Clinical trial NCT04099966: AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Official title: Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Acute Leukemia
Severe Aplastic Anemia
Non-hodgkin Lymphoma
Hodgkin Lymphoma
Kostmann
Diamond Blackfan Anemia
Amegakaryocytic Thrombocytopenia
Sickle Cell Disease
Beta-Thalassemia
Keywords: allogeneic stem cell transplantation
t-cell depletion
alpha beta cell depletion
Lead sponsor: New York Medical College
Sponsor class: OTHER
First posted: 2019-09-23
Enrollment: 20
Sex: ALL
Minimum age (years): 1
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: incidence of adverse events related to administration of α/β CD3+/CD19+ cell depleted stem cells
timeFrame: 1 year
Secondary outcomes:
measure: incidence of hematpoitic engraftment following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion
timeFrame: 1 year
measure: incidence of GVHD following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion
timeFrame: 1 year
Other outcomes:
Locations:
city: Valhalla
country: United States
facility: New York Medical College
state: New York
Study officials:
affiliation: New York Medical College
name: Mitchell S Cairo
role: STUDY_CHAIR
References: