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

• Register: ClinicalTrials.gov
• Last refreshed on: 6 November 2023
• Main ID: NCT04099966
• Date of registration: 18/09/2019
• Prospective Registration: Yes
• Primary sponsor: Mitchell Cairo
• Public title: AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
• Scientific title: Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588
• Date of first enrolment: April 1, 2021
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04099966
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Mitchell S Cairo, MD

• Name: Mitchell S Cairo
• Affiliation: New York Medical College

• Name: Mitchell S Cairo, MD

Key inclusion & exclusion 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 ß 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


Age minimum: 1 Day
Age maximum: 30 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amegakaryocytic Thrombocytopenia

Non-hodgkin Lymphoma

Severe Aplastic Anemia

Hodgkin Lymphoma

Beta-Thalassemia

Diamond Blackfan Anemia

Sickle Cell Disease

Acute Leukemia

Kostmann

Intervention(s)

Drug: alpha beta depletion

Primary Outcome(s)

incidence of adverse events related to administration of a/ß CD3+/CD19+ cell depleted stem cells [Time Frame: 1 year]

Secondary Outcome(s)

incidence of GVHD following Allogeneic stem cell transplantation (AlloSCT) utilizing a/ß CD3+/CD19+ cell depletion [Time Frame: 1 year]

incidence of hematpoitic engraftment following Allogeneic stem cell transplantation (AlloSCT) utilizing a/ß CD3+/CD19+ cell depletion [Time Frame: 1 year]

Secondary ID(s)

NYMC 588

Source(s) of Monetary Support

Please refer to primary and secondary sponsors