Myelodysplastic Syndromes (MDS) Clinical Trial
Official title:
Clinical Phase II Trial to Describe the Safety and Efficacy of Treosulfan-based Conditioning Therapy Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Paediatric Patients With Haematological Malignancies
The primary goal of this study is to evaluate an alternative myeloablative, but reduced toxicity conditioning regimen in children, to describe the safety and efficacy of intravenous (i.v.) Treosulfan administered as part of a standardised Fludarabine-containing conditioning and to contribute to the current pharmacokinetic model to be able to finally give age (or body surface area) dependent dose recommendations. The treatment regimens given in the protocol MC-FludT.17/M are based on sufficient clinical safety and efficacy data. Considering the vital indication for allogeneic haematopoietic stem cell transplantation of the selected patient population, the risk-benefit assessment is therefore reasonably in favour of the study conduct.
The protocol MC-FludT.17/M is a clinical phase II trial to describe the safety and efficacy
of Treosulfan-based conditioning therapy prior to allogeneic haematopoietic stem cell
transplantation (allo-HSCT) in at least 70 paediatric patients with haematological
malignancies (male and female children with haematological malignant diseases as acute
lymphoblastic leukaemias (ALL), acute myeloid leukaemias (AML), myelodysplastic syndromes
(MDS) and juvenile myelomonocytic leukaemias (JMML), requiring myeloablative conditioning
treatment with following allo-HSCT).
Treosulfan dose per day is to be calculated by using body surface area (BSA). Two background
conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised
Fludarabine-containing regimen and the other consists of an intensified regimen with
Fludarabine and ThioTEPA.
Freedom from transplant (treatment)-related mortality (TRM), defined as death from any
transplant-related cause from the day of first administration of study medication until day
+100 after HSCT is the primary objective of the trial.
Moreover, the current pharmacokinetic (PK) model should be contributed to be able to finally
give age (or BSA) dependent dose recommendations.
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