmol/l) of OGTT, change from baseline at one year of Lumacaftor-Ivacaftor treatment

    Time frame: Day 0 (traitement beginning) and year 1

Secondary outcomes

  1. Fasting and one hour glucose value of OGTT (mmol/l)

    Time frame: Day 0 (traitement beginning) and year 1

  2. C peptide and insulin values at T0, 1 , 2 hours of OGTT (µg/l)

    Time frame: Day 0 (traitement beginning) and year 1

  3. Glucose, insulin and C peptide AUC of OGTT (µU/L)

    Time frame: Day 0 (traitement beginning) and year 1

  4. HOMA -R , HOMA-S

    Time frame: Day 0 (traitement beginning) and year 1

  5. Mean glucose value per day and 2 h after meal (mg/dl)

    Time frame: Day 0 (traitement beginning) and year 1

  6. Duration in hypoglycemic area [hypo CGM = 2 consecutive values below 3.3 mmol/l - % of time spent]

    Time frame: Day 0 (traitement beginning) and year 1

  7. Duration in hyperglycemic area [for glucose value higher than 7.7 mmol/l, % time /24h]

    Time frame: Day 0 (traitement beginning) and year 1

    Number hypoglycaemic events (below 3.3mmol/L, from midnight to 6 am) Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)

  8. Number hypoglycaemic events (below 3.3mmol/L, from midnight to 6 am)

    Time frame: Day 0 (traitement beginning) and year 1

    Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)

  9. Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl)

    Time frame: Day 0 (traitement beginning) and year 1

  10. HbA1c (mmol/l and %)

    Time frame: Day 0 (traitement beginning) and year 1

  11. Daily insulin doses (UI/day)

    Time frame: Day 0 (traitement beginning) and year 1

  12. (BMI) body mass index

    Time frame: Day 0 (traitement beginning) and year 1

  13. Weight (Kg) maximum weight never reached

    Time frame: Day 0 (traitement beginning) and year 1

  14. Albumin and Pre albumin (g/l)

    Time frame: Day 0 (traitement beginning) and year 1

  15. FEV1, Vital Capacity (VC) (L and %)

    Time frame: Day 0 (traitement beginning) and year 1

  16. O2 saturation (%)

    Time frame: Day 0 (traitement beginning) and year 1

  17. Number of cures of antibiotics IV and per os /year and interval between 2 cures (week)

    Time frame: Day 0 (traitement beginning) and year 1

Sponsors and collaborators

Lead sponsor

University Hospital, Strasbourg, France

Other

Registry information

Acronym: GLUCORRECTOR

Important dates

Study start
2016
Primary completion
2019
Study completion
2019
First posted
Apr 30, 2018
Registry last updated
Nov 24, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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