Overview

This trial is active, not recruiting.

Conditions ewing's sarcoma/soft tissue sarcoma, neuroblastoma, brain tumors
Treatments plerixafor
Phase phase 1/phase 2
Sponsor Genzyme, a Sanofi Company
Collaborator Sanofi
Start date March 2014
End date May 2017
Trial size 30 participants
Trial identifier NCT01288573, 2010-019340-40, DFI12860, MOZ15609

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Patients will receive subcutaneous (SC) injection of 160 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
plerixafor
160 μg/kg subcutaneous (SC) injection
(Experimental)
Patients will receive subcutaneous (SC) injection of 240 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
plerixafor
240 μg/kg subcutaneous (SC) injection
(Experimental)
Patients will receive subcutaneous (SC) injection of 320 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
plerixafor
320 μg/kg subcutaneous (SC) injection

Primary Outcomes

Measure
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
time frame: Up to 5 days

Secondary Outcomes

Measure
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
time frame: Up to 5 days
Yield of CD34+ cells for each apheresis
time frame: Up to 5 days
Total CD34+ cell yield
time frame: Up to 5 days
Percentage of patients proceeding to transplant
time frame: Within 6 months of last apheresis
Percentage of patients successfully engrafting
time frame: 3, 6, 12 and 24 months post-transplant
Percentage of patients with durable engraftment
time frame: 3, 6, 12 and 24 months post-transplant
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)
Duration of hospitalizations (planned or unplanned)
time frame: Throughout the duration of the study
Mobilization of tumor cells into peripheral blood
time frame: Up to 5 days
Relapse rates
time frame: 3, 6, 12 and 24 months post-transplant
Occurrence of secondary malignancies
time frame: 3, 6, 12 and 24 months post-transplant
Incidence of primary and secondary graft failure
time frame: 3, 6, 12 and 24 months post-transplant
Time to secondary graft failure
time frame: Up to 24 months post-transplant
Survival rates
time frame: 3, 6, 12 and 24 months post-transplant

Eligibility Criteria

Male or female participants from 1 year up to 18 years old.

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

Additional Information

Official title A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Sanofi.