Cystic FibrosisNCT05526027
Real-world Efficacy and Safety of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy in Adult Patients With Cystic Fibrosis (CF)
A study testing CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement in people with Cystic Fibrosis, run by Universitair Ziekenhuis Brussel.
Trial summary
In this trial real-world data on the safety (side effects and medication interactions) and efficacy (evolution of lung function testing, chronic bacterial airway infection, quality of life and endo- and exocrine pancreatic function) will be collected in adult people with cystic fibrosis (pwCF) eligible for… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 85 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2022to 31 Dec 2024
- Phase
- Interventional
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- eligible for ETI (i.e. age above 18 years and CFTR genotype F508del/any) based on reimbursement criteria in Belgium
Who cannot join
- inability to perform lung function testing
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Real-world Efficacy and Safety of CFTR Modulator Therapy in Adult Patients With Cystic Fibrosis
In this trial real-world data on the safety (side effects and medication interactions) and efficacy (evolution of lung function testing, chronic bacterial airway infection, quality of life and endo- and exocrine pancreatic function) will be collected in adult people with cystic fibrosis (pwCF) eligible for elexacaftor-tezacaftor-ivacaftor (ETI) up until 2 years after the start of this therapy.
Read the detailed description
PwCF group A: CFTR-modulator-naive pwCF eligible for ETI (based on age and CFTR genotype)
PwCF group B: pwCF already on CFTR modulating therapy (i.e. ivacaftor-lumacaftor or ivacaftor-tezacaftor) and switching to ETI.
Patients in both groups will undergo these examinations:
- Physical examination (including weight)
- Anamnesis for current and recent medication use (including dosage of pancreatic enzymes) and for acute respiratory exacerbations
- Lung function testing including spiometry, multiple breath washout testing and fractional exhaled nitric oxide
- Blood sampling: liver function tests, creatine kinase, albumin, PT, red and white blood cell count, platelet count
- Sputum/cough swab sampling
- fecal elastase measurement
- Cystic fibrosis questionnaire-revised (CFQ-R) questionnaire
- Patient health questionnaire-9 (PHQ-9) questionnaire
- General anxiety disorder-7 (GAD-7) questionnaire
- Sino-nasal outcome test-22 (SNOT-22) questionnaire
These will be performed at baseline (prior to the start of ETI, on the same day of start of ETI), and every 3 months (+/- 7 days) thereafter. A blood sample will also be performed 14 (+/- 7 days) days after start of ETI (for safety). Fecal elastase measurement will only be performed at baseline if not available in the patient's medical record, and only 6 months after start of ETI.
- Conditions
- Cystic Fibrosis
- Being tested
- CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurementDiagnostic test
cf supra
| Study group | Role | What they receive |
|---|---|---|
| ETI | Other | pwCF which are CFTR-modulator naive and pwCF previously treated with a CFTR-modulator (i.e. tezacaftor-ivacaftor or lumacaftor-ivacaftor) will undergo standard-of-care examinations as well as examinations in the context of this trial (i.e. CFQ-R, PHQ-9, GAD-7 and SNOT-22 questionnaires, fecal elastase measurement) Receives: Diagnostic Test: CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement |
What is being measured
The primary outcome is the main question the study is designed to answer.
percent predicted forced expiratory volume in 1 second (ppFEV1)
Measured: 2 years
Lung clearance index (LCI)
Measured: 2 years
Fractional excretion of nitric oxide (FeNO)
Measured: 2 years
CFQ-R questionnaire
Measured: 2 years
fecal elastase (microgram per gram of feces)
the concentration of elastase (a pancreatic enzyme) in feces before and 6 months after start of CFTR modulating treatment, as a surrogate measure for pancreatic function
Measured: 2 years
the amount of participants experiencing a treatment-related adverse event
Measured: 2 years
annual acute exacerbation rate
Measured: 2 years
body mass index (BMI)
Measured: 2 years
Secondary outcomes (5)
SNOT-22 questionnaire
Measured: 2 years
GAD-7 questionnaire
Measured: 2 years
PHQ-9 questionnaire
Measured: 2 years
aerobic culture on sputum or cough swab sample
Measured: 2 years
dosage of pancreatic enzyme replacement therapy
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2022
- Study start1 Sep 2022 (estimated)
- Primary completion31 Dec 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Universitair Ziekenhuis BrusselThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 85 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universitair Ziekenhuis Brussel
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Stefanie Vincken, Universitair Ziekenhuis Brussel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05526027
- Sponsor's protocol ID
- BUN1432021000472
- Status last verified
- Aug 2022
- First submitted
- 18 Aug 2022
- Last updated
- 2 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-world Efficacy and Safety of CFTR Modulator Therapy in Adult Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT05526027
- Acronym / sponsor ID
- BUN1432021000472
- Conditions
- Cystic Fibrosis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 85 participants (estimated)
- Lead sponsor
- Universitair Ziekenhuis BrusselUniversity, hospital or other
- Study start
- 1 Sep 2022 (estimated)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 2 Sep 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.
In this trial real-world data on the safety (side effects and medication interactions) and efficacy (evolution of lung function testing, chronic bacterial airway infection, quality of life and endo- and exocrine pancreatic function) will be collected in adult people with cystic fibrosis (pwCF) eligible for elexacaftor-tezacaftor-ivacaftor (ETI) up until 2 years after the start of this therapy.
Detailed description
PwCF group A: CFTR-modulator-naive pwCF eligible for ETI (based on age and CFTR genotype)
PwCF group B: pwCF already on CFTR modulating therapy (i.e. ivacaftor-lumacaftor or ivacaftor-tezacaftor) and switching to ETI.
Patients in both groups will undergo these examinations:
- Physical examination (including weight)
- Anamnesis for current and recent medication use (including dosage of pancreatic enzymes) and for acute respiratory exacerbations
- Lung function testing including spiometry, multiple breath washout testing and fractional exhaled nitric oxide
- Blood sampling: liver function tests, creatine kinase, albumin, PT, red and white blood cell count, platelet count
- Sputum/cough swab sampling
- fecal elastase measurement
- Cystic fibrosis questionnaire-revised (CFQ-R) questionnaire
- Patient health questionnaire-9 (PHQ-9) questionnaire
- General anxiety disorder-7 (GAD-7) questionnaire
- Sino-nasal outcome test-22 (SNOT-22) questionnaire
These will be performed at baseline (prior to the start of ETI, on the same day of start of ETI), and every 3 months (+/- 7 days) thereafter. A blood sample will also be performed 14 (+/- 7 days) days after start of ETI (for safety). Fecal elastase measurement will only be performed at baseline if not available in the patient's medical record, and only 6 months after start of ETI.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ETI | Other | Diagnostic Test: CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurementpwCF which are CFTR-modulator naive and pwCF previously treated with a CFTR-modulator (i.e. tezacaftor-ivacaftor or lumacaftor-ivacaftor) will undergo standard-of-care examinations as well as examinations in the context of this trial (i.e. CFQ-R, PHQ-9, GAD-7 and SNOT-22 questionnaires, fecal elastase measurement) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- eligible for ETI (i.e. age above 18 years and CFTR genotype F508del/any) based on reimbursement criteria in Belgium
Key exclusion criteria (1 listed)
- inability to perform lung function testing
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 |
|---|---|---|
| CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurementcf supra | Diagnostic test | ETI |
Primary outcome
percent predicted forced expiratory volume in 1 second (ppFEV1)
Time frame: 2 years
Lung clearance index (LCI)
Time frame: 2 years
Fractional excretion of nitric oxide (FeNO)
Time frame: 2 years
CFQ-R questionnaire
Time frame: 2 years
fecal elastase (microgram per gram of feces)
Time frame: 2 years
the concentration of elastase (a pancreatic enzyme) in feces before and 6 months after start of CFTR modulating treatment, as a surrogate measure for pancreatic function
the amount of participants experiencing a treatment-related adverse event
Time frame: 2 years
annual acute exacerbation rate
Time frame: 2 years
body mass index (BMI)
Time frame: 2 years
5 secondary outcomes
SNOT-22 questionnaire
Time frame: 2 years
GAD-7 questionnaire
Time frame: 2 years
PHQ-9 questionnaire
Time frame: 2 years
aerobic culture on sputum or cough swab sample
Time frame: 2 years
dosage of pancreatic enzyme replacement therapy
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2022.
- 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.
Questions about this trial
What is this clinical trial studying?
A study testing CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement in people with Cystic Fibrosis, run by Universitair Ziekenhuis Brussel.
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 1 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 2 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Stefanie Vincken, M.D. (+32 2 477 68 41, stefanie.vincken@uzbrussel.be), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
percent predicted forced expiratory volume in 1 second (ppFEV1) (time frame: 2 years). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Sep 2022.