Trial Outcomes & Findings for Tocilizumab for the Prevention of Graft Versus Host Disease After Cord Blood Transplantation (NCT NCT03434730)
NCT ID: NCT03434730
Last Updated: 2026-05-26
Results Overview
Median Time in Days to Onset of Grade II-IV aGVHD by Day 100 After Study Treatment
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
46 participants
Primary outcome timeframe
100 days post treatment
Results posted on
2026-05-26
Participant Flow
Participant milestones
| Measure |
Adult Participants With High Risk Hematologic Malignancies
Total Body Irradiation: 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.
|
|---|---|
|
Overall Study
STARTED
|
46
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
16
|
Reasons for withdrawal
| Measure |
Adult Participants With High Risk Hematologic Malignancies
Total Body Irradiation: 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.
|
|---|---|
|
Overall Study
Death
|
9
|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Relapsed Disease
|
5
|
Baseline Characteristics
Tocilizumab for the Prevention of Graft Versus Host Disease After Cord Blood Transplantation
Baseline characteristics by cohort
| Measure |
Adult Participants With High Risk Hematologic Malignancies
n=46 Participants
Total Body Irradiation: 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.
|
|---|---|
|
Age, Continuous
|
45 years
n=20 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
9 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
36 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Asian
|
8 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=20 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=20 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=20 Participants
|
|
Region of Enrollment
United States
|
46 Participants
n=20 Participants
|
PRIMARY outcome
Timeframe: 100 days post treatmentMedian Time in Days to Onset of Grade II-IV aGVHD by Day 100 After Study Treatment
Outcome measures
| Measure |
Adult Participants With High Risk Hematologic Malignancies
n=46 Participants
Total Body Irradiation: 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.
Cyclosporine: 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 Mofetil: A 1000 mg dose should be placed in 140 ml of D5W. Administer only with D5W, over at least 2 hours.
Tocilizumab: 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.
Filgrastim: 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.
|
|---|---|
|
Median Time in Days to Onset of Grade II-IV aGVHD by Day 100 After Study Treatment
|
34 days
Interval 14.0 to 90.0
|
Adverse Events
Adult Participants With High Risk Hematologic Malignancies
Serious events: 5 serious events
Other events: 26 other events
Deaths: 16 deaths
Serious adverse events
| Measure |
Adult Participants With High Risk Hematologic Malignancies
n=45 participants at risk
Total Body Irradiation: 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.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
General disorders
Death, NOS
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Infections and infestations
Infections and infestations - Other, specify
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Infections and infestations
Sepsis
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
Other adverse events
| Measure |
Adult Participants With High Risk Hematologic Malignancies
n=45 participants at risk
Total Body Irradiation: 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.
|
|---|---|
|
Renal and urinary disorders
Acute Renal Failure
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Metabolism and nutrition disorders
Anorexia
|
13.3%
6/45 • through 100 days post treatment, up to 160 days total
|
|
Infections and infestations
Bacteremia
|
13.3%
6/45 • through 100 days post treatment, up to 160 days total
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Cardiac disorders
Cardiac Arrest
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Cardiac disorders
Heart failure
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Hepatobiliary disorders
Hepatic Failure
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Vascular disorders
Hypertension
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.7%
3/45 • through 100 days post treatment, up to 160 days total
|
|
Injury, poisoning and procedural complications
Infusion Related Reaction
|
8.9%
4/45 • through 100 days post treatment, up to 160 days total
|
|
Gastrointestinal disorders
Mucositis
|
2.2%
1/45 • through 100 days post treatment, up to 160 days total
|
|
General disorders
Pain
|
33.3%
15/45 • through 100 days post treatment, up to 160 days total
|
|
Skin and subcutaneous tissue disorders
Rash-Maculopapular
|
6.7%
3/45 • through 100 days post treatment, up to 160 days total
|
|
Infections and infestations
Sepsis
|
4.4%
2/45 • through 100 days post treatment, up to 160 days total
|
Additional Information
Ioannis Politikos, MD
Memorial Sloan Kettering Cancer Center
Phone: 212-639-2054
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place