Acute Myeloid Leukemia (AML)
Conditions
Keywords
relapsed or refractory AML, in children, adolescents and young adults
Brief summary
1. To evaluate whether stem cell transplantation from a matched sibling donor is equivalent to a matched unrelated donor in in a second complete remission of acute myeloid leukemia (AML). 2. To evaluate whether stem cell transplantation (SCT) after chemotherapy (FLAMSA-schema) increases survival compared to a threshold derived from historical data 3. To evaluate whether SCT from haploidentical donors for children having no matched donor will result in better survival with acceptable toxicity.
Detailed description
Target variables: * Treatment response * Event Free Survival * Leukemia Free Survival * Graft Versus Host Disease * Regimen related toxicity
Interventions
BIOLOGICALHematopoietic stem cells from bone marrow or peripheral blood
\> = 2 x 10\*8 nucleated cells (WBC)/kg body weight of the recipient or rather \> = 4 x 10\*6 nucleated CD34+ cells / kg body weight are required for engraftment. Suspension of stem cells is administered via intravenous infusion.
Sponsors
Hannover Medical School
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 21 Years
Healthy volunteers
No
Inclusion criteria
* Aged between 0-21 years * Patients suffering from either refractory de novo AML or relapsed AML or patients with very high risk AML in CR1 * In sexually active patients two reliable contraception methods are used. This includes every combination of a hormonal contraceptive (such as injection, transdermal patch, implant, cervical ring) or of an intrauterine device(IUD) with a barrier method (e.g. diaphragm, cervical cap, or condom) or with a spermicide. * Written informed consent of patient, parents or legal guardians
Exclusion criteria
* Severe renal impairment (GFR \< 30% predicted for age) * Pregnant or lactating females * Current participation in another clinical trial * Patients ≥ 12 years old for Group 1 (BuCyMel) (patients younger that 12 years continue to be included)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate whether stem cell transplantation (SCT) from a matched sibling donor (MSD) is equivalent to a matched unrelated donor (MUD) in second complete remission (CR2). | day 100 |
| To evaluate whether FLAMSA increases leukemia free survival (LFS) and overall survival (OS) as compared to a historic control group | day 100 |
| To evaluate whether SCT from haploidentical donors for children having no matched donor will result in an acceptable toxicity profile and a better LFS as compared to historic controls. | day 100 |
Secondary
| Measure | Time frame |
|---|---|
| To further evaluate the contribution of immunomediated effects for the treatment of children suffering from very high risk AML | day 100 |
| Prospective evaluation of event free survival (EFS), LFS, and OS after SCT from either a MSD or a MUD | day 100 |
| Prospective evaluation of late toxicities | day 100 and year 5 |
| To evaluate whether it is feasible to standardize transplantation procedures in children with AML within the AML-Berlin/Frankfurt/Münster (BFM) study network | day 100 |
| Decrease of transplantation associated mortality by standardized donor selection criteria | day 100 |
Countries
Austria, Czechia, Germany
Outcome results
None listed