Clinical trial NCT06072365: Treatment With Elexacaftor/Tezacaftor/Ivacaftor, in Patients With Cystic Fibrosis and Caloric Intake
Official title: 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.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Keywords: caloric intake
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
First posted: 2023-10-10
Enrollment: 120
Sex: ALL
Minimum age (years): 2
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

Brief summary: 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.

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.

Eligibility criteria: 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:

-

Primary outcomes:
measure: caloric intake 12 months after the beginning of treatment
timeFrame: 12 months
Secondary outcomes:
measure: caloric intake 3 months after the beginning of treatment
timeFrame: 3 months
Other outcomes:
Locations:
city: Bordeaux
country: France
facility: UHBordeaux
city: Limoges
country: France
facility: UHLimoges
city: Toulouse
country: France
facility: UHToulouse
Study officials:
affiliation: University Hospital, Toulouse
name: Marion LAGARRIGUE
role: PRINCIPAL_INVESTIGATOR
References: