NCT04000698

Personalized Targeted Preparative Regimen Before T-depleted Allogeneic HSCT in Children With Chemoresistent Acute Leukemias

A Phase I-II Pilot Clinical Trial of Safety and Efficacy of Personalized Targeted Preparative Regimen With Allogeneic TcRαβ/CD19-depleted Hematopoietic Stem Cell Transplantation and Posttransplant Donor T- Cells Infusion in Children With Chemoresistаnt Acute Leukemia.

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 25 (estimated)
• Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology · Academic / Other
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and efficiency of personalized targeted therapy in combination with high-dose chemotherapy as part of a preparative regimen before T-depleted allogeneic hematopoietic stem cell transplantation in children with chemoresistant acute leukemias

Detailed description

The outcome of hematopoietic stem cell transplantation (HSCT) in a cohort of children with chemorefractory leukemia is poor. The incidence of relapse exceeds 50% and survival varies from 10 to 40%. Additional attempts at remission induction with various combinations of chemotherapy are unlikely to improve the outcome and will contribute to toxicity. The hypothesis of the study is that personalized targeted therapy combined with high-dose chemotherapy may improve the outcome of allogeneic HSCT in a cohort of pediatric patients with refractory leukemia. Bcl-2, CD38, CD184 were chosen as potential targets due to frequent expression in pediatric acute leukemias, availability of marketed targeted therapies venetoclax, daratumumab and prelixafor, and expected non-overlapping toxicity profile of these agents and the conditioning regimen.

Conditions

• Refractory Acute Myeloid Leukemia
• Refractory Acute Lymphoblastic Leukemia

Interventions

Type	Name	Description
DRUG	Preparative regimen	Preparative chemotherapy before allogeneic HSCT * Fludarabin * Cytarabine * Venetoclax * Daratumomab * Vecanoid Condition * treosulfan * fludarabine * thiophosphomide * Venetoclax * Plerixafor GVHD prophylaxis * abatacept * tocilizumab * rituximab * HSCT from the haploidentical donor, ex vivo depleted of alpha/beta T lymphocytes

Timeline

• Start date: 2019-10-15
• Primary completion: 2022-07-01
• Completion: 2022-12-01
• First posted: 2019-06-27
• Last updated: 2020-11-20

Locations

1 site across 1 country: Russia