E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
All patients (0-65y) with a malignancy qualifying for allogenic hematopoietic stem cell transplantation (HSCT) who either (and/or) - are not eligible to other ongoing allo-HSCT protocols, because of disease or disease stage - relapsed after 1st transplant with a standerd allo HSCT protocol - have no conventional related or unrelated donor available |
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MedDRA Classification |
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 study the feasibility and safety (TRM/ a GVHD incidence) of co-infusion of selected haematopoietic cells from a haplo-identical donor and a single unit cord blood unit.
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E.2.2 | Secondary objectives of the trial |
engraftment rate full cord blood chimerism Immune reconstitution number of viral reactivations |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
All of the following five criteria: 1) All patients with a malignancy qualifying for allogenic hematopoietic stem cell transplantation (HSCT) (based on national or international study protocols) and either:
- Relapse after first transplant with a SIB or MUD/UCB donor - Having NHL or HD (refractory, ≥2CR) for which no standard allo-transplantation protocols are available, or ALL ≥CR2 if not eligible to other running SCT protocols. - Having relapse AML/ refractory AML - Problems finding a donor: no fully matched family donor or matched (9-10/10) unrelated donor available and / or no single or double unit cord blood available with sufficient cell numbers according to ongoing, open study protocols. 2) having a single matching (≥ 4/6) umbilical CB unit available with total NC count > 1,5 E7/kg [22] 3) Lansky / Karnofsky > 40 4) Age 0-65 * (*= age ≤ 65 and 364 days ) 5) Signed Informed Consent
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E.4 | Principal exclusion criteria |
• Creatinine clearance < 40 ml/min • Cardiac dysfunction (SF < 45%) (Ejection fraction < 45%), unstable angina, or unstable cardiac arrhythmias • Pulmonary function test VC, FEV1 and/ or DOC< 50%
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E.5 End points |
E.5.1 | Primary end point(s) |
• The cumulative incidence of TRM (non relapse mortality < 100 days) • Acute- GVHD (Grades II-IV: Gluckberg Criteria)
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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 | 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) | Yes |
E.7.1.1 | First administration to humans | Yes |
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 | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Prospective study, Phase l/ll trial with an optimal 2-stage design |
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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.8.5 | 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 | 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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the inclusion periode is 3 years from the start of the sudy. 100 days after the last inclusion the study has ended |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 10 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 10 |