Phase II Trial of Individualized Pk-Targeted Preparative Approaches in TCRaB/CD19 Depleted HCT
Study of Personalized Conditioning Before Haploidentical Stem Cell Transplant in Children With High-Risk Blood Cancers
Jonathan Bardahl
Brief description of study
To evaluate the effectiveness of individually targeted, model-based dosing of serotherapy and chemotherapy conditioning on two-year post-HCT DFS in children with high-risk ALL, AML, MDS, or other hematological malignancies who are MRD-negative at the time of ex vivo TCRαβ+/CD19+-depleted haploidentical HCT
To evaluate the effectiveness of individually targeted, model-based dosing of serotherapy and chemotherapy conditioning on two-year post-HCT DFS in children with high-risk ALL, AML, MDS, or other hematological malignancies who are MRD-positive at the time of ex vivo TCRαβ+/CD19+-depleted haploidentical HCT.
To characterize overall survival (OS), graft-versus-host disease (GVHD)-free relapse-relapse free survival (GRFS), and chronic graft-versus-host disease-free relapse-free survival (CGRFS) at 2-year post-HCT for children following model-based dosing of serotherapy and chemotherapy conditioning followed by ex vivo TCRαβCD3+/CD19+ depleted HCT.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: ALL, AML, MDS, or other hematological malignancies, Cancer, Riley
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Age: 17 years or below
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Gender: All
The purpose of this research study is to learn about the effects of using individualized (personalized) dosing of conditioning treatment before a specific type of stem cell transplant in children with high-risk blood cancers. The study includes children with diseases such as acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or other blood cancers who are having a haploidentical stem cell transplant (a transplant from a half-matched family donor). The study includes children who are either MRD-negative or MRD-positive at the time of transplant (MRD means “minimal residual disease,” or very small amounts of cancer cells that can remain after treatment).
Participants will receive conditioning treatment that includes serotherapy and chemotherapy. In this study, the doses of these medicines are individually targeted using a model-based approach (a method that uses a dosing model to help tailor the dose to the individual). Participants will then receive a haploidentical stem cell transplant in which the donor cells are processed outside the body to remove certain immune cells (TCRαβ and CD19 cells) before the transplant.
Who can participate:
This study is enrolling by invitation only.
Children with high-risk ALL, AML, MDS, or other blood cancers may be able to participate if they:
- Are having a haploidentical stem cell transplant with donor cells that are processed to remove TCRαβ and CD19 cells
- Are MRD-negative or MRD-positive at the time of transplant
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