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

Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588

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.

Study Overview

• Status: Recruiting
• Conditions: Hodgkin Lymphoma; Sickle Cell Disease; Acute Leukemia; Beta-Thalassemia; Non-hodgkin Lymphoma; Severe Aplastic Anemia; Diamond Blackfan Anemia; Amegakaryocytic Thrombocytopenia; Kostmann
• Intervention / Treatment: Drug: alpha beta depletion

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.

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Lauren Harrison, RN; Phone Number: 6172857844; Email: lauren_harrison@nymc.edu
• Study Contact Backup: Name: Mitchell S Cairo, MD; Phone Number: 9145942150; Email: mitchell_cairo@nymc.edu

Study Locations

• United States
  • New York
    • Valhalla, New York, United States, 10595
      • Recruiting
      • New York Medical College
      • Contact: Lauren Harrison, MSN; Phone Number: 6172857844; Email: lauren_harrison@nymc.edu
      • Contact: Mitchell S Cairo, MD; Phone Number: 914-594-2150; Email: mitchell_cairo@nymc.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 28 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: alpha beta cell depletion; 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; donor cells will be collected and subsequently undergo α/β CD3+/CD19+ cell depletion.; Other Names: α/β CD3+/CD19+ cell depletion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
incidence of hematpoitic engraftment following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion	patients will have routine chimerism performed to monitoring engraftment of donor cells	1 year
incidence of GVHD following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion	patients will be monitored post transplant for signs of acute and chronic GVHD	1 year

Collaborators and Investigators

• Sponsor: Mitchell Cairo
• Investigators: Study Chair: Mitchell S Cairo, New York Medical College

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2021
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: September 18, 2019
• First Submitted That Met QC Criteria: September 20, 2019
• First Posted (Actual): September 23, 2019

Study Record Updates

• Last Update Posted (Actual): October 26, 2023
• Last Update Submitted That Met QC Criteria: October 24, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: allogeneic stem cell transplantation; t-cell depletion; alpha beta cell depletion
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Blood Platelet Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Hypoplastic, Congenital; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Red-Cell Aplasia, Pure; Lymphoma; Hematologic Diseases; Anemia; Anemia, Sickle Cell; Thrombocytopenia; Thalassemia; beta-Thalassemia; Anemia, Aplastic; Anemia, Diamond-Blackfan
• Other Study ID Numbers: NYMC 588

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No