Trial Outcomes & Findings for Tacrolimus, Bortezomib, & Thymoglobulin in Preventing Low Toxicity GVHD in Donor Blood Stem Cell Transplant Patients (NCT NCT02877082)

Last Updated: 2018-04-30

Results Overview

An adverse event (AE) is defined as any untoward medical experience or change of an existing condition that occurs during or after treatment. All AEs occurring during this study, whether observed by the physician, nurse, or reported by the patient, will be graded per NCI CTCAE version 4.0 and recorded on protocol-specific case report forms. A serious adverse event (SAE) is defined as any expected or unexpected adverse event (AE, generally equivalent to CTCAE grades 3, 4 or 5) that results in any of the following outcomes: * Death * Life-threatening event * In-patient hospitalization (not required as part of the treatment) or prolongation of existing hospitalization * Persistent or significant disability/incapacity * Congenital anomaly/birth defect * Cancer * Overdose

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

Up to 6 months post-transplant

Results posted on

2018-04-30

Participant Flow

Patients were recruited from 9/9/2016 to 7/11/2017 at Winship Cancer Institute of Emory University.

Participant milestones

Participant milestones
Measure	Tacrolimus, Bortezomib, Thymoglobulin Patients receive tacrolimus IV on day -3 through day 180. Patients may receive tacrolimus PO later at the doctor's discretion. Patients receive thymoglobulin IV on days -3, -2, and -1 and bortezomib IV on day 0 and day 3. Patients undergo allogeneic bone marrow transplant on day 0. Thymoglobulin: Given IV Bortezomib: Given IV Tacrolimus: Given IV and PO
Overall Study STARTED	5
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Tacrolimus, Bortezomib, Thymoglobulin Patients receive tacrolimus IV on day -3 through day 180. Patients may receive tacrolimus PO later at the doctor's discretion. Patients receive thymoglobulin IV on days -3, -2, and -1 and bortezomib IV on day 0 and day 3. Patients undergo allogeneic bone marrow transplant on day 0. Thymoglobulin: Given IV Bortezomib: Given IV Tacrolimus: Given IV and PO
Overall Study Trial was closed early	5

Baseline Characteristics

Tacrolimus, Bortezomib, & Thymoglobulin in Preventing Low Toxicity GVHD in Donor Blood Stem Cell Transplant Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tacrolimus, Bortezomib, Thymoglobulin n=5 Participants Patients receive tacrolimus IV on day -3 through day 180. Patients may receive tacrolimus PO later at the doctor's discretion. Patients receive thymoglobulin IV on days -3, -2, and -1 and bortezomib IV on day 0 and day 3. Patients undergo allogeneic bone marrow transplant on day 0. Thymoglobulin: Given IV Bortezomib: Given IV Tacrolimus: Given IV and PO
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 6 months post-transplant

Outcome measures

Outcome measures
Measure	Tacrolimus, Bortezomib, Thymoglobulin n=5 Participants Patients receive tacrolimus IV on day -3 through day 180. Patients may receive tacrolimus PO later at the doctor's discretion. Patients receive thymoglobulin IV on days -3, -2, and -1 and bortezomib IV on day 0 and day 3. Patients undergo allogeneic bone marrow transplant on day 0. Thymoglobulin: Given IV Bortezomib: Given IV Tacrolimus: Given IV and PO
Total Number of Serious Adverse Events and Adverse Events Related to This Immunosuppressive Regimen	9 events

PRIMARY outcome

Timeframe: At 6 months post-transplant

Will use patient counts for the number of patients alive and free of severe acute graft versus host disease (GVHD) following human leukocyte antigen (HLA) matched related or unrelated donor hematopoietic peripheral blood transplant.

Outcome measures

Outcome measures
Measure	Tacrolimus, Bortezomib, Thymoglobulin n=5 Participants Patients receive tacrolimus IV on day -3 through day 180. Patients may receive tacrolimus PO later at the doctor's discretion. Patients receive thymoglobulin IV on days -3, -2, and -1 and bortezomib IV on day 0 and day 3. Patients undergo allogeneic bone marrow transplant on day 0. Thymoglobulin: Given IV Bortezomib: Given IV Tacrolimus: Given IV and PO
Number of Patients Alive and Free of Severe Acute GVHD Following HLA Matched Related or Unrelated Donor Hematopoietic Peripheral Blood Transplant	1 Participants

SECONDARY outcome

Timeframe: Up to 2 years post-transplant

Population: Data were not collected because trial closed early.

Will be summarized as percentage and 95% confidence level will be also constructed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years post-transplant

Population: Data were not collected because trial closed early.

Will be summarized as percentage and 95% confidence level will be also constructed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At 1 year post-transplant

Population: Data were not collected because trial closed early.

Will be analyzed with Kaplan Meier method and Logrank test.

Outcome measures

Outcome data not reported

Adverse Events

Tacrolimus, Bortezomib, Thymoglobulin

Serious events: 4 serious events

Other events: 0 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Tacrolimus, Bortezomib, Thymoglobulin n=5 participants at risk Patients receive tacrolimus IV on day -3 through day 180. Patients may receive tacrolimus PO later at the doctor's discretion. Patients receive thymoglobulin IV on days -3, -2, and -1 and bortezomib IV on day 0 and day 3. Patients undergo allogeneic bone marrow transplant on day 0. Thymoglobulin: Given IV Bortezomib: Given IV Tacrolimus: Given IV and PO
Blood and lymphatic system disorders Thrombotic thrombocytopenic purpura	20.0% 1/5 • Adverse event data were collected up to 180 days post-transplant.
Infections and infestations Device related infection	20.0% 1/5 • Adverse event data were collected up to 180 days post-transplant.
Psychiatric disorders Confusion	20.0% 1/5 • Adverse event data were collected up to 180 days post-transplant.
Gastrointestinal disorders Diarrhea	40.0% 2/5 • Adverse event data were collected up to 180 days post-transplant.
Respiratory, thoracic and mediastinal disorders Respiratory failure	20.0% 1/5 • Adverse event data were collected up to 180 days post-transplant.

Other adverse events

Adverse event data not reported

Additional Information

Zaid Al-Kadhimi, MD

Emory University/Winship Cancer Institute

Phone: 404-778-1900

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place