E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with lymphoma or myeloma, who require high dose chemotherapy with autologous stem cell rescue (known colloquially as an autograft). Plerixafor is used to mobilise the autologous stem cells for routine harvesting, a few weeks before the transplant. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10053946 |
E.1.2 | Term | Stem cell mobilization |
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 | 14.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10019028 |
E.1.2 | Term | Blood and blood product treatment |
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 | 14.0 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10042613 |
E.1.2 | Term | Surgical and medical procedures |
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 | 14.0 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10025309 |
E.1.2 | Term | Haematological and lymphoid tissue therapeutic procedures |
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 | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10053943 |
E.1.2 | Term | Haematopoietic stem cell mobilisation |
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 |
Using plerixafor plus granulocyte-colony stimulating factor (G-CSF), is the yield of stem (CD34+) cells at least as good as can be obtained with conventional chemotherapy plus G-CSF?, and can this be accomplished in the same or fewer stem cell collections (known as aphereses)?
Stated more formally, the primary study endpoint is a composite of BOTH an adequate stem cell harvest (at least 4 x 10 E6 CD34+ cells per kg body weight in no more than 2 aphereses) AND a neutrophil count that never falls below 1.0 x 10 E9 / Litre in the 3 weeks following initiation of mobilisation. |
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E.2.2 | Secondary objectives of the trial |
Secondary endpoints include the total stem cell yield in 1-2 aphereses, serial neutrophil and platelet counts in the blood during mobilisation, the time to neutrophil and platelet engraftment after subsequent stem cell transplantation (SCT), the usage of plerixafor and the number and timing of apheresis collections.
These data will be compared to those in an immediately preceding consecutive cohort of 120 myeloma and lymphoma patients mobilised with chemotherapy plus G-CSF. This comparator population is suitable, as the case mix and induction therapies are unlikely to change significantly from that of the control group during the period of the study.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
All referrals to the transplant programme at our hospital are invariably seen by one of the investigators. All eligible patients will be offered entry into the trial at that initial visit.
Inclusion criteria; all of the following must be satisfied:
Aged 18 or over
Able to give informed written consent.
Diagnosis of EITHER multiple myeloma or related plasma cell dyscrasia, OR any form of lymphoma or associated lymphoproliferative disease
Autologous stem cell transplantation is planned as the next course of treatment.
The patient has not previously undergone a mobilisation attempt for the current transplant. Patients who have received previous autologous transplants at least 2 years previously are eligible, as long as stem cell mobilisation has not been attempted for the current transplant.
No serious concomitant illness (e.g. heart disease) that might preclude completion of the study.
Creatinine clearance of at least 30 mls/min. Dose reduction of plerixafor is required where the creatinine clearance is between 30-50 mls/min.
Negative pregnancy test in women of childbearing age. |
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E.4 | Principal exclusion criteria |
Exclusion criteria are ANY of the following:
Unable to give informed written consent
Pregnancy or lactating
Creatinine clearance of less than 30 mls/min. Patients with clearances lower than this may still be able to receive plerixafor at reduced dosage following discussion with the trial co-ordinators, but are not eligible for entry into this trial.
Any previous attempt at mobilisation for the current transplant.
Patients with any form of leukaemia, INCLUDING PLASMA CELL LEUKAEMIA, are not eligible.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary study endpoint is a composite of BOTH an adequate stem cell harvest (at least 4 x 10 E6 CD34+ cells per kg body weight in no more than 2 aphereses) AND a neutrophil count that never falls below 1.0 x 10 E9 / Litre in the 3 weeks following initiation of mobilisation.
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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 | 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) | Yes |
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 | 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 | No |
E.8.7 | Trial has a data monitoring committee | No |
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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12 months following the transplant date of the last entrant into the study. |
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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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |