A Phase 2 interventional study of Tocilizumab and Fludarabine in Hematologic Malignancy and Bone Marrow Transplant, sponsored by Weill Medical College of Cornell University. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-01-06.
Sponsored by Weill Medical College of Cornell University · Phase 2, Interventional, and Treatment
The purpose of this study is to evaluate the safety of reducing and ultimately eliminating anti-thymocyte globulin (ATG) from the haplo-cord transplant conditioning regimen and replacing it with tocilizumab, an IL-6 receptor monoclonal antibody, to improve immune reconstitution and reduce relapse while preserving low rates of graft failure and graft versus host disease (GVHD).
This study is a prospective phase II non-inferiority study investigating tocilizumab as a potential alternative to anti-thymocyte globulin (ATG) in haplo-cord transplantation. It is a single-center study based at Weill Cornell Medicine/NewYork Presbyterian Hospital.
The hypothesis is that tocilizumab is a safe and effective alternative to ATG in haplo-cord transplantation, facilitating transient engraftment of the haplo-identical stem cell graft without prolonged neutropenia or second nadir prior to durable cord engraftment while also preventing graft versus host disease (GVHD).
This study plans to enroll patients with hematologic malignancies in need of alternate donor transplant. All subjects will be conditioned with fludarabine, melphalan and total body irradiation (TBI), followed by a single dose of tocilizumab 8 mg/kg on Day -1. Patients will be enrolled into 4 successive cohorts, initially administering the current standard 3 doses of ATG 1.5 mg/kg (total 4.5 mg/kg). In the absence of safety signals, we will drop one dose of ATG in successive cohorts until the drug ultimately has been eliminated.
The primary endpoint of the study is successful haplo-derived neutrophil engraftment. Treatment will only be of interest if there is evidence that this rate is greater than 60%. If there are 4 or fewer successes, that dose group will be deemed unacceptable and the next higher ATG dose for which there were 5 or more success will be expanded.
1,464 studies on the registry are indexed under Hematologic Neoplasms; 433 are open to participants now.
This study's enrollment of 21 is below the median of 45 across 1,068 interventional studies indexed under Hematologic Neoplasms.
Browse Hematologic Neoplasms studies →Weill Medical College of Cornell University is the lead sponsor of 867 studies on the registry; 160 are open to participants now.
Of its 119 completed or terminated interventional studies of FDA-regulated products, 91 (76%) have results posted.
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Subject must have a confirmed diagnosis of one of the following:
Acceptable organ function as defined below:
Exclusion Criteria:
Anti-thymocyte Globulin (ATG) 1.5 mg/kg administered on Day -5, Day -3 and Day -1 of the transplant conditioning regimen. * Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.
Drug: Tocilizumab · Drug: Fludarabine · Drug: Melphalan · Drug: Anti-thymocyte globulin (rabbit) · Radiation: Total Body Irradiation
Anti-thymocyte Globulin (ATG) 1.5 mg/kg administered on Day -5, and Day -3 of the transplant conditioning regimen. * Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.
Drug: Tocilizumab · Drug: Fludarabine · Drug: Melphalan · Drug: Anti-thymocyte globulin (rabbit) · Radiation: Total Body Irradiation
Anti-thymocyte Globulin (ATG) 1.5 mg/kg administered on Day -5 of the transplant conditioning regimen. * Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.
Drug: Tocilizumab · Drug: Fludarabine · Drug: Melphalan · Drug: Anti-thymocyte globulin (rabbit) · Radiation: Total Body Irradiation
* Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.
Drug: Tocilizumab · Drug: Fludarabine · Drug: Melphalan · Radiation: Total Body Irradiation
Tocilizumab 8 mg/kg intravenously administered as a single dose on Day -1 of transplant conditioning regimen
Also known as: Actemra
Fludarabine 30 mg/m2 intravenously administered on Day -7, Day -6, Day -5, Day -4, Day -3 of transplant conditioning regimen if under the age of 60. If over the age of 60, Fludarabine 30 mg/m2 intravenously administered on Day -5, Day -4 and Day -3 of transplant conditioning regimen.
Also known as: Fludara
Melphalan 140 mg/m2 intravenously administered on Day -2 of transplant conditioning regimen.
Also known as: Alkeran
Anti-thymocyte globulin (ATG) 1.5 mg/kg
Also known as: Thymoglobulin
Total Body Irradiation (TBI) 2 Gray, administered on Day -4 and Day -3 of transplant conditioning regimen
Percentage of Subjects With Successful Haplo-derived Neutrophil Engraftment
This is defined as: 1. Achieve an absolute neutrophil count (ANC) of 500 cells/microL for three consecutive days with the first on or prior to Day +21 post-transplant, AND 2. Absence of a second nadir - a drop in the ANC to \<300 cells/microL for five consecutive days - after initial neutrophil recovery.
Time frame: 21 days post-transplant
Progression-Free Survival
Time elapsed between Day 0 and progression of the underlying malignancy for which the transplant was performed, assessed up to 5 years post-transplant.
Time frame: 5 years post-transplant
Overall Survival
Time elapsed between Day 0 and death from any cause, assessed up to 5 years post-transplant.
Time frame: 5 years post-transplant
Transplant-Related Mortality
Proportion of deaths which cannot be explained by persistence, relapse or progression of the underlying malignancy once the preparative regimen starts, assessed up to 5 years post-transplant.
Time frame: 5 years post-transplant
Proportion of Platelet Engraftment Success
Proportion of patients who successfully achieve platelet engraftment, defined as a platelet count of \>20k/microL for three consecutive days without transfusion support for seven consecutive days.
Time frame: 6 months post-transplant
Proportion of Failure of the Haplo-Graft
Proportion of patients with a failed haplo-graft, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to \<0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation (second nadir)
Time frame: 21 days post-transplant
Proportion of Acute Graft-versus-Host Disease
Proportion of patients who develop acute graft-versus-host disease
Time frame: 1 year post-transplant
Percentage of Chronic Graft-versus-Host Disease
Percentage of patients who develop chronic graft-versus-host disease
Time frame: 5 years post-transplant
| Milestone | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| Started | 10 | 10 | 1 | 0 |
| Completed | 10 | 10 | 1 | 0 |
| Not completed | 0 | 0 | 0 | 0 |
This is defined as: 1. Achieve an absolute neutrophil count (ANC) of 500 cells/microL for three consecutive days with the first on or prior to Day +21 post-transplant, AND 2. Absence of a second nadir - a drop in the ANC to \<300 cells/microL for five consecutive days - after initial neutrophil recovery.
| percentage of participants | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| Percentage of Subjects With Successful Haplo-derived Neutrophil Engraftment | 80 | 50 | 0 | — |
Time elapsed between Day 0 and progression of the underlying malignancy for which the transplant was performed, assessed up to 5 years post-transplant.
Results for this outcome have not been posted.
Time elapsed between Day 0 and death from any cause, assessed up to 5 years post-transplant.
Results for this outcome have not been posted.
Proportion of deaths which cannot be explained by persistence, relapse or progression of the underlying malignancy once the preparative regimen starts, assessed up to 5 years post-transplant.
Results for this outcome have not been posted.
Proportion of patients who successfully achieve platelet engraftment, defined as a platelet count of \>20k/microL for three consecutive days without transfusion support for seven consecutive days.
| Participants | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| Proportion of Platelet Engraftment Success | 10 | 10 | 0 | 0 |
Proportion of patients with a failed haplo-graft, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to \<0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation (second nadir)
| Participants | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| Proportion of Failure of the Haplo-Graft | 2 | 5 | 1 | — |
Proportion of patients who develop acute graft-versus-host disease
| Participants | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| Proportion of Acute Graft-versus-Host Disease | 2 | 1 | 1 | 0 |
Percentage of patients who develop chronic graft-versus-host disease
Results for this outcome have not been posted.
Collected over All-cause mortality is being assessed up to 5 years. SAEs and other non-serious AEs were assessed up to 1 year.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| ATG Group I | 5/10 (50%) | 8/10 (80%) | 10/10 (100%) |
| ATG Group II | 1/10 (10%) | 9/10 (90%) | 10/10 (100%) |
| ATG Group III | 1/1 (100%) | 1/1 (100%) | 1/1 (100%) |
| ATG Group IV | — | — | — |
| Event | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| Acute Graft-versus-Host DiseaseInvestigations | 1/10 | 0/10 | 1/1 | — |
| Delayed EngraftmentInvestigations | 0/10 | 0/10 | 1/1 | — |
| Hypovolemic shockGeneral disorders | 0/10 | 0/10 | 1/1 | — |
| Septic ShockInvestigations | 2/10 | 1/10 | 1/1 | — |
| BacteremiaInfections and infestations | 2/10 | 2/10 | 0/1 | — |
| DiarrheaGastrointestinal disorders | 2/10 | 1/10 | 0/1 | — |
| SepsisInfections and infestations | 2/10 | 1/10 | 0/1 | — |
| Hemophagocytic lymphohistiocytosisBlood and lymphatic system disorders | 0/10 | 1/10 | 0/1 | — |
| CholecystitisHepatobiliary disorders | 1/10 | 0/10 | 0/1 | — |
| Colitis - CytomegalovirusInfections and infestations | 0/10 | 1/10 | 0/1 | — |
| Event | ATG Group I | ATG Group II | ATG Group III | ATG Group IV |
|---|---|---|---|---|
| AnemiaBlood and lymphatic system disorders | 9/10 | 10/10 | 1/1 | — |
| HypoalbuminemiaMetabolism and nutrition disorders | 3/10 | 3/10 | 1/1 | — |
| HypocalcemiaMetabolism and nutrition disorders | 3/10 | 2/10 | 1/1 | — |
| HypokalemiaMetabolism and nutrition disorders | 4/10 | 4/10 | 1/1 | — |
| HypomagnesemiaMetabolism and nutrition disorders | 0/10 | 3/10 | 1/1 | — |
| Platelet count decreasedInvestigations | 6/10 | 4/10 | 1/1 | — |
| White blood cell decreasedInvestigations | 1/10 | 3/10 | 1/1 | — |
| BacteremiaInfections and infestations | 2/10 | 3/10 | 1/1 | — |
| AnorexiaMetabolism and nutrition disorders | 4/10 | 2/10 | 0/1 | — |
| Clostridium difficile colitisInfections and infestations | 0/10 | 4/10 | 0/1 | — |
No subjects were enrolled to ATG Group IV; study enrollment was stopped before accrual reached that cohort.
| Age, Customized(Participants) | ATG Group I | ATG Group II | ATG Group III | ATG Group IV | Total |
|---|---|---|---|---|---|
| 18-29 years | 1 | 2 | 0 | 0 | 3 |
| 30-39 years | 1 | 2 | 0 | 0 | 3 |
| 40-49 years | 1 | 2 | 0 | 0 | 3 |
| 50-59 years | 0 | 1 | 0 | 0 | 1 |
| 60-69 years | 7 | 3 | 1 | 0 | 11 |
| Sex: Female, Male(Participants) | ATG Group I | ATG Group II | ATG Group III | ATG Group IV | Total |
|---|---|---|---|---|---|
| Female | 5 | 5 | 1 | 0 | 11 |
| Male | 5 | 5 | 0 | 0 | 10 |
| Ethnicity (NIH/OMB)(Participants) | ATG Group I | ATG Group II | ATG Group III | ATG Group IV | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 3 | 0 | 0 | 4 |
| Not Hispanic or Latino | 8 | 7 | 1 | 0 | 16 |
| Unknown or Not Reported | 1 | 0 | 0 | 0 | 1 |
| Race (NIH/OMB)(Participants) | ATG Group I | ATG Group II | ATG Group III | ATG Group IV | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Asian | 1 | 1 | 0 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 1 | 2 | 0 | 0 | 3 |
| White | 8 | 6 | 1 | 0 | 15 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 1 | 0 | 0 | 1 |
| Region of Enrollment(participants) | ATG Group I | ATG Group II | ATG Group III | ATG Group IV | Total |
|---|---|---|---|---|---|
| United States | 10 | 10 | 1 | — | 21 |
| Primary Malignancy(Participants) | ATG Group I | ATG Group II | ATG Group III | ATG Group IV | Total |
|---|---|---|---|---|---|
| Acute Myeloid Leukemia | 5 | 5 | 0 | 0 | 10 |
| Myelodysplastic Syndrome | 1 | 3 | 1 | 0 | 5 |
| Myeloproliferative Neoplasm | 1 | 1 | 0 | 0 | 2 |
| Non-Hodgkin Lymphoma | 2 | 1 | 0 | 0 | 3 |
| Other Acute Leukemia | 1 | 0 | 0 | 0 | 1 |
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Weill Medical College of Cornell University