E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adult and paediatric cystic fibrosis (CF) patients with pulmonary Pseudomonas aeruginosa (PA) infection. |
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E.1.1.1 | Medical condition in easily understood language |
Cystic fibrosis (CF) in adults and children with a lung infection caused by the bacteria Pseudomonas aeruginosa (PA). |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to provide expanded access to AZLI 75 mg prior to its commercial availability and establishment of reimbursement programs through Provincial Ministries of Health to patients in Canada with CF and PA airway infection who have limited treatment options and are at risk for disease progression. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
> 6 years of age
Patient has CF as diagnosed by one of the following:
-Documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis
test, or
-Two well characterized genetic mutations in the CFTR gene, or
-Abnormal nasal potential difference with accompanying symptoms characteristic of
CF
PA present in expectorated sputum or throat swab culture within 2 months prior to
consent
Patient must be able to provide written informed consent/assent prior to any study related
procedure; parent/guardian must be able to give written informed consent as necessary
prior to any study related procedure
At high risk for disease progression as defined by one of the following patient
populations:
FEV1 < 50 00 predicted at the time of consent
Or
Completed participation in CP-Al-006 (through Visit 20). Patients who withdraw
from CP-Al-006 prior to completing all courses of AZLI and all study visits will
not be eligible for this protocol. |
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E.4 | Principal exclusion criteria |
Patients with any serious or active medical or psychiatric illness that, in the opinion of the investigator, would interfere with patient treatment, assessment or compliance with the protocol or dosing requirements
Patients with hypersensitivity to any of the components of the drug product
Currently enrolled in another clinical trial
Pregnant or lactating females |
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E.5 End points |
E.5.1 | Primary end point(s) |
Not applicable: Expanded Access Program |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 10 |