E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis (ABPA) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10021881 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the tolerability of Amphotericin B lipid complex (Abelcet) by aerosol with a special focus on kidney involvement |
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E.2.2 | Secondary objectives of the trial |
To evaluate the decrease of total and specific IgE values at the end of the treatment (at the 4th and 8th week of treatment) − To evaluate the FEV1 variation − To evaluate the quality of life |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Males/females age > = 12 years. 2. Cystic Fibrosis diagnosed by either gene profiling and/or sweat test. 3. ABPA diagnosis defined as: i) Acute or sub-acute clinical deterioration (cough, wheeze, exercise intolerance, exercise-induced asthma, change in pulmonary function, or increased sputum production) not attributable to another etiology. ii) Total serum IgE concentration of >500 IU/mL (1200 ng/mL). iii) presence of IgE antibody to A. fumigatus. iv) One of the following: a) presence of precipitans to A.fumigatus or b) new or recent abnormalities on chest radiography (infiltrates or mucus plugging) or chest CT (bronchiectasis) that has not cleared with antibiotics and standard physiotherapy. 4. Total serum IgE level of > 500 IU/ml (will be measured locally at Screening Visit). 5. Patients who present with an acute episode of ABPA, either as a first presentation or as a recurrence, and who were taking a maximum dose of prednisolone of 20mg/day (or equivalent) 6. FEV1 > = 40% 7. Suitable contraceptive measure 8. Prior to administration of any study procedures, eligible patients (and parents for patients below age 18) must provide written informed consent |
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E.4 | Principal exclusion criteria |
1. History of cancer in the past 10 years (except surgically-cured basal cell or squamous cell skin cancer). 2. Any previous history of anaphylaxis. 3. Any other medical condition that in the opinion of the investigator may cause the patient to be unsuitable for completion of the study or place the patient at potential risk from being in the study. 4. Pregnant and lactating women. 5. Lung or other transplant or in a transplantation list. 6. Participation in any clinical trial within four (4) weeks prior to initial dosing. 7. Significant illness other than CF/ABPA within two (2) weeks prior to initial dosing. 8. Patients who are known to be positive for chronic atypical Mycobacteria and Burkholderia cepacia including subspecies. 9. Subjects must be able to communicate well with the investigator; understand and comply with the requirements of the study; and understand and sign the written informed consent (parental consent and assent for minors, if applicable). 10. medical history of kidney problems defined like creatinine values equal the double of normal value 11. intake of itraconazole in the precedent 15 days before the screening visit. 12. steroidal therapy > 20 mg/die |
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E.5 End points |
E.5.1 | Primary end point(s) |
Appearance of an adverse event defined like an increase of creatinine between the beginning and the end of the treatment |
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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 | No |
E.6.4 | Safety | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
tollerabilita` dell`Amfotericina B complesso lipidico per via aerosolica |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |
E.8.9.2 | In all countries concerned by the trial days | 0 |