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| ID | Type | Description | Link |
|---|---|---|---|
| CCTIRS 13.652 | Other Identifier | CCTIRS |
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| Name | Class |
|---|---|
| Vaincre la Mucoviscidose | OTHER |
| URC-CIC Paris Descartes Necker Cochin | OTHER |
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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.
The aims of our study are:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ivacaftor 1 | patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CF patients with a G551D mutation and treated with Ivacaftor | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| FEV1 (in liters and in % predicted) | pulmonary function | until one years after initiation of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| pulmonary exacerbations | until one years after initiation of treatment | |
| number of hospitalizations and number of days of hospitalization per year | until one year after initiation of treatment | |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with CF aged 6 or older and cared in French CF centres
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| Name | Affiliation | Role |
|---|---|---|
| Dominique Hubert, MD | Assistance Publique - Hôpitaux de Paris | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Cochin | Paris | 75014 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28711222 | Result | 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. |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D013812 | Therapeutics |
| C545203 | ivacaftor |
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| 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 |
| until one year after initiation of treatment |
| respiratory colonization | Evolution of bacteria and fungi in sputum | until least one year after initiation of treatment |
| nutritional status | Weight (and BMI-Zscore for children) | 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 | until one year after initiation of treatment |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |