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.1.3 | 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 program is to provide expanded access to aztreonam lysine for inhalation 75 mng prior to its
commercial availability to patients with cystic fibrosis and chronic P. aeruginosa 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 |
> 6years 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:
-First Cohort Criteria:
Those who are waitlisted or eligible for lung transplant based on FEV1 criteria.
Patients who have a level of lung function impairment consistent with lung transplantation criteria, but who are ineligible for transplantation for other reasons, can enroll in this program.
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.
- Second Cohort Criteria:
Meets first cohort criteria; or
FEV1 < 40 % predicted at the time of consent
- Third Cohort Criteria:
Meets first or second cohort criteria; or
FEV1 < 50 % predicted at the time of consent |
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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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 4 |