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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

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ClinicalTrials.gov Identifier: NCT01288573
Recruitment Status : Completed
First Posted : February 2, 2011
Last Update Posted : May 16, 2017
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief 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.


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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 46 participants
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.
Study Start Date : March 3, 2014
Actual Primary Completion Date : May 9, 2017
Actual Study Completion Date : May 9, 2017


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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 :
  1. 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

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

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

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

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

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

  7. 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

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

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

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

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

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

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

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



Information from the National Library of Medicine

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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

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


  Hide Study Locations
Locations
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Belgium
Investigational Site Number 51
Gent, Belgium, 9000
Czechia
Investigational Site Number 81
Brno, Czechia, 62500
Investigational Site Number 82
Praha 5 - Motol, Czechia, 15006
Denmark
Investigational Site Number 61
København Ø, Denmark, 2100
France
Investigational Site Number 42
Lyon, France, 69373
Investigational Site Number 43
Paris Cedex 05, France, 75248
Germany
Investigational Site Number 33
Frankfurt Am Main, Germany, 60590
Investigational Site Number 34
Freiburg, Germany, 79106
Investigational Site Number 35
Hamburg, Germany, 20246
Investigational Site Number 31
Hannover, Germany, 30625
Investigational Site Number 36
München, Germany, 80337
Hungary
Investigational Site Number 83
Budapest, Hungary, 1097
Israel
Investigational Site Number 92
Petach Tikva, Israel, 4920235
Investigational Site Number 91
Tel-Aviv, Israel, 64239
Italy
Investigational Site Number 21
Genova, Italy, 16100
Investigational Site Number 24
Milano, Italy, 20133
Investigational Site Number 23
Padova, Italy, 35128
Investigational Site Number 22
Roma, Italy, 00165
Investigational Site Number 26
Torino, Italy, 10126
Netherlands
Investigational Site Number 72
Amsterdam, Netherlands, 1105 AZ
Investigational Site Number 71
Rotterdam, Netherlands, 3015 GJ
Poland
Investigational Site Number 85
Krakow, Poland, 30-663
Investigational Site Number 84
Wroclaw, Poland, 50-368
Spain
Investigational Site Number 94
Barcelona, Spain, 08035
Investigational Site Number 93
Madrid, Spain, 28009
United Kingdom
Investigational Site Number 11
Birmingham, United Kingdom, B4 6NH
Investigational Site Number 13
Glasgow, United Kingdom, G51 4TF
Sponsors and Collaborators
Genzyme, a Sanofi Company
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi

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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 Posted: February 2, 2011    Key Record Dates
Last Update Posted: May 16, 2017
Last Verified: May 2017
Additional relevant MeSH terms:
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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
Plerixafor octahydrochloride
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents