Trial Outcomes & Findings for Study on the Safety and Effectiveness of VELCADE® in the Treatment of Graft-Versus-Host Disease (NCT NCT00408928)
NCT ID: NCT00408928
Last Updated: 2025-05-04
Results Overview
Response is the primary endpoint of this study and will be scored on day 21 (3 weeks after the first dose of VELCADE) and every 3 weeks subsequently. Patients who progress or expire before the end of the study will be considered non-responders. Patients are evaluated for response in an organ if they have AGVHD in that organ at the start of treatment with VELCADE or if AGVHD develops after the start of VELCADE, but before the time period of evaluation. Complete response in an organ is defined as no evidence clinical or biochemical signs of AGVHD. For the overall assessment, it is defined as complete resolution of rash, abnormal LFTs, and absence of diarrhea attributed to AGVHD. Partial response is defined as a one stage decrease in any organ system without worsening in other organ systems.
COMPLETED
PHASE2
11 participants
Through 30 days post-treatment
2025-05-04
Participant Flow
Participant milestones
| Measure |
Bortezomib for Treatment of GHVD
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Bortezomib for Treatment of GHVD
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Death
|
1
|
|
Overall Study
Infection indistinguishable from GVHD
|
1
|
Baseline Characteristics
Study on the Safety and Effectiveness of VELCADE® in the Treatment of Graft-Versus-Host Disease
Baseline characteristics by cohort
| Measure |
Bortezomib for Treatment of GHVD
n=11 Participants
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=99 Participants
|
|
Age, Continuous
|
45.68 years
STANDARD_DEVIATION 15.43 • n=99 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Through 30 days post-treatmentResponse is the primary endpoint of this study and will be scored on day 21 (3 weeks after the first dose of VELCADE) and every 3 weeks subsequently. Patients who progress or expire before the end of the study will be considered non-responders. Patients are evaluated for response in an organ if they have AGVHD in that organ at the start of treatment with VELCADE or if AGVHD develops after the start of VELCADE, but before the time period of evaluation. Complete response in an organ is defined as no evidence clinical or biochemical signs of AGVHD. For the overall assessment, it is defined as complete resolution of rash, abnormal LFTs, and absence of diarrhea attributed to AGVHD. Partial response is defined as a one stage decrease in any organ system without worsening in other organ systems.
Outcome measures
| Measure |
Bortezomib for Treatment of GHVD
n=8 Participants
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
Response to Bortezomib (VELCADE®)
Complete response
|
2 participants
|
|
Response to Bortezomib (VELCADE®)
Partial response
|
2 participants
|
|
Response to Bortezomib (VELCADE®)
No response
|
4 participants
|
SECONDARY outcome
Timeframe: Through 30 days post-traeatmentObserve the toxicities of VELCADE® when administered to recipients of allogeneic hematopoietic stem cell transplant in the setting of steroid refractory or steroid dependent acute graft-versus-host disease.
Outcome measures
| Measure |
Bortezomib for Treatment of GHVD
n=8 Participants
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
Number of Toxicities Related to Bortezomib (VELCADE®)
|
18 toxicities
|
Adverse Events
Bortezomib for Treatment of GHVD
Serious adverse events
| Measure |
Bortezomib for Treatment of GHVD
n=11 participants at risk
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
General disorders
Nausea
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Vomiting
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Diarrhea
|
36.4%
4/11 • Number of events 5
|
|
General disorders
GVHD
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Hypotension
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Seizure
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Cystitis
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Lack of appetite
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Febrile neutropenia
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Infection
|
9.1%
1/11 • Number of events 1
|
|
General disorders
Death
|
9.1%
1/11 • Number of events 1
|
Other adverse events
| Measure |
Bortezomib for Treatment of GHVD
n=11 participants at risk
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
|
|---|---|
|
Nervous system disorders
Neuropathy
|
27.3%
3/11 • Number of events 3
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place