Trial Outcomes & Findings for Phase II Study of Tocilizumab for Patients With Glucocorticoid-refractory Acute GVHD After Allogeneic Hematopoetic Stem Cell Transplant (HSCT) (NCT NCT01757197)

Results Overview

Both subjects enrolled on this study experienced failed response to glucocorticosteroid treatment but were able to tolerate tocilizumab. We were unable to collect extensive data on these 2 subjects because they both died early on in the study due to disease complications.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

Day 28

Results posted on

2018-06-11

Participant Flow

Participant milestones

Participant milestones
Measure	All Patients Toclizumab will be administered on Day 0. The administration of tocilizumab will be every 2 weeks for a total of 8 doses. Toclizumab: 8 mg/kg IV, once every 1-2 weeks. The maximum dose per infusion should not exceed 800 mg.
Overall Study STARTED	2
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients Toclizumab will be administered on Day 0. The administration of tocilizumab will be every 2 weeks for a total of 8 doses. Toclizumab: 8 mg/kg IV, once every 1-2 weeks. The maximum dose per infusion should not exceed 800 mg.
Overall Study Death	2

Baseline Characteristics

Phase II Study of Tocilizumab for Patients With Glucocorticoid-refractory Acute GVHD After Allogeneic Hematopoetic Stem Cell Transplant (HSCT)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=2 Participants Toclizumab will be administered on Day 0. The administration of tocilizumab will be every 2 weeks for a total of 8 doses. Toclizumab: 8 mg/kg IV, once every 1-2 weeks. The maximum dose per infusion should not exceed 800 mg.
Age, Continuous	60 years n=99 Participants
Sex: Female, Male Female	0 Participants n=99 Participants
Sex: Female, Male Male	2 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	1 Participants n=99 Participants
Race (NIH/OMB) White	1 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants

PRIMARY outcome

Timeframe: Day 28

Population: Not evaluable.

Both subjects enrolled on this study experienced failed response to glucocorticosteroid treatment but were able to tolerate tocilizumab. We were unable to collect extensive data on these 2 subjects because they both died early on in the study due to disease complications.

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants Toclizumab will be administered on Day 0. The administration of tocilizumab will be every 2 weeks for a total of 8 doses. Toclizumab: 8 mg/kg IV, once every 1-2 weeks. The maximum dose per infusion should not exceed 800 mg.
Number of Subjects With GVHD Who Are Tolerable to Tocilizumab After Having Failed Response to Glucocorticosteroid Treatment	2 Participants

SECONDARY outcome

Timeframe: Approximately 1 year

Population: We were unable to evaluate disease-free survival at 100 days, 6 months and one year from the time of the first tocilizumab infusion. All subjects enrolled on this study died before this time point and the study closed to enrollment due to slow accrual.

We were unable to evaluate disease-free survival at 100 days, 6 months and one year from the time of the first tocilizumab infusion. All subjects enrolled on this study died before this time point and the study closed to enrollment due to slow accrual.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

We were unable to assess the Tocilizumab response in each organ at 1 year. All subjects enrolled on this study died before this time point and the study closed to enrollment due to slow accrual.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

We were unable to evaluate the effect of tocilizumab on Karnofsky Performance Status at 1 year. All subjects enrolled on this study died before this time point and the study closed to enrollment due to slow accrual.

Outcome measures

Outcome data not reported

Adverse Events

All Patients

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=2 participants at risk Toclizumab will be administered on Day 0. The administration of tocilizumab will be every 2 weeks for a total of 8 doses. Toclizumab: 8 mg/kg IV, once every 1-2 weeks. The maximum dose per infusion should not exceed 800 mg.
Gastrointestinal disorders GVHD	50.0% 1/2 • Number of events 1
Respiratory, thoracic and mediastinal disorders pulmonary infilatrates	50.0% 1/2

Other adverse events

Adverse event data not reported

Additional Information

Usama Gergis, MD

Weill Cornell Medicine

Phone: (212) 746-2119

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place