Paediatric cancer patients (aged 1 to <18 years) with Ewing’s sarcoma/soft tissue sarcoma, lymphoma, neuroblastoma and all other malignancies (excluding leukaemia) who are planned to undergo high dose chemotherapy followed by autologous haematopoietic stem cell transplantation (HSCT) rescue
Conditions
Interventions
Trade Name: Mozobil
Product Name: Plerixafor
Pharmaceutical Form: Solution for injection
INN or Proposed INN: plerixafor
CAS Number: 110
Sponsors
Genzyme Europe B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age 1 to 60 – for patients 60 6. Absolute neutrophil count >0.75 × 10P9/L 7. Platelet count >50 × 10P9/L 8. Calculated creatinine clearance (using the Schwartz method): – during study Stage 2, >60 mL/min/1.73mP2 9. Liver functions =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any form of leukaemia 2. A co-morbid condition, such as ventricular arrhythmias, which, in the view of the Investigator, renders the patient at high-risk from treatment complications 3. Previous stem cell transplantation 4. Patients with persistent high percentage marrow involvement prior to mobilisation will be prohibited. 5. On-going toxicities (excluding alopecia) Grade =2 resulting from prior chemotherapy 6. Acute infection 7. Fever (temperature >38.5°C) - if fever is between 37°C and 38.5°C, infection must be excluded as a cause 8. Known HIV seropositivity, AIDS, hepatitis C or active hepatitis B infections. 9. Positive pregnancy test in post pubertal girls 10. History of clinically significant cardiac abnormality or arrhythmia 11. Use of an investigational drug which is not approved in any indication either in adults or paediatrics within 2 weeks prior to the first dose of G-CSF to be administered as part of the patient’s planned standard mobilisation regimen, and/or during the study up until engraftment of the transplant. If patients are on investigational drugs as part of their anti-cancer regimen, this should be discussed with the Sponsor before screening. Drugs approved for other indications that are being used in a manner considered standard of care for this transplant procedure are allowed 12. The patient (and/or their parent/legal guardian), in the opinion of the Investigator, is unable to adhere to the requirements of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Up to 5 days;Secondary Objective: The secondary objective of this study is to confirm the efficacy and safety of plerixafor in addition to standard mobilisation of HSCs into peripheral blood, and subsequent collection by apheresis, in paediatric cancer patients.;Main Objective: The primary objective of this study is to confirm the appropriate dose and efficacy, and to characterise the safety, pharmacokinetics and pharmacodynamics of plerixafor across age and size in paediatric cancer patients when given in addition to standard mobilisation of HSCs into peripheral blood.;Primary end point(s): The primary efficacy endpoint will be the difference between the 2 treatment arms in Stage 2 (comparative part of the study) in the proportion of patients achieving at least a doubling of peripheral blood CD34+ count from the morning of the day preceding the first apheresis day to the morning prior to apheresis. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Number of days of apheresis required to reach =2 × 10^6 CD34+ cells/kg [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2 •Yield of CD34+ cells for each apheresis [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2 •Total CD34+ cell yield [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2 •Percentage of patients proceeding to transplant [ Time Frame: Within 6 months of last apheresis ] During Stage 1 and Stage 2 •Percentage of patients successfully engrafting [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2 •Percentage of patients with durable engraftment [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2 •Summary of adverse events (AEs) [ Time Frame: Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis) ] During Stage 1 and Stage 2 •Duration of hospitalizations (planned or unplanned) [ Time Frame: Throughout the duration of the study ] During Stage 1 and Stage 2 •Mobilization of tumor cells into peripheral blood [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2 •Relapse rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2 •Occurrence of secondary malignancies [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2 •Incidence of primary and secondary graft failure [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2 •Time to secondary graft failure [ Time Frame: Up to 24 months posttransplant] During Stage 1 and Stage 2 •Survival rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 | — |
Countries
Belgium, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Netherlands, Poland, Spain, United Kingdom
Contacts
Public ContactMedical Information
Genzyme Europe B.V.
Outcome results
None listed