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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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01288573
First Posted: February 2, 2011
Last Update Posted: May 16, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  Purpose

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.


Condition Intervention Phase
Ewing's Sarcoma/Soft Tissue Sarcoma Neuroblastoma Brain Tumors Drug: plerixafor Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.

Resource links provided by NLM:


Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome Measures:
  • Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2 [ Time Frame: Up to 5 days ]

Secondary Outcome Measures:
  • Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg [ Time Frame: Up to 5 days ]
    During Stage 1 and Stage 2

  • Yield of CD34+ cells for each apheresis [ Time Frame: Up to 5 days ]
    During Stage 1 and Stage 2

  • Total CD34+ cell yield [ Time Frame: Up to 5 days ]
    During Stage 1 and Stage 2

  • Percentage of patients proceeding to transplant [ Time Frame: Within 6 months of last apheresis ]
    During Stage 1 and Stage 2

  • Percentage of patients successfully engrafting [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2

  • Percentage of patients with durable engraftment [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2

  • 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) ]
    During Stage 1 and Stage 2

  • Duration of hospitalizations (planned or unplanned) [ Time Frame: Throughout the duration of the study ]
    During Stage 1 and Stage 2

  • Mobilization of tumor cells into peripheral blood [ Time Frame: Up to 5 days ]
    During Stage 1 and Stage 2

  • Relapse rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2

  • Occurrence of secondary malignancies [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2

  • Incidence of primary and secondary graft failure [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2

  • Time to secondary graft failure [ Time Frame: Up to 24 months post-transplant ]
    During Stage 1 and Stage 2

  • Survival rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2


Enrollment: 46
Study Start Date: March 3, 2014
Study Completion Date: May 9, 2017
Primary Completion Date: May 9, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Plerixafor 160 μg/kg
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).
Drug: plerixafor
160 μg/kg subcutaneous (SC) injection
Experimental: Plerixafor 240 μg/kg
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).
Drug: plerixafor
240 μg/kg subcutaneous (SC) injection
Experimental: Plerixafor 320 μg/kg
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).
Drug: plerixafor
320 μg/kg subcutaneous (SC) injection

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01288573


  Show 27 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Sanofi
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01288573     History of Changes
Other Study ID Numbers: DFI12860
2010-019340-40 ( EudraCT Number )
MOZ15609 ( Other Identifier: Genzyme other study code )
First Submitted: January 28, 2011
First Posted: February 2, 2011
Last Update Posted: May 16, 2017
Last Verified: May 2017

Additional relevant MeSH terms:
Sarcoma
Neuroblastoma
Sarcoma, Ewing
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Osteosarcoma
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
JM 3100
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents