E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Mobilisation of peripheral blood stem cells prior to autologous stem cell transplantation in patients with Non-Hodgkin’s Lymphoma (NHL), Hodgkin’s Disease (HD) or Multiple Myeloma (MM). |
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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 | 10029547 |
E.1.2 | Term | Non-Hodgkin's lymphoma |
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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 |
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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 | 10028228 |
E.1.2 | Term | Multiple myeloma |
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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 |
Confirm the safety profile of plerixafor to mobilise stem cells when used in patients with lymphoma or multiple myeloma who are eligible to undergo treatment with an autologous haematopoietic stem cell transplant |
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E.2.2 | Secondary objectives of the trial |
- Assess the efficacy of plerixafor and G-CSF as a mobilisation regimen as measured by the number of CD34+ cells collected in each apheresis session - Assess the clinical effectiveness of plerixafor and G-CSF mobilised stem cells by examining haematopoietic cell engraftment and graft status - Examine the influence of CD34+ cell dose infused on time to engraftment, engraftment and graft status
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients must meet the following criteria to be enrolled in the study: 1. Diagnosis of MM, NHL, or HD in partial response (PR) or complete response (CR) 2. Eligible and planned for an autologous haematopoietic stem cell transplantation 3. Written informed consent 4. At least 18 years of age (inclusive) 5. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 6. White blood cell (WBC) count ≥2.5 x 10E9/L 7. Absolute neutrophil count (ANC) ≥1.5 x 10E9/L 8. Platelet count ≥100 x 10E9/L 9. Serum creatinine equal or less than 2.2 mg/dL 10. AST/SGOT, ALT/SGPT, and total bilirubin less than 2.5 x upper limit of normal (ULN) 11. Adequate cardiac, renal, and pulmonary function sufficient to undergo apheresis and transplantation, i.e., eligible by institutional standards for autologous stem cell transplant 12. All patients must agree to use a highly effective method of contraception whilst on study treatment and for at least 3 months following plerixafor treatment (including both female patients of child-bearing potential and male patients with partners of child-bearing potential). Effective birth control includes: a) birth control pills, depot progesterone, or an intrauterine device (IUD) plus one barrier method, or b) two barrier methods. Effective barrier methods are: male and female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm). For patients using a hormonal contraceptive method, information about any interaction of plerixafor with hormonal contraceptives is not known.
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E.4 | Principal exclusion criteria |
Patients who meet any of the following criteria will be excluded from the study: 1. History of any acute or chronic leukaemia (including myelodysplastic syndrome) 2. Prior allogeneic transplantation or more than one prior autologous transplantation 3. Failed previous CD34+ cell collection attempts (either due to insufficient yield in apheresis product, or ineligible for apheresis because of inadequate mobilisation of CD34+ cells into peripheral blood) 4. Less than 4 weeks since last anti-cancer therapy (including chemotherapy, biologic/immunologic, radiation) or less than 6 weeks if prior therapy with nitrosourea or mitomycin (for therapies with long-acting agents, a treatment-free interval of at least 2 half-lives should be considered) with the exception of - Treatment with thalidomide, dexamethasone, lenalidomide (Revlimid R), and/or bortezomib (Velcade R) which is allowed up to 7 days prior to the first dose of G-CSF. 5. Bone marrow involvement >20% assessed based on the most recent bone marrow aspirate or biopsy 6. Treated with G-CSF or other cytokine within 14 days prior to the first dose of G-CSF for mobilisation 7. Known to be HIV positive 8. Active hepatitis B or hepatitis C 9. Acute infection (febrile, i.e., temperature >38°C) within 24 hours prior to dosing or antibiotic therapy within 7 days prior to the first dose of G-CSF 10. Hypercalcaemia as evidenced by >1 mg/dL above ULN 11. Previously received investigational therapy within 4 weeks of enrolling in this protocol or currently enrolled in another investigational protocol during the mobilisation phase 12. Central nervous system involvement including brain metastases or leptomeningeal disease 13. Pregnant or nursing women 14. Electrocardiogram (ECG) or study result (exercise study, scan) indicative of cardiac ischaemia or a history of clinically significant rhythm disturbance (arrhythmias), or other conduction abnormality in the last year that in the opinion of the Investigator warrants exclusion of the subject from the trial. 15. Co-morbid condition(s), which in the opinion of the Investigator, renders the patient at high risk from treatment complications or impairs their ability to comply with the study treatment and protocol
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy will be based on the quantity of CD34+ cells harvested and the subsequent engraftment and graft status: • Number of days to neutrophil engraftment and platelet engraftment • Increase in number of circulating CD34+ cells from time 0 to 10-11 hours after the first dose of plerixafor • Total number of CD34+ cells collected by disease type • Total number of CD34+ cells collected per apheresis day • Graft status (as measured by trilineage counts and supportive treatment, e.g., transfusion, G-CSF) at 100 days, 6 months, and 12 months following transplant
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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 | 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 | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
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 | Information not present in EudraCT |
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
| 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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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 | 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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 24 |
E.8.9.2 | In all countries concerned by the trial days | 0 |