Cystic FibrosisMUCOSINTRIONCT05581056
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
An observational study of Cystic Fibrosis, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 to 11 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- CFTR modulators
- How many people
- 27 participants
- Where
- 1 siteFrance
- Study dates
- 3 Mar 2023to 2 Sep 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 11 years
- All sexes
- No healthy volunteers
- Child
Study population: Children between 6 and 11 years of age
Who may be able to join
- Children between 6 and 11 years of age
- Compatible genotype and patient treated with KAFTRIO (treatment initiation is independent of this study)
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read more
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.
- Conditions
- Cystic Fibrosis
- Being tested
- CFTR modulators
- Keywords
- Cystic Fibrosis, Naso sinusal symptoms
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CFTR modulatorsDrug
Introduction of CFTR modulators by pneumologists for pneumologic issues
| Study group | Role | What they receive |
|---|---|---|
| CFTR modulators group | Children between 6 and 11 years old that will be treated by CFTR modulators. Receives: Drug: CFTR modulators |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evolution of quality of life, evaluated with SN-5 scale.
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
Measured: Baseline, 6 months, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2022
- Study start3 Mar 2023
- Primary completion2 Sep 2024
- Study completion2 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05581056
- Sponsor's protocol ID
- 69HCL22_0836
- Acronym
- MUCOSINTRIO
- Status last verified
- Sep 2024
- First submitted
- 12 Oct 2022
- Last updated
- 18 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Petit G, Coudert A, Hermann R, Truy E, Bonjour M, Reix P, Ayari S. Effects of Elexacaftor-Tezacaftor-Ivacaftor on Nasal and Sinus Symptoms in Children With Cystic Fibrosis. Pediatr Pulmonol. 2025 Jan;60(1):e27493. doi: 10.1002/ppul.27493.
PubMed 39868969
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05581056
- Acronym / sponsor ID
- MUCOSINTRIO / 69HCL22_0836
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Naso sinusal symptoms
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 3 Mar 2023 (actual)
- Primary completion
- 2 Sep 2024 (actual)
- Study completion
- 2 Sep 2024 (actual)
- First posted
- 14 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CFTR modulators group | – | Drug: CFTR modulatorsChildren between 6 and 11 years old that will be treated by CFTR modulators. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children between 6 and 11 years of age
Key inclusion criteria (2 listed)
- Children between 6 and 11 years of age
- Compatible genotype and patient treated with KAFTRIO (treatment initiation is independent of this study)
Key exclusion criteria (6 listed)
- 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
- Sinus surgery during the observation period
- Poor tolerance of the treatment (pneumological opinion)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CFTR modulatorsIntroduction of CFTR modulators by pneumologists for pneumologic issues | Drug | CFTR modulators group |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2025.
- Results publication: PubMed 39868969Petit G, Coudert A, Hermann R, Truy E, Bonjour M, Reix P, Ayari S. Effects of Elexacaftor-Tezacaftor-Ivacaftor on Nasal and Sinus Symptoms in Children With Cystic Fibrosis. Pediatr Pulmonol. 2025 Jan;60(1):e27493. doi: 10.1002/ppul.27493.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Hospices Civils de Lyon at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evolution of quality of life, evaluated with SN-5 scale. (time frame: Baseline, 6 months, 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05581056, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2025.