E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with high-risk hematological diseases, who need a allogeneic
stem cell transplantation but who lack a matched unrelated donor |
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E.1.1.1 | Medical condition in easily understood language |
Patients with high-risk hematological diseases, who need a allogeneic
stem cell transplantation but who lack a matched unrelated donor |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061187 |
E.1.2 | Term | Haematopoietic neoplasm |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 |
To study the feasibility of single UCBT using one SR-1 expanded unit |
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E.2.2 | Secondary objectives of the trial |
To assess engraftment and engraftment kinetics; to evaluate immune reconstitution, acute and chronic GVHD, chimerism, toxicity, progression-free survival and overall survival after single unit UCBT, using with one ex-vivo expanded unit. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Age 18-70 years inclusive
- Diagnosis of poor-risk hematological malignancy or (V)SAA relapsing after or failing immunosuppressive therapy and meeting the criteria for a MUD allo SCT
- Lacking a sufficiently matched volunteer unrelated donor or lacking such a donor within the required time period of ≤ 2 months in case of urgently needed alloSCT
- Availability of 1 (≥5/6) matched UCB graft with a nuclear cell count > 2,7 x 107/kg (see paragraph 8.2).
- Availability of an back-up autograft, harvested and frozen earlier in the course of treatment, (harvest according to local aphersis policies)
- WHO performance status 0-2
- Written informed consent
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E.4 | Principal exclusion criteria |
• Bilirubin and/or transaminases > 2.5 x normal value
• Creatinine clearance < 40 ml/min
• Cardiac dysfunction as defined by:
-Reduced left ventricular function with an ejection fraction < 45% as measured by MUGA scan or echocardiogram (another method for measuring cardiac function is acceptable)
- Unstable angina
- Unstable cardiac arrhythmias
• Pulmonary function test with VC, FEV1 and/ or DCO < 50%
• Active, uncontrolled infection
• History of high dose (≥ 8 Gy) total body irradiation
• Pregnant or lactating females
• HIV positivity
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E.5 End points |
E.5.1 | Primary end point(s) |
Feasibility as defined by, and to be achieved in ≥ 80% of (evaluable) patients:
1. SR-1 mediated expansion, resulting in > 20-fold expansion of CD34+ cells, and
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
at preparation Expanded UCB-derived HSPCs |
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E.5.2 | Secondary end point(s) |
effective hematopoietic (neutrophils > 0.5 x 109/L) engraftment within 30 days upon transplantation
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
within 30 days upon transplantation
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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 | 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 | 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 | 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 | 2 |
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
|
Last patient last visit (5 years after registration last patient) |
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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 | 0 |