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 is currently recruiting participants.
Verified March 2014 by Sanofi
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01288573
First received: January 28, 2011
Last updated: March 31, 2014
Last verified: March 2014
  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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg [ Time Frame: Up to 5 days ] [ Designated as safety issue: No ]
    During Stage 1 and Stage 2

  • Yield of CD34+ cells for each apheresis [ Time Frame: Up to 5 days ] [ Designated as safety issue: No ]
    During Stage 1 and Stage 2

  • Total CD34+ cell yield [ Time Frame: Up to 5 days ] [ Designated as safety issue: No ]
    During Stage 1 and Stage 2

  • Percentage of patients proceeding to transplant [ Time Frame: Within 6 months of last apheresis ] [ Designated as safety issue: No ]
    During Stage 1 and Stage 2

  • Percentage of patients successfully engrafting [ Time Frame: 3, 6, 12 and 24 months post-transplant ] [ Designated as safety issue: No ]
    During Stage 1 and Stage 2

  • Percentage of patients with durable engraftment [ Time Frame: 3, 6, 12 and 24 months post-transplant ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: Yes ]
    During Stage 1 and Stage 2

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

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

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

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

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

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

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


Estimated Enrollment: 46
Study Start Date: February 2014
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: June 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

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01288573

Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com

Locations
Czech Republic
Investigational Site Number 81 Recruiting
Brno, Czech Republic, 62500
Denmark
Investigational Site Number 62 Recruiting
Aarhus, Denmark, 8200
Investigational Site Number 61 Recruiting
København Ø, Denmark, 2100
Spain
Investigational Site Number 94 Recruiting
Barcelona, Spain, 08035
Investigational Site Number 95 Recruiting
Esplugues De Llobregat, Spain, 08950
Investigational Site Number 93 Recruiting
Madrid, Spain, 28009
United Kingdom
Investigational Site Number 11 Recruiting
Birmingham, United Kingdom, B4 6NH
Investigational Site Number 13 Recruiting
Glasgow, United Kingdom, G3 8SJ
Sponsors and Collaborators
Genzyme, a Sanofi Company
Sanofi
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01288573     History of Changes
Other Study ID Numbers: DFI12860, 2010-019340-40, MOZ15609
Study First Received: January 28, 2011
Last Updated: March 31, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Italy: ISS (Instituto Superiore de Sanità)
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Brain Neoplasms
Neuroblastoma
Sarcoma, Ewing's
Sarcoma
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Osteosarcoma
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
JM 3100
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 16, 2014