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

German Pediatric Oncology Group (GPOH) gGmbH0 个研究点目标入组 10 人开始时间: 待定最近更新: 2024年6月11日

试验速览

暂无简介。

•-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

•- 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
•- Symptomatic cardiac disorders (CTCAE 4.0 Grade 3 or 4).
•- Diagnosed Wilsons 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
•- 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.

发起方

German Pediatric Oncology Group (GPOH) gGmbH