Klinische Prüfung der Phase III mit CPX-351 zur Behandlung der Myeloischen Leukämie bei Kindern mit Down Syndrom 2018

Trade Name: Vyxeos liposomal 44mg/100mg Pulver f?r ein Konzentrat zur Herstellung einer infusionsl?sung
Product Name: Vyxeos
Product Code: CPX-351
Pharmaceutical Form: Powder for concentrate for solution for injection/infusion
INN or Proposed INN: Cytarabine
CAS Number: 147-94-4
Other descriptive name: CYTARABINE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
INN or Proposed INN: DAUNORUBICIN
CAS Number: 20830-81-3
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 44-

Gender:
Female: yes
Male: yes

Inclusion criteria:
?Myeloid Leukemia (ML) or Myelodysplastic Syndrome (MDS), according to WHO
?Trisomy 21: Down syndrome or mosaic
?Age: > 6 months and = 4 years of age with/without GATA1 mutation OR > 4 years of age < 6 years of age with GATA1 mutation
?Morphology/Immunophenotyping: FAB M0, M6 or M7
?Lansky performance score at least equal to 50; or Karnofsky performance status at least equal to 50, whichever is applicable
?Understand and voluntarily provide written permission of parental/legal representative(s) to the ICF prior to conducting any study related assessments/procedures, also concerning data and tumor material transfer according to ICH/GCP and national/local regulations
?Able to adhere to the study visit schedule and other protocol requirements
? Acceptance that vaccination with live vaccines is not possible while participating in the trial
Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
?Children with Transient Abnormal Myelopoiesis (TAM), according to WHO

?Cytogenetics: AML with recurrent genetic abnormalities (WHO 2016)

?Previous allogeneic bone marrow, stem cell or organ transplantation

?Evidence of invasive fungal infection or other severe systemic infection requiring treatment doses of systemic/parenteral therapy including known active viral infection with human immunodeficiency virus (HIV) or Hepatitis Type B and C

?Symptomatic cardiac disorders (CTCAE 4.0 Grade 3 or 4)

? Diagnosed Wilson?s Disease

?Major surgery within 21 days of the first dose.

?Any anti-cancer therapy (e.g., intensive chemotherapy, biologics or radiotherapy) for more than 14 days or within 4 weeks before start of therapy, except low-dose cytarabine for the treatment of TAM.

?Concomitant treatment with any other anticancer therapy except those specified in protocol during the study therapy

?Treated by any investigational agent in a clinical study within previous 4 weeks

?History of hypersensitivity to the investigational medicinal product or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of the investigational medicinal product

?Former Enrolment to this study

?The patient concerned has been committed to an institution by virtue of an order issued either by the judicial or the administrative authorities