E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
High-risk haematological malignancies (including AML, ALL, CML, NHL, HD, and MDS) with an indication for allogeneic haematopoietic stem cell transplantation, to support haematopoietic reconstitution following myeloablative therapy.
Neoplasias hematológicas de alto riesgo (incluyendo LMA, LLA, LMC, SMD, EH y LNH), con indicación de transplante de líneas celulares hematopoyéticas alogénicas como apoyo para la reconstitución hematopoyética tras el tratamiento mielosupresor. |
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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 | 10000886 |
E.1.2 | Term | Acute myeloid leukemia |
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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 | 10009015 |
E.1.2 | Term | Chronic myeloid leukemia |
|
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 | 10001690 |
E.1.2 | Term | ALL |
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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 | 10028533 |
E.1.2 | Term | Myelodysplastic syndrome |
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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 | 10020206 |
E.1.2 | Term | Hodgkin's disease |
|
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 | 10029547 |
E.1.2 | Term | Non-Hodgkin's lymphoma |
|
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 | 10001756 |
E.1.2 | Term | Allogenic bone marrow transplantation therapy |
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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 |
Assess the effect of StemEx transplantation on overall 100-day mortality. |
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E.2.2 | Secondary objectives of the trial |
? Time from infusion of CB cells to overall mortality at 100 days. ? Proportion of overall mortality at 180 days. ? Time from infusion of CB cells to overall mortality at 180 days. ? Proportion of subjects who developed acute Graft versus Host Disease (GvHD) grades III-IV. ? Proportion of subjects with graft failure |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
a. Subject is a candidate for allogeneic myeloablative SCT for treatment of one of the following haematologic malignancies:
a.1 Clinical diagnosis of AML: CR2 or subsequent complete remission (CR) or CR1 with high-risk features including any of the following: stem cell or biphenotypic classification (AML-M0), erythroleukemia (AML-M6), acute megakaryocytic leukemia (AML-M7), high-risk cytogenetics, or failure to achieve CR after standard induction therapy or presentation with extramedullary disease or relapse with < 10% blasts in BM and no circulating blasts.
a.2 Clinical diagnosis of ALL: CR2 or subsequent CR or CR1 with high-risk features including: high-risk cytogenetics or with failure to achieve CR after standard induction therapy or elevated WBC at presentation (greater/equal 50,000 age <17 or greater/equal 20,000 age greater/equal 17) or presentation with extramedullary disease or relapse with < 10% blasts in BM and no circulating blasts.
a.3 Clinical diagnosis of CML: in CP1 (Chronic Phase 1) and resistant or intolerant to Gleevec or in CP2 or subsequent CP or in accelerated phase.
a.4 Clinical diagnosis of HD: induction failure or relapse and sensitive to last chemotherapy course.
a.5 Clinical diagnosis of NHL induction failure or relapse and sensitive to last chemotherapy course.
a.6 Clinical diagnosis of MDS with intermediate 2- or high-risk IPSS score.
b. Age 12 to 55.
c. Availability of CBU meeting requirements as laid down in protocol.
d. Subject's performance score greater or equal 70% by Karnofsky (age greater or equal 13) or a Lansky Play-Performance scale of greater or equal 70% (age above 13).
e. Subject has sufficient physiologic reserves as laid down in the protocol.
f. Subject must have either of the following back-up stem cell sources in case of engraftment failure:
f.1 Subject willing to undergo BM harvest or peripheral blood progenitor cell collection for use in case of engraftment failure.
f.2 Subject has a second CBU as a possible back up.
f.3 Subject's haploidentical family member has been identified and agreed to donate haematopoietic stem cells in case of engraftement failure.
g. Subject or guardian signs written informed consent. |
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E.4 | Principal exclusion criteria |
a. Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except Hydroxyurea).
b. HIV positive.
c. Pregnancy or lactation.
d. Uncontrolled bacterial, fungal or viral infection.
e. Subjects with signs and symptoms of active central nervous system (CNS) disease.
f. Availability of appropriate related and willing stem cell donor, who is HLA-matched at 5 or 6/6 antigens.
g. Prior allogeneic cell transplant.
h. Allergy to bovine proteins or to aminoglyoside antibiotics (e.g. gentamicin) or to any product, which may interfere with the treatment.
i. Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall 100 day mortality |
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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 | 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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Historical Cohort Controlled study |
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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 |
Historical controls of international cord blood transplantation registries (CIBMTR, NCBP, EuroCord). |
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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.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 | 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
| |
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 | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |