Cystic FibrosisNCT05599230
Daily Monitoring of Respiratory Symptoms and Spirometry During ETI Treatment in Persons With Cystic Fibrosis.
An observational study of Cystic Fibrosis, run by CHC Montlegia at 1 site in Belgium.
Trial summary
The goal of this observational study is to provide optimal monitoring and support when initiating ETI treatment in eligible persons with cystic fibrosis (aged 12 y +) and to document on a daily basis, from 72 hours before the start of treatment and then for 14 days i) i) FEV1 changes (home spirometry), ii) ii)… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each evening
- Type of treatment
- Pill or oral medicine
- How many people
- 20 participants
- Where
- 1 siteBelgium
- Study dates
- 1 Sep 2022to 30 Nov 2022
- 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 12 years and older
- All sexes
- Child, Adult, Older adult
Study population: CF patients aged 12 y or more, carrying at least one copy of F508del mutation, in stable condition, reliably performing spirometry
Who may be able to join
- Cystic fibrosis diagnosis
- At least 12 years old
- Carrier of at least one copy of F508del mutation
- Ability to perform reliable and reproducible spirometry
- Medical and psychological stability
- written consent
- owning a smartphone
Who cannot join
- Lung transplant
- FEV1 > 120 % pr (%GLI) at the inclusion test
- Pulmonary exacerbation
Trial locations and contacts
1 site in 1 country.
About this study
The goal of this observational study is to provide optimal monitoring and support when initiating ETI treatment in eligible persons with cystic fibrosis (aged 12 y +) and to document on a daily basis, from 72 hours before the start of treatment and then for 14 days i) i) FEV1 changes (home spirometry), ii) ii) respiratory symptoms changes, iii) any possible side effects. Through a dedicated electronic platform, these data will be monitored every day by the medical team, which will be fully available for any questions or concerns patients may have.
- Conditions
- Cystic Fibrosis
- Being tested
- Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each evening
- Keywords
- elexacaftor, time course, FEV1, Respiratory symptoms
- Medical subject headings
- Cystic Fibrosis, Signs and Symptoms, Respiratory
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each eveningDrug
Patients will daily perform home spirometry and complete a respiratory symptoms score before (3 days) and during (first 14 days) ETI treatment.
| Study group | Role | What they receive |
|---|---|---|
| CF patients before (3 days) and during (14 days) ETI treatment | Patients over 12 years old and eligible will start ETI treatment ( current standart treatment).
* Morning: Elexacaftor 100 mg, Tezacaftor 50 mg, Ivacaftor 75 mg, 2 tablets
* Evening: Ivacaftor 150 mg Receives: Drug: Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each evening |
What is being measured
The primary outcome is the main question the study is designed to answer.
FEV1 changes from baseline (3 days before treatment) to Day 14 under ETI
Daily home spirometry with Spirobank device, between 14H-20H, before nebulization and / or physiotherapy
Measured: 17 days
Secondary outcomes (1)
Respiratory symptoms score changes from baseline (3 days before treatment) to Day 14 under ETI
Daily scoring unsing a respiratory questionnaire (8 questions); each item is scored 1 to 5 (worse score: 40)
Measured: 17 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2022
- Study start1 Sep 2022
- Primary completion30 Nov 2022 (estimated)
- Study completion30 Nov 2022 (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.
- 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
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHC Montlegia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lebecque Patrick, CHC Montlegia
- Investigators
- Patrick Lebecque, PhD, Principal investigator, CHC Montlegia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05599230
- Sponsor's protocol ID
- ML001
- Status last verified
- Oct 2022
- First submitted
- 21 Oct 2022
- Last updated
- 31 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Daily Monitoring of Respiratory Symptoms and Spirometry During ETI Treatment in Persons With Cystic Fibrosis.
- ClinicalTrials.gov ID
- NCT05599230
- Acronym / sponsor ID
- ML001
- Conditions
- Cystic Fibrosis
- Keywords
- elexacaftor, time course, FEV1, Respiratory symptoms
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (actual)
- Lead sponsor
- CHC MontlegiaUniversity, hospital or other
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 30 Nov 2022 (estimated)
- Study completion
- 30 Nov 2022 (estimated)
- First posted
- 31 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.
The goal of this observational study is to provide optimal monitoring and support when initiating ETI treatment in eligible persons with cystic fibrosis (aged 12 y +) and to document on a daily basis, from 72 hours before the start of treatment and then for 14 days i) i) FEV1 changes (home spirometry), ii) ii) respiratory symptoms changes, iii) any possible side effects. Through a dedicated electronic platform, these data will be monitored every day by the medical team, which will be fully available for any questions or concerns patients may have.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CF patients before (3 days) and during (14 days) ETI treatment | – | Drug: Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each eveningPatients over 12 years old and eligible will start ETI treatment ( current standart treatment). * Morning: Elexacaftor 100 mg, Tezacaftor 50 mg, Ivacaftor 75 mg, 2 tablets * Evening: Ivacaftor 150 mg |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- CF patients aged 12 y or more, carrying at least one copy of F508del mutation, in stable condition, reliably performing spirometry
Key inclusion criteria (7 listed)
- Cystic fibrosis diagnosis
- At least 12 years old
- Carrier of at least one copy of F508del mutation
- Ability to perform reliable and reproducible spirometry
- Medical and psychological stability
Key exclusion criteria (3 listed)
- Lung transplant
- FEV1 > 120 % pr (%GLI) at the inclusion test
- Pulmonary exacerbation
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 |
|---|---|---|
| Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each eveningPatients will daily perform home spirometry and complete a respiratory symptoms score before (3 days) and during (first 14 days) ETI treatment. | Drug | CF patients before (3 days) and during (14 days) ETI treatment |
Primary outcome
FEV1 changes from baseline (3 days before treatment) to Day 14 under ETI
Time frame: 17 days
Daily home spirometry with Spirobank device, between 14H-20H, before nebulization and / or physiotherapy
1 secondary outcome
Respiratory symptoms score changes from baseline (3 days before treatment) to Day 14 under ETI
Time frame: 17 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2022.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
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.
Patrick Lebecque, PhD principal investigator
CHC Montlegia
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 CHC Montlegia at 1 site in Belgium.
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 7 inclusion and 3 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 31 Oct 2022. 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 Belgium. 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?
Results are not available in the source record.
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?
FEV1 changes from baseline (3 days before treatment) to Day 14 under ETI (time frame: 17 days). 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 31 Oct 2022.