Acute LeukemiaPhase 2NCT04099966
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
A Phase 2 study testing alpha beta depletion in people with Acute Leukemia and Severe Aplastic Anemia, run by Mitchell Cairo at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 day to 30 years
- Conditions the study is for
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma
- What is being tested?
- alpha beta depletion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Apr 2021to 31 Dec 2027
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
New York Medical College
Valhalla, New York, 10595
Mitchell S Cairo, MD, 914-594-2150, mitchell_cairo@nymc.edu
Lauren Harrison, MSN, 6172857844, lauren_harrison@nymc.edu
About this study
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
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.
Read the 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.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia
- Being tested
- alpha beta depletion
- Keywords
- allogeneic stem cell transplantation, t-cell depletion, alpha beta cell depletion
- Medical subject headings
- Anemia, Aplastic, Lymphoma, Non-Hodgkin, Hodgkin Disease, Neutropenia, Severe Congenital, Autosomal Recessive 3, Anemia, Diamond-Blackfan, Anemia, Sickle Cell, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alpha beta depletionDrug
Also called α/β CD3+/CD19+ cell depletion
donor cells will be collected and subsequently undergo α/β CD3+/CD19+ cell depletion.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: alpha beta depletion |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 1 year
Secondary outcomes (2)
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
Measured: 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
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2019
- Study start1 Apr 2021
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mitchell Cairo
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Mitchell Cairo, New York Medical College
- Investigators
- Mitchell S Cairo, Study chair, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04099966
- Sponsor's protocol ID
- NYMC 588
- Status last verified
- Oct 2026
- First submitted
- 18 Sep 2019
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No