Effect of One Year Elexacaftor-tezacaftor-ivacaftor Treatment

NCT06331000 | Completed

• 1 other identifier: 9248
• Study Type: observational
• Target: 184
• Locations: 1 country
• Sites: 1

Brief Summary

The development of CFTR (cystic fibrosis conductance transmembrane regulator) modulators for people with cystic fibrosis (pwCF) and eligible for these treatments is a true therapeutic revolution. The major beneficial effect of CFTR modulators (CFTRm) on pulmonary function and the reduction of pulmonary exacerbations should have a considerable impact on the quality of life and patient's life expectancy. Data on the impact of CFTRm on glucose tolerance abnormalities are still very fragmentary. The investigators can think that their use, earlier and earlier in the history of the disease, will transform the evolutionary trajectories of patients on the respiratory, nutritional and metabolic levels. Diabetes represents a major challenge in the management of pwCF because it is a factor in morbidity and mortality at all stages of the disease, from children to patients with terminal respiratory failure requiring lung transplantation. Early abnormalities in glucose tolerance observed in childhood, before the stage of diabetes, are also associated with poor pulmonary and nutritional outcomes. Experimental data suggest a positive effect of CFTRm on insulin secretion. However, investigators do not currently know the impact of CFTRm in patients with very early glucose disorders or at the stage of diabetes treated with insulin. Recently continuous glucose measurement (CGM) devices represent very effective tools for assessing abnormalities in glucose tolerance before the stage of diabetes and for monitoring patients treated with insulin.

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosis; Elexacaftor-Tezacaftor-Ivacaftor; Glucose tolerance abnormalities

Outcome Measures

Primary Outcomes (1)

• Evaluate time in range of blood glucose variation >140 mg/dl during 2 weeks in a cohort of adult patients treated for one year with elexacaftor-tezacaftor-ivacaftor

  time in range of blood glucose variation >140 mg/dl one year before and one year after the begining of elexacaftor-tezacaftor-ivacaftor treatment

Secondary Outcomes (10)

• Evaluate other CGM parameters : time in range of blood glucose >180mg/dl ; >140mg/dl ; <70mg/dl ; 70-180 mg/dl ; 70-140mg/dl

  One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment

• Marker of metabolic status: HbA1c (mmol/l)

  One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment

• Marker of metabolic status: HbA1c (%)

  One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment

• Marker of metabolic status: C-Peptid (µg/L)

  One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment

• Marker of metabolic status: C-Peptid (nmol/L)

  One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment

Study Arms (1)

Adult patients with cystic fibrosis

Drug: elexacaftor-tezacaftor-ivacaftor treatment

Interventions

elexacaftor-tezacaftor-ivacaftor treatment DRUG

Effect of one year elexacaftor-tezacaftor-ivacaftor treatment on glucose tolerance abnormalities in adult patients with cystic fibrosis

Adult patients with cystic fibrosis

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with cystic fibrosis aged over 18, followed in cystic fibrosis ambulatory care services of 18 french centers

You may qualify if:

• Patients with cystic fibrosis aged over 18
• Patients treated for 1 year by elexacaftortezacaftor-ivacaftor
• Normal tolerant or intolerant to glucose or presenting with cystic fibrosis related diabetes treated or not with insulin
• Patient who had a continuous glucose monitoring in the 6 months before and 6 months after one year of treatment with elexacaftor-tezacaftor-ivacaftor

You may not qualify if:

• Patient not eligible for triple CFTR modulator
• Patient intolerant to triple corrector
• Pregnant woman

Sponsors & Collaborators

• University Hospital, Strasbourg, France: lead

Study Sites (1)

Hopitaux Universitaires de Strasbourg

Strasbourg, 67000, France

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 23, 2024
• First Posted: March 26, 2024
• Study Start: April 8, 2024
• Primary Completion: February 20, 2025
• Study Completion: February 24, 2025
• Last Updated: May 8, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)