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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 mai 2017 mis à jour par: 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.

Aperçu de l'étude

Statut

Complété

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Réel)

46

Phase

  • Phase 2
  • La phase 1

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

1 an à 18 ans (Enfant, Adulte)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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).
Médicament: plerixafor
160 μg/kg subcutaneous (SC) injection
Médicament: plerixafor
240 μg/kg subcutaneous (SC) injection
Médicament: plerixafor
320 μg/kg subcutaneous (SC) injection
Expérimental: 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).
Médicament: plerixafor
160 μg/kg subcutaneous (SC) injection
Médicament: plerixafor
240 μg/kg subcutaneous (SC) injection
Médicament: plerixafor
320 μg/kg subcutaneous (SC) injection
Expérimental: 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).
Médicament: plerixafor
160 μg/kg subcutaneous (SC) injection
Médicament: plerixafor
240 μg/kg subcutaneous (SC) injection
Médicament: plerixafor
320 μg/kg subcutaneous (SC) injection

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Délai
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Délai: Up to 5 days
Up to 5 days

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
Délai: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Yield of CD34+ cells for each apheresis
Délai: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Total CD34+ cell yield
Délai: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Percentage of patients proceeding to transplant
Délai: Within 6 months of last apheresis
During Stage 1 and Stage 2
Within 6 months of last apheresis
Percentage of patients successfully engrafting
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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
Délai: Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Relapse rates
Délai: 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
Délai: 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
Délai: 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
Délai: Up to 24 months post-transplant
During Stage 1 and Stage 2
Up to 24 months post-transplant
Survival rates
Délai: 3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Genzyme, a Sanofi Company

Collaborateurs

Sanofi

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude

3 mars 2014

Achèvement primaire (Réel)

9 mai 2017

Achèvement de l'étude (Réel)

9 mai 2017

Dates d'inscription aux études

Première soumission

28 janvier 2011

Première soumission répondant aux critères de contrôle qualité

1 février 2011

Première publication (Estimation)

2 février 2011

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

16 mai 2017

Dernière mise à jour soumise répondant aux critères de contrôle qualité

15 mai 2017

Dernière vérification

1 mai 2017

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • DFI12860
  • 2010-019340-40 (Numéro EudraCT)
  • MOZ15609 (Autre identifiant: Genzyme other study code)

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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