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

NCT04099966 | Recruiting Phase 2 FDA Drug

• 1 other identifier: NYMC 588
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (1)

• incidence of adverse events related to administration of α/β CD3+/CD19+ cell depleted stem cells

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

  1 year

Secondary Outcomes (2)

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

  1 year

• incidence of GVHD following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion

  1 year

Study Arms (1)

alpha beta cell depletion

EXPERIMENTAL

Matched 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.

Drug: alpha beta depletion

Interventions

alpha beta depletion DRUG

donor cells will be collected and subsequently undergo α/β CD3+/CD19+ cell depletion.

Also known as: α/β CD3+/CD19+ cell depletion

alpha beta cell depletion

Eligibility Criteria

• Age: 1 Day - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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;
• 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
• 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
• \. 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

You may not qualify if:

• Females who are pregnant or breast-feeding are not eligible.
• Patients with documented uncontrolled infection at the time of study entry are not eligible.
• Karnofsky/Lansky (age appropriate) Performance Score \<60
• Demonstrated lack of compliance with medical care
• Patients who have received allogeneic HSCT within 6 months, unless being done as a boost.
• Patients with active \<Grade 2 GVHD.

Sponsors & Collaborators

• Mitchell Cairo: lead

Study Sites (1)

New York Medical College

Valhalla, New York, 10595, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic; Lymphoma, Non-Hodgkin; Hodgkin Disease; Neutropenia, Severe Congenital, Autosomal Recessive 3; Anemia, Diamond-Blackfan; Anemia, Sickle Cell; beta-Thalassemia

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Anemia, Hypoplastic, Congenital; Red-Cell Aplasia, Pure; Congenital Bone Marrow Failure Syndromes; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia

Study Officials

• Mitchell S Cairo

  New York Medical College

  STUDY CHAIR

Central Study Contacts

Mitchell S Cairo, MD

CONTACT

9145942150; mitchell_cairo@nymc.edu

Lauren Harrison, RN

CONTACT

6172857844; lauren_harrison@nymc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Co-Investgiator

Study Record Dates

• First Submitted: September 18, 2019
• First Posted: September 23, 2019
• Study Start: April 1, 2021
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: August 8, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)