AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Launched by MITCHELL CAIRO · Sep 20, 2019

Keywords

ClinConnect Summary

This clinical trial is studying a specific type of stem cell transplant called allogeneic stem cell transplantation (AlloSCT) for children, adolescents, and young adults with certain blood-related diseases, including types of leukemia, lymphoma, and other blood disorders like sickle cell disease and Diamond-Blackfan anemia. The researchers are investigating a method that uses special techniques to remove certain types of cells from the donor’s stem cells to improve the transplant's effectiveness. All other treatments provided during the study will follow standard medical practices.

To be eligible for this trial, participants should be between the ages of 1 and 30 and have high-risk forms of the mentioned conditions, or certain types of bone marrow failure. For example, they may need to have had treatment failures or specific genetic abnormalities. Participants can expect to receive a stem cell transplant while being closely monitored by medical professionals. It’s important to note that pregnant or breastfeeding women, those with uncontrolled infections, and patients who have recently had a stem cell transplant are not eligible for this study. This trial aims to provide new insights and potentially better outcomes for those struggling with these challenging health conditions.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. ALL:ALL high risk including one or more of the following: (t(9;22) or 11q23 chromosomal abnormality, primary induction failure (\<15% blasts at time of registration), mixed phenotype acute leukemia (MPAL), persistent MRD (\<0.01% by flow or persistent abnormal karyotype detected by cytogenetics) or hypodiploidy (44 chromosomes)) in first remission ' ALL in second remission and beyond;
• • 2. AML: History of AML induction/reinduction Failure (\<15% blasts at time of registration); AML in CR1 with poor cytogenetics (i.e. 12p, 5a, -7, FLT3 mutation/duplication, t(9;11) and others); AML with persistent minimal residual disease (MRD) in CR1(\<0.01% on flow or persistent abnormal karyotype detected by cytogenetics); AML CR2 or beyond; AML in refractory relapse but ≤15% bone marrow leukemia blasts; Therapy-related AML
• • 3. High Risk Myelodysplastic syndrome (MDS) 4 Lymphoma: Hodgkin (HL) or Non-Hodgkin (NHL): HL or NHL in induction failure; HL or NHL in PR1 or PR2 ; HL or NHL in CR2 or subsequent remission
• • 5. Bone marrow failure syndromes: Kostmann syndrome refractory or intolerant to granulocyte colony-33stimulating factor; Diamond-Blackfan anemia refractory or intolerant to corticosteroids and/or cyclosporine'; amegakaryocytic thrombocytopenia 6. Sickle Cell Disease (Homozygous Hemoglobin S Disease, or Hemoglobin S β 0/+ thalassemia, or Hemoglobin SC Disease) 7. age 0-30 years 8. adequate organ function
• Exclusion Criteria:
• • 1. Females who are pregnant or breast-feeding are not eligible.
• • 2. Patients with documented uncontrolled infection at the time of study entry are not eligible.
• • 3. Karnofsky/Lansky (age appropriate) Performance Score \<60
• • 4. Demonstrated lack of compliance with medical care
• • 5. Patients who have received allogeneic HSCT within 6 months, unless being done as a boost.
• • 6. Patients with active \<Grade 2 GVHD.