A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone
- Conditions
- 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) rescueTherapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2010-019340-40-NL
- Lead Sponsor
- Genzyme Europe B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 70
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 :
Aspartate aminotransferase/serum glutamic oxaloacetic transaminase
(AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase
(ALT/SGPT) and total bilirubin
10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
11. Patients who are sexually active must be willing to abstain from sexual intercourse or agree to use an approved form of contraception while receiving plerixafor and/or standard mobilisation treatment and for at least 3 months following any plerixafor treatment.
Are the trial subjects under 18? yes
Number of subjects for this age range: 70
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
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
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.;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.;Timepoint(s) of evaluation of this end point: Up to 5 days
- Secondary Outcome Measures
Name Time Method