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 de mayo de 2017 actualizado por: 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.
Descripción general del estudio
Estado
Terminado
Intervención / Tratamiento
Tipo de estudio
Intervencionista
Inscripción (Actual)
46
Fase
- Fase 2
- Fase 1
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
-
-
-
Frankfurt Am Main, Alemania, 60590
- Investigational Site Number 33
-
Freiburg, Alemania, 79106
- Investigational Site Number 34
-
Hamburg, Alemania, 20246
- Investigational Site Number 35
-
Hannover, Alemania, 30625
- Investigational Site Number 31
-
München, Alemania, 80337
- Investigational Site Number 36
-
-
-
-
-
Gent, Bélgica, 9000
- Investigational Site Number 51
-
-
-
-
-
Brno, Chequia, 62500
- Investigational Site Number 81
-
Praha 5 - Motol, Chequia, 15006
- Investigational Site Number 82
-
-
-
-
-
København Ø, Dinamarca, 2100
- Investigational Site Number 61
-
-
-
-
-
Barcelona, España, 08035
- Investigational Site Number 94
-
Madrid, España, 28009
- Investigational Site Number 93
-
-
-
-
-
Lyon, Francia, 69373
- Investigational Site Number 42
-
Paris Cedex 05, Francia, 75248
- Investigational Site Number 43
-
-
-
-
-
Budapest, Hungría, 1097
- Investigational Site Number 83
-
-
-
-
-
Petach Tikva, Israel, 4920235
- Investigational Site Number 92
-
Tel-Aviv, Israel, 64239
- Investigational Site Number 91
-
-
-
-
-
Genova, Italia, 16100
- Investigational Site Number 21
-
Milano, Italia, 20133
- Investigational Site Number 24
-
Padova, Italia, 35128
- Investigational Site Number 23
-
Roma, Italia, 00165
- Investigational Site Number 22
-
Torino, Italia, 10126
- Investigational Site Number 26
-
-
-
-
-
Amsterdam, Países Bajos, 1105 AZ
- Investigational Site Number 72
-
Rotterdam, Países Bajos, 3015 GJ
- Investigational Site Number 71
-
-
-
-
-
Krakow, Polonia, 30-663
- Investigational Site Number 85
-
Wroclaw, Polonia, 50-368
- Investigational Site Number 84
-
-
-
-
-
Birmingham, Reino Unido, B4 6NH
- Investigational Site Number 11
-
Glasgow, Reino Unido, G51 4TF
- Investigational Site Number 13
-
-
Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
1 año a 18 años (Niño, Adulto)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Todos
Descripción
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 de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
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).
|
160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 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).
|
160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 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).
|
160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 μg/kg subcutaneous (SC) injection
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Periodo de tiempo |
|---|---|
|
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Periodo de tiempo: Up to 5 days
|
Up to 5 days
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
Periodo de tiempo: Up to 5 days
|
During Stage 1 and Stage 2
|
Up to 5 days
|
|
Yield of CD34+ cells for each apheresis
Periodo de tiempo: Up to 5 days
|
During Stage 1 and Stage 2
|
Up to 5 days
|
|
Total CD34+ cell yield
Periodo de tiempo: Up to 5 days
|
During Stage 1 and Stage 2
|
Up to 5 days
|
|
Percentage of patients proceeding to transplant
Periodo de tiempo: Within 6 months of last apheresis
|
During Stage 1 and Stage 2
|
Within 6 months of last apheresis
|
|
Percentage of patients successfully engrafting
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: Up to 5 days
|
During Stage 1 and Stage 2
|
Up to 5 days
|
|
Relapse rates
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: Up to 24 months post-transplant
|
During Stage 1 and Stage 2
|
Up to 24 months post-transplant
|
|
Survival rates
Periodo de tiempo: 3, 6, 12 and 24 months post-transplant
|
During Stage 1 and Stage 2
|
3, 6, 12 and 24 months post-transplant
|
Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Colaboradores
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Publicaciones Generales
- Sebastien B, Cheverton P, Magnin C, Aouni J, Castan R. Development and validation of a predictive model to guide the use of plerixafor in pediatric population. Bone Marrow Transplant. 2022 Dec;57(12):1827-1832. doi: 10.1038/s41409-022-01831-2. Epub 2022 Sep 26.
- Morland B, Kepak T, Dallorso S, Sevilla J, Murphy D, Luksch R, Yaniv I, Bader P, Rossler J, Bisogno G, Maecker-Kolhoff B, Lang P, Zwaan CM, Sumerauer D, Krivan G, Bernard J, Liu Q, Doyle E, Locatelli F. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC). Bone Marrow Transplant. 2020 Sep;55(9):1744-1753. doi: 10.1038/s41409-020-0836-2. Epub 2020 Mar 3.
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio
3 de marzo de 2014
Finalización primaria (Actual)
9 de mayo de 2017
Finalización del estudio (Actual)
9 de mayo de 2017
Fechas de registro del estudio
Enviado por primera vez
28 de enero de 2011
Primero enviado que cumplió con los criterios de control de calidad
1 de febrero de 2011
Publicado por primera vez (Estimar)
2 de febrero de 2011
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
16 de mayo de 2017
Última actualización enviada que cumplió con los criterios de control de calidad
15 de mayo de 2017
Última verificación
1 de mayo de 2017
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Neoplasias De Tejidos Conectivos Y Blandos
- Neoplasias por tipo histológico
- Neoplasias
- Neoplasias Glandulares y Epiteliales
- Neoplasias Neuroepiteliales
- Tumores neuroectodérmicos
- Neoplasias De Células Germinales Y Embrionarias
- Neoplasias De Tejido Nervioso
- Osteosarcoma
- Neoplasias De Tejido Óseo
- Neoplasias Del Tejido Conectivo
- Tumores Neuroectodérmicos Primitivos
- Tumores Neuroectodérmicos Primitivos Periféricos
- Sarcoma
- Sarcoma de Ewing
- Neuroblastoma
- Agentes antiinfecciosos
- Agentes Antivirales
- Agentes Anti-VIH
- Agentes antirretrovirales
- Plerixafor
Otros números de identificación del estudio
- DFI12860
- 2010-019340-40 (Número EudraCT)
- MOZ15609 (Otro identificador: Genzyme other study code)
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .