Haploidentical Stem Cell Transplantation for Children With Therapy Resistant Leukemia
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|ClinicalTrials.gov Identifier: NCT01025778|
Recruitment Status : Completed
First Posted : December 4, 2009
Last Update Posted : March 31, 2015
Despite substantial progress in the treatment pediatric acute leukemia a significant number of children will experience primary or secondary resistance to the treatment. In other words it will be not possible to achieve remission using standard chemotherapy (primary resistance) or the patients will develop chemotherapy resistant relapse (secondary resistance). Children failing to achieve remission or children relapsing after previous allogeneic stem cell transplantation have short life expectancy and palliative treatment still remains the most reasonable option as the escalation of conventional chemotherapy is not longer effective.
The role of Graft versus Leukemia effect was postulated as one of the mechanisms contributing to the leukemia control/eradication after transplantation of hematopoietic stem cells.
In this study the investigators combine intensified multiagent Clofarabine containing chemotherapy with post-induction treatment intensification using reduced intensity conditioning followed by haploidentical hematopoietic stem cell transplantation. Introducing a new drug to the treatment of resistant leukemia the investigators want to achieve a response which allows us to proceed to immediate haploidentical transplantation. Using a haploidentical donor the investigators can avoid time consuming search for an unrelated donor and perform the transplantation at the optimal time-point. Combating therapy resistant leukemia the investigators would like to evoke and utilize potential Graft-versus-Leukemia effect which is much more pronounced in the haploidentical setting, as it is well documented that allogeneic transplantation with a matched donor is not effective in resistant disease. The use of best KIR mismatch donor and post-transplant donor lymphocyte infusion will be implemented in order to develop/intensify graft versus leukemia effect.
|Condition or disease||Intervention/treatment||Phase|
|Acute Lymphoblastic Leukemia Acute Myeloid Leukemia||Drug: Clofarabine for remission induction Drug: Etoposide for remission induction Drug: Cyclophosphamide for remission induction Drug: Clofarabine in conditioning before transplantation Drug: Thiotepa in conditioning before transplantation Drug: Melfalan in conditioning before transplantation Procedure: Haploidentical transplantation of T-cell depleted graft Procedure: Donor lymphocyte infusion||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Clofarabine Based Remission Induction Followed by Haploidentical Stem Cell Transplantation in Children With Refractory Hematological Malignancies|
|Study Start Date :||December 2009|
|Actual Primary Completion Date :||December 2012|
|Actual Study Completion Date :||December 2012|
Experimental: Remission induction and haplo-SCT
Remission induction with Clofaranie, Etoposide and Cyclophosphamide combination followed by haplidentical stem cella transplantation if remission achieved.
Drug: Clofarabine for remission induction
Drug: Etoposide for remission induction
Drug: Cyclophosphamide for remission induction
Drug: Clofarabine in conditioning before transplantation
Drug: Thiotepa in conditioning before transplantation
Drug: Melfalan in conditioning before transplantation
Procedure: Haploidentical transplantation of T-cell depleted graft
Procedure: Donor lymphocyte infusion
- Event free survival [ Time Frame: 1 year from transplantation ]
- Evaluation of induction efficacy by response rate and the number of children proceeding to transplant [ Time Frame: 3 months from induction start ]
- Tolerance, safety and quality of life [ Time Frame: 1 year from transplantation ]
- Hematological and immunological recovery [ Time Frame: 100 days fron tranplantation ]
- Incidence of graft versus host disease [ Time Frame: 100 days from transplantation ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01025778
|Lund University Hospital|
|Lund, Sweden, SE-22185|
|Principal Investigator:||Jacek Toporski, MD, PhD||Lund University Hospital|