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 1 to <18 Years, with Solid Tumours Eligible for Autologous Transplants

Phase 2

Completed

Conditions: solide tumoren
solid tumor

Registration Number: NL-OMON40176

Lead Sponsor: Sanofi-aventis

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 3

Inclusion Criteria

1. Age 1 to < 18 years
2. Ewing*s sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, or other malignancy including brain tumours (excluding any form of leukaemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
3. Eligible for autologous transplantation
4. Recovered from all acute significant toxic effects of prior chemotherapy
5. Adequate performance status
- for patients >=16 years of age, defined as Karnofsky score >60
- for patients <16 years of age, defined as Lansky score > 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 < 3 × upper limit of normal
10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent

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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary efficacy endpoint will be the difference between the 2 treatment<br /><br>arms in the proportion of patients achieving at least a doubling of peripheral<br /><br>blood CD34+ count from the morning of the day preceding the first apheresis day<br /><br>to the morning prior to apheresis.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• Number of days of apheresis required to reach >=2 × 106 CD34+ cells/kg<br /><br>• CD34+ yield for each apheresis<br /><br>• Total CD34+ yield<br /><br>• Percentage of patients proceeding to transplant<br /><br>• Percentage of patients successfully engrafting<br /><br>• Percentage of patients with durable engraftment at 3, 6, 12 and 24 months<br /><br>post-transplant</p><br>