NCT01025778

Haploidentical Stem Cell Transplantation for Children With Therapy Resistant Leukemia

Clofarabine Based Remission Induction Followed by Haploidentical Stem Cell Transplantation in Children With Refractory Hematological Malignancies

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Lund University Hospital · Academic / Other
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia

Interventions

Type	Name	Description
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

Timeline

• Start date: 2009-12-01
• Primary completion: 2012-12-01
• Completion: 2012-12-01
• First posted: 2009-12-04
• Last updated: 2021-02-21

Locations

1 site across 1 country: Sweden