E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Invasive aspergillosis in patients with haematological malignancies (HM)/autologous Hematopoietic Stem Cell Transplantation (HSCT) – group A- and patients with allogeneic HSCT transplantation – group B |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10003488 |
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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 |
The primary objective of this trial is to estimate the activity of caspofungin as first line therapy in the treatment of Invasive aspergillosis in patients with haematological malignancies (HM)/autologous Hematopoietic Stem Cell Transplantation (HSCT) – group A- and patients with allogeneic HSCT transplantation – group B. The activity of caspofungin will be assessed separately in these two groups of patients since response rates and survival differ substantially in these 2 patient populations.
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
- Patients eligible for this study will be patients 18 years of age or older with HM/auto-HSCT or allo-HSCT and with a diagnosis of proven or probable IA, according to EORTC-MSG criteria. All patients must be followed in centers belonging to the EORTC network. - The inclusion of patients with possible IA will be allowed. However, study drug treatment should be stopped if the case cannot be upgraded to probable or proven IA within 7 days after enrolment, based on tests already performed prior to or within 48 hrs after registration into the study, but with pending results. These include culture and/or histology results and Aspergillus galactomannan evaluations. - A course of no more than 72 hours of empirical antifungal therapy, not including an echinocandin, prior to enter into the study will be allowed. - Oral antifungal prophylaxis is allowed, but prophylaxis must be discontinued at study entry. - Intravenous fluconazole for prophylaxis is allowed, but prophylaxis must be discontinued at study entry. - Absence of a history of allergy or any adverse reaction to echinocandin drugs - Absence of severe renal failure requiring hemodialysis or peritoneal dialysis and a serum creatinine level lower than 300 µmol/L or 3.4 mg/dL - Absence of significant liver function test abnormalities, defined as an increase in ASAT/ALAT higher than 5x upper normal limits (UNL) or total serum bilirubin/ALP 5xUNL. - Patients with severe hepatic insufficiency are excluded - Karnofski performance status score ≥ 20. Comatose and moribund patients or those who have little hope of recovery are excluded - Absence of known bacterial infection not adequately treated at time of enrolment - Pregnant and lactating women are excluded. Women of child bearing potential should have a negative pregnancy test within 7 days from registration. - Patients receiving an investigational agent within 14 days prior to study entry or during the study course will be excluded. - Absence of documented HIV infection - Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial. - Before patient registration/randomization, written informed consent must be given according to ICH/GCP, and national/local regulations. |
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E.4 | Principal exclusion criteria | |
E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint of the study will be the response rate at the end of caspofungin therapy using the standard response assessment criteria as defined in the protocol. |
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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 | 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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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End of study occurs when all of the following criteria have been satisfied: 1. Thirty days after all patients have stopped protocol treatment 2. The trial is mature for the analysis of the primary endpoint as defined in the protocol 3. The database has been fully cleaned and frozen for this analysis |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
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 | 6 |