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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

15 mei 2017 bijgewerkt door: Genzyme, a Sanofi Company

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.

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.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

46

Fase

  • Fase 2
  • Fase 1

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • België
      • Gent, België, 9000
        • Investigational Site Number 51
  • Denemarken
      • København Ø, Denemarken, 2100
        • Investigational Site Number 61
  • Duitsland
      • Frankfurt Am Main, Duitsland, 60590
        • Investigational Site Number 33
      • Freiburg, Duitsland, 79106
        • Investigational Site Number 34
      • Hamburg, Duitsland, 20246
        • Investigational Site Number 35
      • Hannover, Duitsland, 30625
        • Investigational Site Number 31
      • München, Duitsland, 80337
        • Investigational Site Number 36
  • Frankrijk
      • Lyon, Frankrijk, 69373
        • Investigational Site Number 42
      • Paris Cedex 05, Frankrijk, 75248
        • Investigational Site Number 43
  • Hongarije
      • Budapest, Hongarije, 1097
        • Investigational Site Number 83
  • Israël
      • Petach Tikva, Israël, 4920235
        • Investigational Site Number 92
      • Tel-Aviv, Israël, 64239
        • Investigational Site Number 91
  • Italië
      • Genova, Italië, 16100
        • Investigational Site Number 21
      • Milano, Italië, 20133
        • Investigational Site Number 24
      • Padova, Italië, 35128
        • Investigational Site Number 23
      • Roma, Italië, 00165
        • Investigational Site Number 22
      • Torino, Italië, 10126
        • Investigational Site Number 26
  • Nederland
      • Amsterdam, Nederland, 1105 AZ
        • Investigational Site Number 72
      • Rotterdam, Nederland, 3015 GJ
        • Investigational Site Number 71
  • Polen
      • Krakow, Polen, 30-663
        • Investigational Site Number 85
      • Wroclaw, Polen, 50-368
        • Investigational Site Number 84
  • Spanje
      • Barcelona, Spanje, 08035
        • Investigational Site Number 94
      • Madrid, Spanje, 28009
        • Investigational Site Number 93
  • Tsjechië
      • Brno, Tsjechië, 62500
        • Investigational Site Number 81
      • Praha 5 - Motol, Tsjechië, 15006
        • Investigational Site Number 82
  • Verenigd Koninkrijk
      • Birmingham, Verenigd Koninkrijk, B4 6NH
        • Investigational Site Number 11
      • Glasgow, Verenigd Koninkrijk, G51 4TF
        • Investigational Site Number 13

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

1 jaar tot 18 jaar (Kind, Volwassen)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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).
Geneesmiddel: plerixafor
160 μg/kg subcutaneous (SC) injection
Geneesmiddel: plerixafor
240 μg/kg subcutaneous (SC) injection
Geneesmiddel: plerixafor
320 μg/kg subcutaneous (SC) injection
Experimenteel: 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).
Geneesmiddel: plerixafor
160 μg/kg subcutaneous (SC) injection
Geneesmiddel: plerixafor
240 μg/kg subcutaneous (SC) injection
Geneesmiddel: plerixafor
320 μg/kg subcutaneous (SC) injection
Experimenteel: 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).
Geneesmiddel: plerixafor
160 μg/kg subcutaneous (SC) injection
Geneesmiddel: plerixafor
240 μg/kg subcutaneous (SC) injection
Geneesmiddel: plerixafor
320 μg/kg subcutaneous (SC) injection

Wat meet het onderzoek?

Primaire uitkomstmaten

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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
Tijdsspanne: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Yield of CD34+ cells for each apheresis
Tijdsspanne: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Total CD34+ cell yield
Tijdsspanne: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Percentage of patients proceeding to transplant
Tijdsspanne: Within 6 months of last apheresis
During Stage 1 and Stage 2
Within 6 months of last apheresis
Percentage of patients successfully engrafting
Tijdsspanne: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Percentage of patients with durable engraftment
Tijdsspanne: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Summary of adverse events (AEs)
Tijdsspanne: 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
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)
Tijdsspanne: Throughout the duration of the study
During Stage 1 and Stage 2
Throughout the duration of the study
Mobilization of tumor cells into peripheral blood
Tijdsspanne: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Relapse rates
Tijdsspanne: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Occurrence of secondary malignancies
Tijdsspanne: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Incidence of primary and secondary graft failure
Tijdsspanne: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Time to secondary graft failure
Tijdsspanne: Up to 24 months post-transplant
During Stage 1 and Stage 2
Up to 24 months post-transplant
Survival rates
Tijdsspanne: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Genzyme, a Sanofi Company

Medewerkers

Sanofi

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start

3 maart 2014

Primaire voltooiing (Werkelijk)

9 mei 2017

Studie voltooiing (Werkelijk)

9 mei 2017

Studieregistratiedata

Eerst ingediend

28 januari 2011

Eerst ingediend dat voldeed aan de QC-criteria

1 februari 2011

Eerst geplaatst (Schatting)

2 februari 2011

Updates van studierecords

Laatste update geplaatst (Werkelijk)

16 mei 2017

Laatste update ingediend die voldeed aan QC-criteria

15 mei 2017

Laatst geverifieerd

1 mei 2017

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • DFI12860
  • 2010-019340-40 (EudraCT-nummer)
  • MOZ15609 (Andere identificatie: Genzyme other study code)

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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