Stem cell transplantation for blood disorders using specific cell depletion techniques

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

This study is testing a new way of using stem cell transplants for children and young adults with blood disorders to see if removing certain cells helps them recover better and improve their quality of life.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 1 Day to 30 Years
• Sex: All
• Sponsor: New York Medical College (other)
• Locations: 1 site (Valhalla, New York)
• Trial ID: NCT04099966 on ClinicalTrials.gov

What this trial studies

This clinical trial focuses on children, adolescents, and young adults with malignant and non-malignant hematologic diseases who are undergoing allogeneic stem cell transplantation (AlloSCT). Participants will receive stem cells that have undergone α/β T-cell and CD19+ B-cell depletion, with treatment tailored based on their specific disease and health status. The study will monitor various outcomes including engraftment, immune reconstitution, and quality of life. Standard care will be provided alongside this innovative approach.

Who should consider this trial

Good fit: Ideal candidates include children and young adults with high-risk acute leukemia, severe aplastic anemia, or specific types of lymphoma.

Not a fit: Patients with low-risk hematologic conditions or those not meeting the specific eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could improve transplant outcomes and reduce complications for patients with hematologic diseases.

How similar studies have performed: While similar approaches have been explored, this specific method of α/β T-cell and CD19+ B-cell depletion in AlloSCT is relatively novel.

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.

Where this trial is running

Valhalla, New York

• New York Medical College — Valhalla, New York, United States (RECRUITING)

Study contacts

• Study coordinator: Mitchell S Cairo, MD

Conditions: Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease