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

NCT ID: NCT03512119

Last Updated: 2021-11-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

55 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-02-11

Study Completion Date

2019-04-10

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis Homozygous for Phe 508 Del CFTR Glucose Intolerance or Newly Diagnosis Diabetes

Keywords

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Cystic fibrosis Glucose tolerance abnormalities CFTR corrector and potentiator

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Patient

Patient with cystic fibrosis homozygous for Phe 508 del CFTR having a glucose intolerance or newly diagnosis diabetes

Lumacaftor-Ivacaftor treatment

Intervention Type DRUG

Lumacaftor-Ivacaftor treatment during one year

Interventions

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Lumacaftor-Ivacaftor treatment

Lumacaftor-Ivacaftor treatment during one year

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Strasbourg, France

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Hôpitaux Universitaires de Strasbourg

Strasbourg, Alsace, France

Site Status

Centre Hospitalier Universitaire d'Angers

Angers, , France

Site Status

Hôpital Renée Sabran

Giens, , France

Site Status

Centre Hospitalier Lyon Sud

Lyon, , France

Site Status

Hôpital NORD - Assistance Publique Hôpitaux de Marseille

Marseille, , France

Site Status

Hôpital Arnaud de Villeneuve

Montpellier, , France

Site Status

Hôpital Robert Debré

Paris, , France

Site Status

American Memorial Hospital

Reims, , France

Site Status

Clinique "Mucoviscidose" Presqu'île de Perharidy

Roscoff, , France

Site Status

Hôpital Charles Nicolle

Rouen, , France

Site Status

Hôpital FOCH

Suresnes, , France

Site Status

Hôpital Bretonneau - CHRU de Tours

Tours, , France

Site Status

Hôpital de Clocheville - CHRU de Tours

Tours, , France

Site Status

Countries

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France

Other Identifiers

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6403

Identifier Type: -

Identifier Source: org_study_id