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 - ND

Phase 1

• Conditions: Paediatric cancer patients (aged 2 to < 18 years) with Ewing’s sarcoma/soft tissue sarcoma, neuroblastoma, brain tumours, and all other malignancies (excluding leukaemia) who are planned to undergo high dose chemotherapy followed by autologous haematopoietic stem cell transplantation (HSCT) rescue; MedDRA version: 9.1Level: HLGTClassification code 10041299; MedDRA version: 9.1Level: PTClassification code 10029260; MedDRA version: 9.1Level: HLGTClassification code 10029211

• Registration Number: EUCTR2010-019340-40-IT
• Lead Sponsor: Genzyme Europe BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-12-22
• Last Update Posted: 19 February 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 67

Inclusion Criteria

1. Age 2 to <18 years 2. Ewing’s sarcoma, soft tissue sarcoma, neuroblastoma, brain tumours or other malignancy (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 >1.0 ? 10P9/L 7. Platelet count >75 ? 10P9/L 8. Calculated creatinine clearance (using the Schwartz method): – during study Stage 1, >80 mL/min/1.73mP2 – during study Stage 2, >60 mL/min/1.73mP2 9. Aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) and total bilirubin <3 ? upper limit of normal 10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Any form of leukaemia 2. A co-morbid condition which, in the view of the Investigator, renders the patient at high-risk from treatment complications 3. Previous stem cell transplantation 4. Patients with tumours frequently involving bone marrow (e.g., lymphomas and neuroblastoma) will be expected to have had evaluation of their marrow as part of their standard staging evaluations. Persistent high percentage marrow involvement prior to mobilisation will be prohibited. (Specific guidelines for different indications are provided in Section 9.2.1 with details of bone marrow examination requirements at Screening) 5. A residual acute medical condition 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. Pulse oximetry =92% 9. Known HIV positive 10. Positive pregnancy test in post pubertal girls 11. History of clinically significant cardiac abnormality or arrhythmia 12. Use of an investigational drug which is not approved in any indication either in adults or paediatrics within 4 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. Drugs approved for other indications that are being used in a manner considered standard of care for this transplant procedure are allowed 13. The patient (and/or their parent/legal guardian), in the opinion of the Investigator, is unable to adhere to the requirements of the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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. E.;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.;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.