Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018

Who can take part

• 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
• Myeloid Leukemia (ML) or Myelodysplastic Syndrome (MDS), according to WHO

Who cannot take part

• Treated by any investigational agent in a clinical study within previous 4 weeks
• Previous allogeneic bone marrow, stem cell or organ transplantation
• 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
• 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
• 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
• Children with Transient Abnormal Myelopoiesis (TAM), according to WHO
• 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
• Cytogenetics: AML with recurrent genetic abnormalities (WHO 2016)