E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | LLT |
E.1.2 | Classification code | 10037316 |
E.1.2 | Term | Pulmonary alveolar proteinosis |
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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 |
Total resolution of the disease in 100% of patients treated with WLL/inhaled GM-CSF. |
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E.2.2 | Secondary objectives of the trial |
1)to avoid recurrence of the disease requiring multiple WWL, 2) to avoid or reduce the number of respiratory infections due to persistant pulmonary abnormalities PAP associated 3) evaluation of outcome determinants in the diverse treated groups, 4) ancillary study to establish whether the addition of inhaled GM-CSF may influence PAP pathophysiology. |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
ALTRI SOTTOSTUDI: Valutare se la somministrazione di Sargramostim inalatorio aggiunto a lavaggio polmonare massivo puo` influenzare la fisiopatologia della PAP (conta dei CD11b circolanti).
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E.3 | Principal inclusion criteria |
autoimmune PAP diagnosis; age >= 18 years; persistant or progressive respiratory failure (PaO2 < 60 mmHg) or latent respiratory failure (SatO2 < 90% or desaturation > 5% in Bruce`s test) or persistant pulmonary TC abnormalities (intensity score >=3) (reasidual disease treated group) |
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E.4 | Principal exclusion criteria |
non autoimmune PAP; contraindications to WLL; contraindication to GM-CSF; pregnancy; refusal to the use of contraceptive methods during the study period; contraindications to the use of inhaled medicines. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary efficacy endpoint (total resolution of disease in 100% of patients trated with WLL/inhaled GM-CSF) will be documented as follows: a. functional endpoints: measuere of alveolar-arterial oxygen tension gradient (PA-a O2), measure of oxygen saturation during exercise, according to the modified Bruce`s protocol. b. Imaging endpoint: score of the pulmonary CT performed after 3 and 10 months after WLL. c. Severity score: score based on symptoms and PaO2 level at entry. The score will be associated to a quality of life questionnaire (SF36). |
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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 | Yes |
E.6.5 | Efficacy | Yes |
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) | 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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
Farmaco associato a WLL versus solo WLL |
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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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 6 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 0 |