Cystic FibrosisIVACAFTOR1NCT02194881
Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation
An observational study of Cystic Fibrosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- CF patients with a G551D mutation and treated with Ivacaftor
- How many people
- 57 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2014to May 2016
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with CF aged 6 or older and cared in French CF centres
Who may be able to join
- French patients with CF aged 6 or older who are homozygous or heterozygous for the G551D mutation
- Treated with Ivacaftor
- First prescription of Ivacaftor before June 1st 2013 (including patients randomized in the VX770 clinical trials)
Who cannot join
- CF patients younger than 6 years old
- CF patients who have received lung transplantation
- CF patients without a G551D mutation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation - Efficacy and Safety Results After the First Year of Treatment in the Real Life Setting.
The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.
Read the detailed description
The aims of our study are:
- to describe the treated population at initiation of treatment,
- to evaluate clinical parameters during the year before Ivacaftor was started, at initiation of treatment and during at least one year of treatment, until June 2014.
- to evaluate the tolerance and safety of this treatment.
- Conditions
- Cystic Fibrosis
- Being tested
- CF patients with a G551D mutation and treated with Ivacaftor
- Keywords
- Cystic fibrosis, CFTR, G551D mutation, Novel treatments, Potentiator, Ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CF patients with a G551D mutation and treated with IvacaftorDrug
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor 1 | patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor Receives: Drug: CF patients with a G551D mutation and treated with Ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
FEV1 (in liters and in % predicted)
pulmonary function
Measured: until one years after initiation of treatment
Secondary outcomes (6)
pulmonary exacerbations
Measured: until one years after initiation of treatment
number of hospitalizations and number of days of hospitalization per year
Measured: until one year after initiation of treatment
number of antibiotic treatments and number of days of antibiotic treatments
number of oral antibiotic treatments and number of days of oral antibiotic treatments, number of IV courses and days of IV antibiotics per year
Measured: until one year after initiation of treatment
respiratory colonization
Evolution of bacteria and fungi in sputum
Measured: until least one year after initiation of treatment
nutritional status
Weight (and BMI-Zscore for children)
Measured: until one year after initiation of treatment
Adverse events
Dates and reasons for interruption and discontinuation of treatment with Ivacaftor Adverse events, indicating what in the physician's opinion might be due to Ivacaftor
Measured: until one year after initiation of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2014 (estimated)
- Study startOct 2014
- Primary completionFeb 2016
- Study completionMay 2016
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 Assistance Publique - Hôpitaux de ParisThe 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
- Retrospective
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Vaincre la Mucoviscidose, URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Dominique Hubert, MD, Study chair, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02194881
- Sponsor's protocol ID
- CCTIRS 13.652
- Other IDs
- CCTIRS 13.652
- Acronym
- IVACAFTOR1
- Status last verified
- Mar 2026
- First submitted
- 17 Jul 2014
- Last updated
- 27 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
Hubert D, Dehillotte C, Munck A, David V, Baek J, Mely L, Dominique S, Ramel S, Danner Boucher I, Lefeuvre S, Reynaud Q, Colomb-Jung V, Bakouboula P, Lemonnier L. Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting. J Cyst Fibros. 2018 Jan;17(1):89-95. doi: 10.1016/j.jcf.2017.07.001. Epub 2017 Jul 12.
PubMed 28711222
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation - Efficacy and Safety Results After the First Year of Treatment in the Real Life Setting.
- ClinicalTrials.gov ID
- NCT02194881
- Acronym / sponsor ID
- IVACAFTOR1 / CCTIRS 13.652
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis, CFTR, G551D mutation, Novel treatments, Potentiator, Ivacaftor
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Vaincre la Mucoviscidose, URC-CIC Paris Descartes Necker Cochin
- Study start
- Oct 2014 (actual)
- Primary completion
- Feb 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 18 Jul 2014 (estimated)
- 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 purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.
Detailed description
The aims of our study are:
- to describe the treated population at initiation of treatment,
- to evaluate clinical parameters during the year before Ivacaftor was started, at initiation of treatment and during at least one year of treatment, until June 2014.
- to evaluate the tolerance and safety of this treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor 1 | – | Drug: CF patients with a G551D mutation and treated with Ivacaftorpatients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with CF aged 6 or older and cared in French CF centres
Key inclusion criteria (3 listed)
- French patients with CF aged 6 or older who are homozygous or heterozygous for the G551D mutation
- Treated with Ivacaftor
- First prescription of Ivacaftor before June 1st 2013 (including patients randomized in the VX770 clinical trials)
Key exclusion criteria (3 listed)
- CF patients younger than 6 years old
- CF patients who have received lung transplantation
- CF patients without a G551D mutation.
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 |
|---|---|---|
| CF patients with a G551D mutation and treated with Ivacaftor | Drug | Ivacaftor 1 |
Primary outcome
FEV1 (in liters and in % predicted)
Time frame: until one years after initiation of treatment
pulmonary function
6 secondary outcomes
pulmonary exacerbations
Time frame: until one years after initiation of treatment
number of hospitalizations and number of days of hospitalization per year
Time frame: until one year after initiation of treatment
number of antibiotic treatments and number of days of antibiotic treatments
Time frame: until one year after initiation of treatment
respiratory colonization
Time frame: until least one year after initiation of treatment
nutritional status
Time frame: until one year after initiation of treatment
Adverse events
Time frame: until one year after initiation of treatment
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 27 Mar 2026.
- Results publication: PubMed 28711222Hubert D, Dehillotte C, Munck A, David V, Baek J, Mely L, Dominique S, Ramel S, Danner Boucher I, Lefeuvre S, Reynaud Q, Colomb-Jung V, Bakouboula P, Lemonnier L. Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting. J Cyst Fibros. 2018 Jan;17(1):89-95. doi: 10.1016/j.jcf.2017.07.001. Epub 2017 Jul 12.
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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.
Dominique Hubert, MD study chair
Assistance Publique - Hôpitaux de Paris
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 Assistance Publique - Hôpitaux de Paris 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 3 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: finished, as of the sponsor's last update on 27 Mar 2026. 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?
FEV1 (in liters and in % predicted) (time frame: until one years after initiation of treatment). 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 NCT02194881, 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 27 Mar 2026.