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

• Register: ClinicalTrials.gov
• Last refreshed on: 6 December 2021
• Main ID: NCT03512119
• Date of registration: 15/01/2018
• Prospective Registration: No
• Primary sponsor: University Hospital, Strasbourg, France
• Public title: Observational Study of Glucose Tolerance Abnormalities in Patient With Cystic Fibrosis Homozygous for Phe 508 Del CFTR Treated by Lumacaftor-Ivacaftor GLUCORRECTOR
• Scientific title: Observational Study of Glucose Tolerance Abnormalities in Patient With Cystic Fibrosis Homozygous for Phe 508 Del CFTR Treated by Lumacaftor-Ivacaftor
• Date of first enrolment: February 11, 2016
• Target sample size: 55
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03512119
• Study type: Observational

Countries of recruitment

France

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Glucose Intolerance or Newly Diagnosis Diabetes

Cystic Fibrosis Homozygous for Phe 508 Del CFTR

Intervention(s)

Drug: Lumacaftor-Ivacaftor treatment

Primary Outcome(s)

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 Outcome(s)

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]

C peptide and insulin values at T0, 1 , 2 hours of OGTT (µg/l) [Time Frame: Day 0 (traitement beginning) and year 1]

Duration in hyperglycemic area [for glucose value higher than 7.7 mmol/l, % time /24h] [Time Frame: Day 0 (traitement beginning) and year 1]

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]

FEV1, Vital Capacity (VC) (L and %) [Time Frame: Day 0 (traitement beginning) and year 1]

HbA1c (mmol/l and %) [Time Frame: Day 0 (traitement beginning) and year 1]

O2 saturation (%) [Time Frame: Day 0 (traitement beginning) and year 1]

Daily insulin doses (UI/day) [Time Frame: Day 0 (traitement beginning) and year 1]

Mean glucose value per day and 2 h after meal (mg/dl) [Time Frame: Day 0 (traitement beginning) and year 1]

Number hypoglycaemic events (below 3.3mmol/L, from midnight to 6 am) [Time Frame: Day 0 (traitement beginning) and year 1]

(BMI) body mass index [Time Frame: Day 0 (traitement beginning) and year 1]

Fasting and one hour glucose value of OGTT (mmol/l) [Time Frame: Day 0 (traitement beginning) and year 1]

Glucose, insulin and C peptide AUC of OGTT (µU/L) [Time Frame: Day 0 (traitement beginning) and year 1]

Variability glycemic indexes: MAGE (mg/dl), SD (mg/dl) [Time Frame: Day 0 (traitement beginning) and year 1]

Albumin and Pre albumin (g/l) [Time Frame: Day 0 (traitement beginning) and year 1]

HOMA -R , HOMA-S [Time Frame: Day 0 (traitement beginning) and year 1]

Weight (Kg) maximum weight never reached [Time Frame: Day 0 (traitement beginning) and year 1]

Secondary ID(s)

6403

Source(s) of Monetary Support

Please refer to primary and secondary sponsors