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Acute Myeloid LeukemiaPhase 2NCT03434730

Tocilizumab for the Prevention of Graft Versus Host Disease After Cord Blood Transplantation

A Phase 2 study testing Total Body Irradiation, Cyclosporine and Mycophenolate Mofetil in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The aim of the research in this study is to make participants' transplant safer by reducing the risk of developing GVHD and GVHD-related complications by giving participants a dose of the drug tocilizumab in addition to the standard approach for GVHD prevention. Read more about this study

Who is it for?
Ages 18 to 65 years
What is being tested?
Total Body Irradiation, Cyclosporine and Mycophenolate Mofetil
Could I receive a placebo?
No, everyone receives an active treatment
How many people
46 participants
Where
1 siteUnited States
Study dates
7 Feb 2018to 18 Apr 2025
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 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Acute myelogenous leukemia (AML)
  • Complete first remission (CR1) at high risk for relapse such as any of the following:
  • Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder
  • Therapy-related AML
  • White cell count at presentation > 100,000
  • Presence of extramedullary leukemia at diagnosis
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  • Any unfavorable subtype by FAB or WHO classification
  • High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high risk molecular abnormalities
  • Requirement for 2 or more induction to achieve CR1
  • Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy
  • Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician
  • Other high risk features not defined above
  • Complete second remission (CR2)
  • Primary refractory or relapsed AML with less than 10% blasts before transplant. Persistent/relapsed AML with cytogenetic, flow cytometric, or molecular aberrations in >/= 10% of cells are eligible
  • Acute lymphoblastic leukemia (ALL)
  • Complete first remission (CR1) at high risk for relapse such as any of the following:
  • White cell count at presentation > 30,000 for B-cell lineage and > 100,00 for T-cell lineage
  • Presence of any high-risk cytogenetic abnormalities such as t(9;22), t(1;19), t(4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality
  • Failure to achieve complete remission after four weeks of induction therapy
  • Persistence or recurrence of minimal residual disease on therapy
  • Any patient with newly diagnosed ALL >/= 50 years-old
  • Any patient unable to tolerate consolidation and/or maintenance chemotherapy as would have been deemed appropriate by the treating physician
  • Other high risk features not defined above
  • Complete second remission (CR2)
  • Primary refractory or relapsed ALL with less than 5% blasts before transplant. Persistent/relapsed ALL with cytogenetic, flow cytometric or molecular aberrations in >/=5% of cells are eligible.
  • Other acute leukemias: leukemias of ambiguous lineage or of other types e.g. blastic plasmacytoid dendritic cell neoplasm with less than 5% blasts. Persistent/relapsed disease with cytogenetic, flow cytometric or molecular aberrations in >/=5% of cells are eligible
  • Myelodysplastic Syndrome (MDS)/ Myeloproliferative Disorders (MPD) other than myelofibrosis:
  • International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis
  • Any IPSS risk category if life-threatening cytopenia(s) exists
  • Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia
  • MDS/ myeloproliferative disorder overlap syndromes without myelofibrosis
  • MDS/ MPD patients must have less than 10% bone marrow myeloblasts and ANC >/= 0.2 (growth factor supported if necessary) at transplant work-up
  • Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high-risk of relapse or progression if not in remission:
  • Eligible patients with aggressive histologies (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histologies) in CR
  • Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease/CR/PR with no single lesion equal to or more than 5 cm.
  • Eligible patients with HL will be without progression of disease (POD) after salvage chemotherapy with no single lesion equal to or more than 5cm.
  • Organ Function and Performance Status Criteria:
  • Karnofsky score >/= 70% (inpatient Leukemia service transfers without discharge are acceptable provided patient has equivalent KPS as if were outpatient)
  • Calculated creatinine clearance >/= 60 ml/min
  • Bilirubin < 1.5 mg/dL (unless benign congenital hyperbilirubinemia)
  • ALT </= 3 x upper limit of normal
  • Pulmonary function (spirometry and corrected DLCO) >/= 50% predicted
  • Left ventricular ejection fraction greater than 50%
  • Albumin >/= 3.0
  • Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7
  • Graft Criteria:
  • 2 CB units will be selected according to current MSKCC unit selection algorithm. High resolution 8 allele HLA typing and recipient HLA antibody profile will be performed. Unit selection will occur based on HLA-match, total nucleated cell (TNC) and CD34+ cell dose adjusted per patient body weight. The bank of origin will also be taken into account. Donor specific HLA antibodies, if present, will also be taken into consideration and may influence the selection of the graft.
  • Each CB unit must be at least 3/8 HLA-matched to the patient considering high resolution 8-allele HLA typing
  • Each CB unit will be required to have a cryopreserved TNC dose of at least 1.5 x 10^7 TNC/ recipient body weight (TNC/ kg)
  • Each CB unit will be required to have a cryopreserved CD34+ cell dose of at least 1.0 x 10^5 CD34+ cells/ recipient body weight (CD34+/kg).
  • A minimum of one domestic will be reserved as a backup unit.

Who cannot join

  • Indolent NHL or Hodgkin lymphoma with POD after most recent salvage chemotherapy
  • Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis
  • Any diagnosis without prior immunosuppressive chemotherapy within 3 months of intended admission for transplant
  • Prior checkpoint inhibitors/ blockade in the last 12 months
  • Two prior stem cell transplants of any kind
  • One prior autologous stem cell transplant within the preceding 12 months
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  • One prior allogeneic stem cell transplant within the preceding 24 months
  • Prior radiation therapy with 400cGy or more of TBI
  • Active and uncontrolled infection at time of transplantation
  • HIV infection
  • Seropositivity for HTLV-1.
  • Inadequate performance status/ organ function.
  • Pregnancy or breast feeding
  • Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

Other trials in United States

About this study

Official title: A Phase II Study of IL-6 Receptor Blockade to Ameliorate Acute Graft Versus Host Disease and Early Toxicity After Double Unit Cord Blood Transplantation in Adults With Hematologic Malignancies.

The aim of the research in this study is to make participants' transplant safer by reducing the risk of developing GVHD and GVHD-related complications by giving participants a dose of the drug tocilizumab in addition to the standard approach for GVHD prevention. Tocilizumab reduces the risk of inflammation by blocking the effect of Interleukin-6, a protein that exists in high levels in the blood when there is inflammation. Participants who receive stem cell transplants have high levels of this protein in their blood early after transplant. Therefore, the goal of this study is to reduce the risk of inflammation after transplant with the addition of Tocilizumab. This could decrease the risk of developing GVHD and GVHD-associated complications.

Being tested
Total Body Irradiation, Cyclosporine, Mycophenolate Mofetil, Tocilizumab, Filgrastim
Keywords
tocilizumab, 17-616, Memorial Sloan Kettering Cancer Center
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Myeloproliferative Disorders, Lymphoma, Non-Hodgkin, Hodgkin Disease, Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Total Body IrradiationRadiation

    Also called TBI

    Participants will receive a total dose of 400 cGy on day -2 and day -1 as 2 fractions (200 cGy x 2). Participants receiving total body irradiation (TBI) are treated in a standing position, and the treatment takes about 20 to 30 minutes.

  • CyclosporineDrug

    Also called Sandimmune

    Dilute in D5W or NS to make a 2.5 mg/ ml solution. Infuse slowly over approximately 1-4 hours (intermittent infusion) or 24 hours for continuous infusion.

  • Mycophenolate MofetilDrug

    Also called CellCept

    A 1000 mg dose should be placed in 140 ml of D5W. Administer only with D5W, over at least 2 hours.

  • TocilizumabDrug

    Also called Acemtra

    For participants < 30kg, dilute with 50mL 0.9% sodium chloride. For participants ≥ 30kg, dilute to 100ml with 0.9% sodium chloride. Administer infusion over 60 minutes with infusion set.

  • FilgrastimDrug

    Also called Granulocyte-Colony Stimulating Factor, Neupogen

    The single use prefilled syringes contain either 300 mcg or 480 mcg Filgrastim at a fill volume of 0.5 mL or 0.8 mL, respectively. For the prevention/treatment of chemotherapy induced neutropenia, the dose of filgrastim is standardized per body weight: ≤ 60 kg = 300 mcg daily subcutaneously; > 60 kg = 480 mcg subcutaneously daily.

Study groups in NCT03434730
Study groupRoleWhat they receive
Adult Participants With High Risk Hematologic MalignanciesExperimentalNot described
Receives: Radiation: Total Body Irradiation; Drug: Cyclosporine; Drug: Mycophenolate Mofetil; Drug: Tocilizumab; Drug: Filgrastim

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Median Time in Days to Onset of Grade II-IV aGVHD by Day 100 After Study Treatment

    Median Time in Days to Onset of Grade II-IV aGVHD by Day 100 After Study Treatment

    Measured: 100 days post treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Feb 2018
  2. Study start7 Feb 2018
  3. Primary completion18 Apr 2025
  4. Study completion18 Apr 2025

Resources

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
46 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03434730
Sponsor's protocol ID
17-616
Status last verified
Apr 2025
First submitted
9 Feb 2018
Last updated
26 May 2026
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Politikos I, Brown S, Fein JA, Eng S, Casem K, Chinapen S, Quach S, Scaradavou A, Cho C, Dahi P, Giralt SA, Gyurkocza B, Hanash AM, Jakubowski AA, Papadopoulos EB, Perales MA, Ponce DM, Shaffer BC, Tamari R, Young JW, Devlin S, Peled JU, Barker JN. Phase 2 trial of cyclosporine-A, mycophenolate mofetil, and tocilizumab GVHD prophylaxis in cord blood transplantation. Blood Adv. 2025 May 27;9(10):2570-2584. doi: 10.1182/bloodadvances.2024014177.

    PubMed 40423982

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