E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adult patients receiving allo-Stem Cell Transplantation using Cord Blood as source of stem cells |
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E.1.1.2 | Therapeutic area | Diseases [C] - Bacterial Infections and Mycoses [C01] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10010980 |
E.1.2 | Term | Cord blood transplant therapy |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10049085 |
E.1.2 | Term | Antifungal prophylaxis |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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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 pilot multicenter study will aim at assessing the safety of micafungin in patients receiving allo-SCT using CB as source of stem cells from the start of prophylaxis until the end of the 4-week post-treatment period. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study will aim at assessing the efficacy of micafungin in preventing fungal infection. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria:
•Adult patients receiving allo-Stem Cell Transplantation using Cord Blood as source of stem cells (cf conditioning regimen recommendation).
•Sex male or female
•Age between 18 and 65 years at the time of signing the informed consent form.
•Diagnosis of an hematologic disease for who a allograft decision has been taken
● not having a (HLA)-matched related or unrelated donor within two month after complete remission achievement
•Able to understand and voluntarily sign an informed consent form.
•Subjects affiliated with an appropriate social security system
•Male, female without childbearing potential or negative urine pregnancy test at the screening visit prior to beginning the treatment. Women of childbearing potential must be following adequate contraceptive measures. Men must agree to use an acceptable method of contraception (for themselves or female partners) for the duration of the study
•Each subject will weigh 40 kg or more
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E.4 | Principal exclusion criteria |
Exclusion criteria
•Any suspected or documented invasive fungal infection at study entry or at any time prior to study entry
•Use of any systemic antifungal therapy within 72 hours prior to study entry
•Known history of allergy, hypersensitivity or intolerance to echinocandin agents
•Patient with any medical, psychological or social condition, which in the opinion of the investigator could increase the risk to the patient, or decrease the chance of obtaining satisfactory data to achieve the objectives of this study.
•Previous participation in this study.
•HIV, HBV or HCV positive
•Pregnant or breast feeding females.
•Subject protected by law. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint:
- Safety assessed by the incidence and type of adverse events (AE) occurring during the course of prophylaxis treatment, AE related to study drug, AE leading to study drug discontinuation, and evolution of vital signs and biological parameters.
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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) |
Secondary endpoints:
-Incidence of Invasive Fungal Infection (IFI) at the end of prophylaxis period and at the end of the 4-week follow-up period
-Incidence of fever of unknown origin requiring empirical antifungal treatment during prophylaxis period
-Survival rate and incidence of mortality related to IFI from the start of prophylaxis until the end of the 4-week follow-up period.
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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 | Yes |
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 | 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 | 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.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
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 | No |
E.8.7 | Trial has a data monitoring committee | Yes |
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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |