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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-002704-24 | EudraCT Number |
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To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ivacaftor | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Pulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment | 48 Months | |
| Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment | 48 Months | |
| Respiratory Microbiology - Percentage of patients with cultures positive for bacteria other than Pseudomonas aeruginosa and for fungi during Kalydeco treatment compared to the period before Kalydeco treatment | 48 Months | |
| FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment | 48 Months | |
| Measures of nutritional status: Absolute change in weight, weight-for-age Z score, body mass index (BMI), and BMI-for-age Z-score during Kalydeco treatment | 48 Months | |
| Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment | 48 Months | |
| Mortality: Incidence and cause of deaths | 48 Months | |
| Organ transplantation: Incidence and reason for organ transplantations | 48 Months |
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| Measure | Description | Time Frame |
|---|---|---|
| To explore the effect of Kalydeco treatment on Health-Related Quality of Life (HRQoL) in patients with CF and in caregivers of pediatric patients enrolled in the study. | 48 Months |
Inclusion Criteria:
Exclusion Criteria:
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Male and female patients with CF, age 6 years or older, who have 1 of the following CFTR mutations on at least 1 allele: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Bari | Italy | |||||
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| Messina |
| Italy |
| Milan | Italy |
| Milan | Italy |
| Naples | Italy |
| Potenza | Italy |
| Verona | Italy |
| The Hague | South Holland | Netherlands |
| Amsterdam | Netherlands |
| Gronningen | Netherlands |
| Heidelberglaan | Netherlands |
| Nijmegen | Netherlands |
| Rotterdam | Netherlands |
| London | Greater London | United Kingdom |
| Birmingham | West Midlands | United Kingdom |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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| ID | Term |
|---|---|
| C545203 | ivacaftor |
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