Cystic Fibrosis (CF)PERCE-MUCONCT07885410
PERCE-MUCO: Perceptions and Expectations of People With Cystic Fibrosis Regarding a Change in Treatment
An observational study of Cystic Fibrosis (CF), run by Fondation Ildys at 11 sites in France.
Trial summary
PERCE-MUCO is a prospective, multicentre, mixed-methods observational study investigating the perceptions, expectations and treatment-change intentions of adults with cystic fibrosis who are currently treated with elexacaftor/tezacaftor/ivacaftor (ETI) or whose treatment has been medically discontinued. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis (CF)
- How many people
- 343 participants
- Where
- 11 sitesFrance
- Study dates
- 9 Jul 2026to Dec 2026
- Phase
- Observational
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults with cystic fibrosis followed at participating French cystic fibrosis resource and competence centres (CRCMs), who currently receive ETI or whose treatment was medically discontinued. The qualitative interview component is limited to eligible patients followed at the Roscoff CRCM.
Who may be able to join
- Diagnosis of cystic fibrosis
- Currently treated with ETI for at least 3 months, OR previously treated with ETI but treatment discontinued for medical reasons
Who cannot join
- Patient objects to the use of their data for this project\n•
- Currently receiving the new CFTR modulator VTD
Trial locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Matthieu Pichelin contact
11 sites in 1 country
France11 sites, 11 recruiting
- Not yet recruiting
CRCM Caen
Caen
Carine Campell
- Recruiting
CRCM Giens
Giens
Nadège Goriot-Raynaud, Research Nurse
- Not yet recruiting
CRCM Lille
Lille
Camille Audousset
- Not yet recruiting
CRCM Lyon
Lyon
Isabelle Durieu
- Recruiting
CRCM Montpellier
Montpellier
Raphael Chiron
- Not yet recruiting
CRCM Paris
Paris
Pierre Regis Burgel, Professor, PI
- Recruiting
CRCM Reims
Reims
Pauline Mulette
- Not yet recruiting
CRCM Rennes adultes
Rennes
Chantal Belleguic, MD, PI
- Recruiting
Fondation ILDYS
Roscoff, 29680
Morgane Auffret, Research Nurse, 0298292523 ext. +33, morgane.auffret@ildys.org
- Recruiting
CRCM Rouen
Rouen
Carine Choubrac
- Not yet recruiting
CRCM Vannes
Vannes
Baptiste Arnouat, MD, PI
About this study
Official title: Perceptions and Expectations of Patients Regarding a Change in Treatment in Cystic Fibrosis (PERCE-MUCO)
PERCE-MUCO is a prospective, multicentre, mixed-methods observational study investigating the perceptions, expectations and treatment-change intentions of adults with cystic fibrosis who are currently treated with elexacaftor/tezacaftor/ivacaftor (ETI) or whose treatment has been medically discontinued. The study focuses on patients' stated willingness to switch from ETI to vanzacaftor/tezacaftor/deutivacaftor (VTD), and on the factors associated with this intention, including treatment satisfaction, knowledge and expectations regarding VTD, quality of life, treatment adherence, reported adverse effects, access-related concerns and desired support from an advanced practice nurse. The quantitative component uses a study-specific questionnaire and routine-care CFQ-R 14+ and Morisky-8 data. A qualitative component consists of semi-structured interviews at the Roscoff cystic fibrosis centre.
Read the detailed description
Cystic fibrosis is a rare genetic disease for which CFTR modulator therapy has substantially changed treatment and patients' daily lives. Although elexacaftor/tezacaftor/ivacaftor (ETI) has improved respiratory outcomes and reduced exacerbations for many eligible patients, some patients experience intolerance or a suboptimal response. Vanzacaftor/tezacaftor/deutivacaftor (VTD) is a newer CFTR modulator combination, and patients' willingness to change from a familiar treatment to a new option cannot be assumed.
The primary objective is to assess the intention of adults with cystic fibrosis treated with ETI to change to VTD.
The primary outcome is the response to a single-choice question asking what the patient would do if their physician offered CTD: accept immediately, need time to think, or refuse.
Secondary objectives are to identify determinants of acceptance; assess the relationship between quality of life and intention to change; assess the relationship of adherence and reported tolerability with intention to change; describe the role and desired support of an advanced practice nurse during a possible transition; and explore psychological and logistical barriers through qualitative interviews.
The quantitative component is conducted across participating French cystic fibrosis centres. Participants complete the PERCE-MUCO questionnaire during a routine follow-up visit or planned hospital stay. The most recent routine-care CFQ-R 14+ and Morisky-8 questionnaires available near inclusion, together with selected clinical and treatment characteristics and adverse effects documented in the medical record, are collected in pseudonymised form.
The qualitative component is conducted at the Roscoff centre and uses individual semi-structured face-to-face interviews lasting approximately 30-45 minutes. Interviews are audio-recorded with the participant's agreement, transcribed verbatim and pseudonymised before thematic analysis.
The quantitative primary outcome is analysed descriptively; secondary analyses include descriptive statistics and comparisons using ANOVA or Kruskal-Wallis, and chi-square or Fisher's exact tests as appropriate.
Qualitative data are analysed thematically using a combination of deductive and inductive coding. The protocol estimates a quantitative sample of 343 participants and approximately 8-12 qualitative interviews, with interview recruitment continuing until data saturation.
- Conditions
- Cystic Fibrosis (CF)
- Keywords
- CFTR modulator; treatment preference; treatment switching; quality of life; adherence; patient experience; advanced practice nursing
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Adults with cystic fibrosis treated with ETI or with medically discontinued ETI | Adults with cystic fibrosis who have received ETI for at least 3 months or whose ETI treatment was discontinued for medical reasons. The study observes treatment preferences and associated characteristics; it does not assign or require a change in treatment. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Intention to switch from ETI to VTD
Percentage of participants selecting each of three responses to the question: 'If your physician offered VTD tomorrow, what would your position be?' Response options: immediate acceptance; need time to think; refusal. Report proportions and 95% confidence intervals
Measured: At completion of the PERCE-MUCO questionnaire at inclusion (single assessment).
Secondary outcomes (6)
Factors influencing willingness to switch
Descriptive proportions for knowledge of VTD and satisfaction with information received; preferred information sources; expectations; concerns about efficacy, adverse effects, replacing a satisfactory treatment and tolerability; and perceived access to treatment
Measured: At completion of the PERCE-MUCO questionnaire at inclusion.
Association between quality of life and intention to switch
Association between CFQ-R 14+ scores, including the health-perception score specified in the protocol, and the three-category intention-to-switch outcome
Measured: CFQ-R 14+ measurement closest to inclusion, collected from routine care
Association between treatment adherence and intention to switch
Association between Morisky-8 score and intention to switch; adherence categories are good (score 8), medium (6-7) and low (<6).
Measured: Morisky-8 measurement closest to inclusion, collected from routine care
Association between reported ETI tolerability and intention to switch
Comparison of intention-to-switch categories according to the presence or absence of adverse effects documented in the medical record
Measured: Adverse effects documented in the medical record and available at inclusion
Need for support during a potential treatment transition
Percentage of participants requesting specific support during transition and descriptive summary of requested support, including support from a physician, coordinating nurse or advanced practice nurse
Measured: At completion of the PERCE-MUCO questionnaire at inclusion
Patient perceptions and barriers identified in semi-structured interviews
Thematic analysis of interview transcripts exploring lived experience of Kaftrio, perception of therapeutic innovation, decision-making and the role of the advanced practice nurse
Measured: One semi-structured interview of approximately 30-45 minutes during the study period.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start9 Jul 2026
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis (CF)Studies of the same condition, open and under way.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 343 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondation Ildys
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07885410
- Sponsor's protocol ID
- RNIPH-2026-007
- Other IDs
- RNIPH-2026-18
- Acronym
- PERCE-MUCO
- Status last verified
- Oct 2026
- First submitted
- 13 Oct 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided