Treatment regimen, safety and efficacy of plerixafor in children

• Research type

  Research Study

• Full title

  A Phase 1/2 Combined Dose Ranging and Randomised, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilisation of Haematopoietic Stem Cells into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilisation Regimens Alone in Paediatric Patients, Aged 2 to <18 Years, with Solid Tumours Eligible for Autologous Transplants

• IRAS ID

  57633

• Contact name

  Bruce Morland

• Sponsor organisation

  Genzyme Europe BV

• Eudract number

  2010-019340-40

• ISRCTN Number

  NA

• Research summary

  The aim of this study is to confirm the appropriate dose, the efficacy and the safety of the drug plerixafor in cancer patients under the age of 18. The best working dose will be determined across age and size of the patients. The drug will be given to the patients who need transplantation with their own stem cells. Plerixafor improves the release of stem cells from the bone marrow where the stem cells are made into the blood stream (mobilisation). Plerixafor will be given in addition to the standard treatment these patients get to collect stem cells for the transplant. The study will be conducted in 2 stages. Patients will participate in either Stage 1 or Stage 2, not in both. In Stage 1 different doses of plerixafor will be compared to determine the most appropriate dose. In Stage 2 patients will be randomly divided into two groups. One third of the patients will be given the standard treatment and the rest of the patients will be given the most appropriate dose of plerixafor in addition to the standard treatment. The results of these two groups will be compared to confirm efficacy of plerixafor. In both stages of the study eligible patients are: aged 2 to <18 years, with Ewing’s sarcoma/soft tissue sarcoma, neuroblastoma, brain tumours, or all other malignancies (excluding leukaemia), for whom high dose chemotherapy is planned, followed by a transplantation of stem cells derived from the bone marrow. The study will be done at approximately 15 different hospitals in the UK, France, Germany and Italy. At least 27 patients will be included in the first stage of the study. In stage two at least 40 patients will be included. Both stages of the study will each take about 30 months.

• REC name

  West Midlands - Coventry & Warwickshire Research Ethics Committee

• REC reference

  10/H1211/25

• Date of REC Opinion

  4 Aug 2010

• REC opinion

  Favourable Opinion