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

Phase 2
Recruiting
Conditions
Diamond Blackfan Anemia
Non-hodgkin Lymphoma
Sickle Cell Disease
Acute Leukemia
Severe Aplastic Anemia
Hodgkin Lymphoma
Kostmann
Amegakaryocytic Thrombocytopenia
Beta-Thalassemia
Interventions
Drug: alpha beta depletion
Registration Number
NCT04099966
Lead Sponsor
Mitchell Cairo
Brief Summary

Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.

Detailed Description

Patients wiith selected malignant or non-malignant conditions meeting eligibility criteria will be enrolled on this study. Patients will receive one of either full intensity, reduced intensity, or reduced toxicity conditioning appropriate based on disease, disease status, organ function and performance status and will undergo α/β T-cell and CD 19+ B cell depleted alloSCT.

Patients will be following for engraftment, chimerism, immune reconstitution, GVHD and QOL.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
20
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

  4. 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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
alpha beta cell depletionalpha beta depletionMatched allogeneic donor stem cells will be processed utilizing α/β CD3+/CD19+ cell depletion with the Prodigy system. Standard pre-conditioning and post-transplant motioning will be given.
Primary Outcome Measures
NameTimeMethod
incidence of adverse events related to administration of α/β CD3+/CD19+ cell depleted stem cells1 year

patients will be monitored for any adverse events related to administration of α/β CD3+/CD19+ cell depleted stem cells

Secondary Outcome Measures
NameTimeMethod
incidence of GVHD following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion1 year

patients will be monitored post transplant for signs of acute and chronic GVHD

incidence of hematpoitic engraftment following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion1 year

patients will have routine chimerism performed to monitoring engraftment of donor cells

Trial Locations

Locations (1)

New York Medical College

🇺🇸

Valhalla, New York, United States

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