E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Study on the impact of stem cell donation either after mobilisation with granulocyte colony stimulating factor (GCSF) or bone marrow harvest on unrelated donors. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10051716 |
E.1.2 | Term | Peripheral blood stem cell apheresis |
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 aim of this study is two fold a) Detection of any genetic differences between bone marrow and PBSC unrelated donors, 3-5 years post donation and confirm or refute the observations of “long-term genetic or epigenetic effects” published by Nagler et al. (completed – none detected see below) b) Screen and compare PBSC donors lymphocytes pre and up to 6 months post G-CSF exposure to detect any ‘short-term’ genetic changes also described by Nagler et al. The employment of more sensitive methods such as iFISH and gene array analysis to assess any permanent genetic changes, our primary objective, will make our study more robust.
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Pre-existing healthy volunteer unrelated donors (who have met strict and extensive entry criteria) on the Anthony Nolan and British Bone Marrow Registries.
Aged 18-60 years.
Minimum weight 51 Kgs.
Participants in the prospective arm are able to undergo peripheral blood collection (apheresis)
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E.4 | Principal exclusion criteria |
Pregnant or breastfeeding female.
Family history of haematological malignance.
Any related donor.
Previously donated bone marrow or PBSC's
Hypersensitive to Lenograstim (or its excipients; arginine, phenlanine, methionine, mannitol (E421) or polysorbate 20).
Weight under 51 Kgs.
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E.5 End points |
E.5.1 | Primary end point(s) |
The binary variable genetic change i.e. clonal aberrations detected in any one of chromosomes 7, 8 and 17.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
There will be two arms:
iii) Retrospective arm – time points 3 to 5 years post donation. (completed)
iv) Prospective arm - Peripheral blood of 50 unrelated PBSC donors only will be examined at the following time points: prior to donation and before exposure to G-CSF (minus 2-3 wks); at donation and after exposure to G-CSF at Day 0 (on the day of Apheresis) and Day 90 +/- 14 days and Day 180 +/- 14 days post-donation. |
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E.5.2 | Secondary end point(s) |
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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 | No |
E.6.3 | Therapy | No |
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) | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | 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
| Information not present in EudraCT |
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 | 7 |
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 Trial shall end when the last sample has been collected from the last trial subject and the analysis of all samples has been completed. |
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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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |