Treosulfan-based conditioning therapy in children with blood cancer
Research type
Research Study
Full title
Clinical phase II trial to describe the safety and efficacy of Treosulfanbased conditioning therapy prior to allogeneic haematopoietic stem cell transplantation in paediatric patients with haematological malignancies
IRAS ID
157769
Contact name
Ann-Kristin Moeller
Contact email
Sponsor organisation
medac Gesellschaft für klinische Spezialpräparate mbH
Eudract number
2013-003604-39
Research summary
Clinical studies are needed to gain or to expand knowledge about the efficacy and tolerability of drugs especially in children. By conducting this study with a larger number of patients it is hope to confirm that Treosulfan-based treatment is proved effective in children and adolescents, and that this conditioning may possibly prove to be somewhat better tolerated.
This study is to determine the safety and efficacy of Treosulfan-based conditioning therapy prior to allogeneic stem cell transplantation in paediatric patients
(infants/toddlers, children, adolescents) with blood cancer.The efficacy and the tolerance of the medication Treosulfan (Treograft®) is to be investigated in this clinical study. This medicine is used during the conditioning treatment to prepare the bone marrow for the stem cell transplantation, i.e. the transfer of healthy stem cells into the bone marrow.
Male and female children with blood cancer requiring conditioning treatment with following stem cell transplantation at time of registration from 28 days to less than 18 years of age would be eligible to take part in this clinical study.
This clinical study will be conducted at several places in United Kingdom and other countries. A total of at least 70 children and adolescents are planned to take part.
The study will last about 12 months. During this time patients will receive the medicine Treosulfan on three occasions (within the week of treatment, conditioning treatment together with other medicines will be included). Patients will then be observed for 12 months afterwards. A long-term follow-up observation is also planned for all patients (once a year). This will end 3 years after the last of at least 70 patients has been included in the study and transplanted.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
14/YH/1034
Date of REC Opinion
20 Aug 2014
REC opinion
Further Information Favourable Opinion