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 maj 2017 uppdaterad av: 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.
Studieöversikt
Status
Avslutad
Intervention / Behandling
Studietyp
Interventionell
Inskrivning (Faktisk)
46
Fas
- Fas 2
- Fas 1
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studieorter
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Gent, Belgien, 9000
- Investigational Site Number 51
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København Ø, Danmark, 2100
- Investigational Site Number 61
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Lyon, Frankrike, 69373
- Investigational Site Number 42
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Paris Cedex 05, Frankrike, 75248
- Investigational Site Number 43
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Petach Tikva, Israel, 4920235
- Investigational Site Number 92
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Tel-Aviv, Israel, 64239
- Investigational Site Number 91
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Genova, Italien, 16100
- Investigational Site Number 21
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Milano, Italien, 20133
- Investigational Site Number 24
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Padova, Italien, 35128
- Investigational Site Number 23
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Roma, Italien, 00165
- Investigational Site Number 22
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Torino, Italien, 10126
- Investigational Site Number 26
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Amsterdam, Nederländerna, 1105 AZ
- Investigational Site Number 72
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Rotterdam, Nederländerna, 3015 GJ
- Investigational Site Number 71
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Krakow, Polen, 30-663
- Investigational Site Number 85
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Wroclaw, Polen, 50-368
- Investigational Site Number 84
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Barcelona, Spanien, 08035
- Investigational Site Number 94
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Madrid, Spanien, 28009
- Investigational Site Number 93
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Birmingham, Storbritannien, B4 6NH
- Investigational Site Number 11
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Glasgow, Storbritannien, G51 4TF
- Investigational Site Number 13
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Brno, Tjeckien, 62500
- Investigational Site Number 81
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Praha 5 - Motol, Tjeckien, 15006
- Investigational Site Number 82
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Frankfurt Am Main, Tyskland, 60590
- Investigational Site Number 33
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Freiburg, Tyskland, 79106
- Investigational Site Number 34
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Hamburg, Tyskland, 20246
- Investigational Site Number 35
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Hannover, Tyskland, 30625
- Investigational Site Number 31
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München, Tyskland, 80337
- Investigational Site Number 36
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Budapest, Ungern, 1097
- Investigational Site Number 83
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Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
1 år till 18 år (Barn, Vuxen)
Tar emot friska volontärer
Nej
Kön som är behöriga för studier
Allt
Beskrivning
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
Studieplan
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Randomiserad
- Interventionsmodell: Parallellt uppdrag
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
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Experimentell: 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).
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160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 μg/kg subcutaneous (SC) injection
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Experimentell: 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).
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160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 μg/kg subcutaneous (SC) injection
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Experimentell: 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).
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160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 μg/kg subcutaneous (SC) injection
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
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Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Tidsram: Up to 5 days
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Up to 5 days
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Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
Tidsram: Up to 5 days
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During Stage 1 and Stage 2
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Up to 5 days
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Yield of CD34+ cells for each apheresis
Tidsram: Up to 5 days
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During Stage 1 and Stage 2
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Up to 5 days
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Total CD34+ cell yield
Tidsram: Up to 5 days
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During Stage 1 and Stage 2
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Up to 5 days
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Percentage of patients proceeding to transplant
Tidsram: Within 6 months of last apheresis
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During Stage 1 and Stage 2
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Within 6 months of last apheresis
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Percentage of patients successfully engrafting
Tidsram: 3, 6, 12 and 24 months post-transplant
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During Stage 1 and Stage 2
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3, 6, 12 and 24 months post-transplant
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Percentage of patients with durable engraftment
Tidsram: 3, 6, 12 and 24 months post-transplant
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During Stage 1 and Stage 2
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3, 6, 12 and 24 months post-transplant
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Summary of adverse events (AEs)
Tidsram: 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)
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During Stage 1 and Stage 2
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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)
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Duration of hospitalizations (planned or unplanned)
Tidsram: Throughout the duration of the study
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During Stage 1 and Stage 2
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Throughout the duration of the study
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Mobilization of tumor cells into peripheral blood
Tidsram: Up to 5 days
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During Stage 1 and Stage 2
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Up to 5 days
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Relapse rates
Tidsram: 3, 6, 12 and 24 months post-transplant
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During Stage 1 and Stage 2
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3, 6, 12 and 24 months post-transplant
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Occurrence of secondary malignancies
Tidsram: 3, 6, 12 and 24 months post-transplant
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During Stage 1 and Stage 2
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3, 6, 12 and 24 months post-transplant
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Incidence of primary and secondary graft failure
Tidsram: 3, 6, 12 and 24 months post-transplant
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During Stage 1 and Stage 2
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3, 6, 12 and 24 months post-transplant
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Time to secondary graft failure
Tidsram: Up to 24 months post-transplant
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During Stage 1 and Stage 2
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Up to 24 months post-transplant
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Survival rates
Tidsram: 3, 6, 12 and 24 months post-transplant
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During Stage 1 and Stage 2
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3, 6, 12 and 24 months post-transplant
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Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Sponsor
Samarbetspartners
Publikationer och användbara länkar
Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.
Allmänna publikationer
- 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.
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart
3 mars 2014
Primärt slutförande (Faktisk)
9 maj 2017
Avslutad studie (Faktisk)
9 maj 2017
Studieregistreringsdatum
Först inskickad
28 januari 2011
Först inskickad som uppfyllde QC-kriterierna
1 februari 2011
Första postat (Uppskatta)
2 februari 2011
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
16 maj 2017
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
15 maj 2017
Senast verifierad
1 maj 2017
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
- Neoplasmer, bindväv och mjukvävnad
- Neoplasmer efter histologisk typ
- Neoplasmer
- Neoplasmer, körtel och epitel
- Neoplasmer, neuroepitelial
- Neuroektodermala tumörer
- Neoplasmer, könsceller och embryonala
- Neoplasmer, nervvävnad
- Osteosarkom
- Neoplasmer, benvävnad
- Neoplasmer, bindväv
- Neuroektodermala tumörer, primitiva
- Neuroektodermala tumörer, primitiva, perifera
- Sarkom
- Sarkom, Ewing
- Neuroblastom
- Anti-infektionsmedel
- Antivirala medel
- Anti-HIV-medel
- Antiretrovirala medel
- Plerixafor
Andra studie-ID-nummer
- DFI12860
- 2010-019340-40 (EudraCT-nummer)
- MOZ15609 (Annan identifierare: Genzyme other study code)
Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .