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A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone
A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone

NCT01288573

CompletedPhase 1, Phase 2

Sponsor: Genzyme, a Sanofi Company

Conditions: Ewing's Sarcoma/Soft Tissue Sarcoma, Neuroblastoma, Brain Tumors

Interventions: plerixafor, plerixafor, plerixafor

Countries: Belgium, Czechia, Denmark, France, Germany, Hungary, Israel, Italy

This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages: * Stage 1 is a dose-escalation study. * Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study. All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative). Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.

Eligibility overview

Sex: ALL

Age: 1 Year to 18 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

* Age 2 to \< 18 years during stage 1 and 1 to \< 18 years during stage 2
* Ewing's sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, brain tumors or other malignancy (excluding any form of leukemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
* Eligible for autologous transplantation
* Recovered from all acute significant toxic effects of prior chemotherapy
* Adequate performance status (for patients ≥16 years of age, defined as Karnofsky score \>60 and for patients \<16 years of age, defined as Lansky score \>60)
* Absolute neutrophil count \>0.75 × 10\^9/L
* Platelet count \>50 × 10\^9/L
* Calculated creatinine clearance (using the Schwartz method): during study Stage 1, \>80 mL/min/1.73m\^2 and during study Stage 2, \>60 mL/min/1.73m\^2
* Aspartate aminotransferase(AST)/serum glutamic oxaloacetic transaminase(SGOT), alanine aminotransferase(ALT)/serum glutamic pyruvic transaminase (SGPT) and total bilirubin \<3 × upper limit of normal
* The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
* 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 mobilization treatment and for at least 3 months following any plerixafor treatment

Exclusion Criteria:

* Any form of leukemia
* A co-morbid condition which, in the view of the Investigator, renders the patient at high-risk from treatment complications
* Previous stem cell transplantation
* Persistent high percentage marrow involvement prior to mobilization will be prohibited.
* On-going toxicities (excluding alopecia) Grade ≥2 resulting from prior chemotherapy
* Acute infection
* Fever (temperature \>38.5°C) - if fever is between 37°C and 38.5°C, infection must be excluded as a cause
* Known HIV seropositivity, AIDS, hepatitis C or active hepatitis B infections
* Positive pregnancy test in post pubertal girls
* History of clinically significant cardiac abnormality or arrhythmia
* Use of an investigational drug which is not approved in any indication either in adults or pediatrics within 2 weeks prior to the first dose of G-CSF to be administered as part of the patient's planned standard mobilization 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
* The patient (and/or their parent/legal guardian), in the opinion of the Investigator, is unable to adhere to the requirements of the study
Locations (27)
  • Ghent, Belgium
  • Brno, Czechia
  • Praha 5 - Motol, Czechia
  • København Ø, Denmark
  • Lyon, France
  • Paris, France
  • Frankfurt am Main, Germany
  • Freiburg im Breisgau, Germany
  • Hamburg, Germany
  • Hanover, Germany
  • München, Germany
  • Budapest, Hungary
  • Petah Tikva, Israel
  • Tel Aviv, Israel
  • Genova, Italy
  • Milan, Italy
  • Padova, Italy
  • Roma, Italy
  • Torino, Italy
  • Amsterdam, Netherlands
  • Rotterdam, Netherlands
  • Krakow, Poland
  • Wroclaw, Poland
  • Barcelona, Spain
  • Madrid, Spain
  • Birmingham, United Kingdom
  • Glasgow, United Kingdom