Treatment With Elexacaftor/Tezacaftor/Ivacaftor, in Patients With Cystic Fibrosis and Caloric Intake (NUTRIMUCO)

Evaluation of the Change in Caloric Intake Before and 12 Months After the Initiation of Treatment With Elexacaftor/Tezacaftor/Ivacaftor, in Patients With Cystic Fibrosis.

The aim of the study is to describe the evolution of caloric intake in patients with cystic fibrosis with an indication to start treatment with Elexacaftor/Tezacaftor/Ivacaftor according to the Marketing Authorization, between the start of treatment and at 12 months.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Other: nutritional intake questionnaire

Detailed Description

Cystic fibrosis is a genetic pathology linked to a dysfunction of the CFTR protein.

Undernutrition is common in the natural course of cystic fibrosis, it is linked to exocrine pancreatic insufficiency but also to the increase in energy expenditure due to respiratory damage.

At a time when these CFTR modulators are greatly modifying the prognosis and management of cystic fibrosis, it is important to describe how caloric and nutritional intake evolve under treatment with Elexacaftor/Tezacaftor/Ivacaftor in order to be able, in the long term, to adapt nutritional recommendations under treatment with CFTR modulators.

The aim of the study is to describe the evolution of caloric intake in patients with cystic fibrosis with an indication to start treatment with Elexacaftor/Tezacaftor/Ivacaftor according to the Marketing Authorization, between the start of treatment and at 12 months.

• Study Type: Observational
• Enrollment (Actual): 120

Contacts and Locations

Study Locations

• France
  • Bordeaux, France, 33404
    • UHBordeaux
  • Limoges, France, 87042
    • UHLimoges
  • Toulouse, France, 31059
    • UHToulouse

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

• Refusal to participate
• Patients and/or holders of parental authority presenting difficulties in understanding and/or communication.
• Inability to complete a reliable food collection.
• Any pathological condition making interpretation of results difficult from the investigator's point of view.
• Transplant patient
• Pregnant or breastfeeding woman
• Person under guardianship or curatorship.

Description

Inclusion Criteria:

• Patient with cystic fibrosis
• Patient with an indication to start treatment with Elexacaftor/Tezacaftor/Ivacaftor according to the Marketing Authorization.
• Patient who has not received treatment with CFTR modulators in the 6 months preceding inclusion.
• No opposition expressed by the patient (if ≥ 18 years) or by at least one of the holders of parental authority and the child (if < 18 years).
• Be affiliated to a social security scheme or be a beneficiary of such a scheme.

Exclusion Criteria:

-

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
cystic fibrosis; patient with cystic fibrosis treated with With Elexacaftor/Tezacaftor/Ivacaftor,	Other: nutritional intake questionnaire; patients completed a nutritional intake questionnaire before to start the treatment, 3mounths after and twelve months after

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
caloric intake 12 months after the beginning of treatment	Describe the evolution of caloric intake in patients with cystic fibrosis with an indication to start treatment with Elexacaftor/Tezacaftor/Ivacaftor according to the Marketing Authorization, between the start of treatment and at 12 months.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
caloric intake 3 months after the beginning of treatment	Describe the evolution of caloric intake in patients with cystic fibrosis with an indication to start treatment with Elexacaftor/Tezacaftor/Ivacaftor according to the Marketing Authorization, between the start of treatment and at 3 months.	3 months

Collaborators and Investigators

• Sponsor: University Hospital, Toulouse
• Investigators: Principal Investigator: Marion LAGARRIGUE, University Hospital, Toulouse

Study record dates

Study Major Dates

• Study Start (Actual): October 21, 2021
• Primary Completion (Actual): September 23, 2022
• Study Completion (Actual): January 15, 2023

Study Registration Dates

• First Submitted: October 2, 2023
• First Submitted That Met QC Criteria: October 2, 2023
• First Posted (Estimated): October 9, 2023

Study Record Updates

• Last Update Posted (Estimated): October 9, 2023
• Last Update Submitted That Met QC Criteria: October 2, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: caloric intake
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: RC31/21/0364; 2021-A02018-33 (Other Identifier: ID-RCB)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No