MAB-CF: Anti-IL5 and Other Biotherapies in Cystic Fibrosis

Sponsor

University Hospital, Montpellier (Other)

Overall Status

Unknown status

CT.gov ID

NCT04256772

Collaborator

(none)

100

Enrollment

Location

Anticipated Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Our project is to describe retrospectively and prospectively CF patients treated with biotherapy in French CF centers.

Main objective: To describe the clinical and paraclinical course of CF patients before and after treatment with anti-IL5 and other biotherapies since 2019.

Secondary objective: To describe adverse events potentially related to the biotherapies.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis

Detailed Description

•Background: ABPA and asthma associated with cystic fibrosis impact the CF course with a more rapid decline in lung function.

Corticosteroid therapy can be harmful and must be avoid in CF to prevent diabetes, osteoporomalacia or mycobacterium infections.

Monoclonal antibodies have the marketing authorization for severe uncontrolled asthma and, up to now, some CF patients with ABPA or severe asthma and high plasma IgE levels benefit from omalizumab.

Anti-Il5 agents are available since February 2019 and have demonstrated their efficacy in severe and hypereosinophilic asthma control (plasma eos.>300mmol/L).

Some patients with CF who have severe asthma or ABPA are still poorly controlled despite Omalizumab with other treatments (steroids and/or azoles). Some of them have persistent hypereosinophilia suggesting a possibility to treat with antiIL5 antibodies.

About 5% of patients have biotherapy treatment criteria, some have already received it, others are elective to such treatment and will receive in the future.

Methods:

Our project is to describe retrospectively and prospectively the clinical history of CF patients eligible for biotherapy in French CF centers.

Main objective: To describe the clinical and paraclinical course of CF patients before and after treatment with anti-IL5 and other biotherapies since 2019.

Secondary objective: To describe any adverse events potentially related to the biotherapies.

Study Design

Study Type:

Observational

Actual Enrollment :

100 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

Anti-IL5 and Other Biotherapies in Cystic Fibrosis in French CF Centers : MAB-CF Cohort

Actual Study Start Date :

Jul 1, 2019

Actual Primary Completion Date :

Jun 1, 2020

Anticipated Study Completion Date :

Mar 31, 2022

Outcome Measures

Primary Outcome Measures

Clinical evolution [1 day]
Physical examination (height in centimeters, weight in kilograms, numerical scales in score, temperature in degrees, SaO2 in percentage). Defines the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis To assess the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis
Spirometry in liters or percentage evolution [1 day]
Spirometry in liters or percentage Defines the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis
Biology evolution [1 day]
Biology (leukocyte formula in 10^9/L, IgE tot, IgE spe, sputum microbiology) Defines the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis
concomitant therapy evolution [1 day]
concomitant therapy Defines the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis
CFQR and SNOT22 evolution [1 day]
CFQR and SNOT22 questionnaires in score Defines the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis
exacerbation evolution [1 day]
Defines the effectiveness of anti-inflammatory biotherapy in patients with cystic fibrosis

Secondary Outcome Measures

Number of any adverse event reported during biotherapy treatments [1 day]
Describe any adverse event potentially related to biotherapies. description of any adverse event reported during biotherapy treatments.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion criteria:

Cystic fibrosis (2 known variants)
Age ≥ 6 years
Plasma hypereosinophilia ≥ 300/µl
Uncontrolled ABPA or uncontrolled asthma, or failure of other biotherapy (intolerance, ineffectiveness)

Exclusion criteria