- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05581056
Evaluation of the Evolution of Quality of Life in Relation to Naso-sinus Symptomatology Under Treatment With CFTE Modulators in Children Aged 6 to 11 Years With Cystic Fibrosis With Compatible Mutation (MUCOSINTRIO)
Evaluation of the Evolution of Quality of Life in Relation to Naso-sinus Symptomatology Under Treatment With CFTR Modulators in Children Aged 6 to 11 Years With Cystic Fibrosis With Compatible Mutation
Cystic fibrosis is a multi-organ disease. It most often results from a genetic mutation, the delta F508 mutation, which prevents the expression of the CFTR 'régulateur de conductance transmembranaire de la fibrose kystique) protein. If the poor prognosis of the disease is correlated to the pulmonary damage, we observe, at the naso-sinus level, a significant functional impact, with chronic rhino-sinusal damage that can alter the quality of life of patients. In addition to this functional impact, some studies suggest that these chronic naso-sinus attacks are involved in the creation of a bacterial reservoir that is secondarily responsible for pulmonary colonization and therefore partly responsible for the poor prognosis of the disease.
The clinical and paraclinical examinations can be used to determine the extent of these disorders. Their functional impact can be assessed using quality of life questionnaires such as the SN-5 scale.
Treatment with CFTR modulators in patients with mutations compatible with the treatment seems to transform their vital prognosis. The scientific rationale of this treatment consists in restoring the activity of the CFTR protein, allowing the recovery of the hydro-electrolytic balance of the mucous secretions, and thus reducing the viscosity of the biological fluids. The various studies carried out all prove a dramatic improvement in pulmonary parameters under treatment, with very limited toxicity.
A marketing authorization for this treatment has been issued on the European market for patients over 18 years of age in 2020, for children over 12 years of age in 2021, and will soon be issued for children aged between 6 and 11 years.
Since the pathophysiology of pulmonary and nasosinus involvement are similar, and since this treatment will be marketed for children between 6 and 11 years of age, we expect an improvement in rhino-sinus symptomatology. To date, clinical studies have focused primarily on pulmonary outcomes. There are only few publications dealing with the evolution of nasosinus symptomatology under treatment, and none concerning the pediatric population.
The aim of our study is to evaluate the evolution of naso-sinusal symptomatology under treatment with CFTR modulators in children aged 6 to 11 years. This will allow us to confirm or deny the interest of these treatments in the extra-pulmonary manifestations of the disease.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Contacts and Locations
Study Contact
- Name: Guillaume PETIT, Dr
- Phone Number: +33 0427855508
- Email: guillaume.petit01@chu-lyon.fr
Study Contact Backup
- Name: Sonia AYARI, Dr
- Phone Number: +33 0427855508
- Email: sonia.ayari-khalfallah@chu-lyon.fr
Study Locations
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-
Rhone
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Bron, Rhone, France, 69500
- Hopital Femme Mere Enfant
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Contact:
- Guillaume PETIT, Dr
- Phone Number: +33 0427855508
- Email: guillaume.petit01@chu-lyon.fr
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
Inclusion Criteria:
- Children between 6 and 11 years of age
- Compatible genotype and patient treated with KAFTRIO (treatment initiation is independent of this study)
Exclusion Criteria:
- Refusal of the child or the holder of parental authority to participate in the study.
- Child not affiliated to a social security system and under legal protection
- Holder of parental authority who does not master the French language
Secondary Exclusion Criteria:
- Sinus surgery during the observation period
- Poor tolerance of the treatment (pneumological opinion)
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
CFTR modulators group
Children between 6 and 11 years old that will be treated by CFTR modulators.
|
Introduction of CFTR modulators by pneumologists for pneumologic issues
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Evolution of quality of life, evaluated with SN-5 scale.
Time Frame: Baseline, 6 months, 12 months
|
The analysis of the primary endpoint is based on the analysis of a quantitative variable, the mean SN-5 score of the entire population, before and at one year after treatment. This statistical analysis is based on a comparison of two averages: the average SN-5 score before treatment and the average SN-5 score at one year after treatment in 90 patients. In previous studies, after external validation, a change of at least 0.57 points in the SN-5 score is used as a threshold to consider at least a slight clinical change |
Baseline, 6 months, 12 months
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 69HCL22_0836
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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