Trial Outcomes & Findings for In-vivo Regulatory T Cell Enhancement With Cyclophosphamide and Sirolimus (NCT NCT01453140)
NCT ID: NCT01453140
Last Updated: 2022-03-02
Results Overview
The primary objective of this study is to determine the response rate of patients treated steroid-refractory graft-versus-host disease (GVHD) using cyclophospahmide and sirolimus combined with 3 variations of low-dose IL 2 and low-dose Vidaza with an outcome goal of promoting CD4+CD25+FoxP3+ Tregs.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
3 participants
Primary outcome timeframe
28 days to 100 days post transplant
Results posted on
2022-03-02
Participant Flow
4/11/2011 - 5/1/2013 - recruitment at Hackensack University Medical Center
Pts.must have a documented clinical diagnosis of grade II-IV acute graft-versus- host disease defined as GVHD (graft versus host disease) within 100 days p transplantation. Patients must be steroid-refractory defined as progression after 3 days of corticosteroid therapy or no response after 5 days of same. see study details.
Participant milestones
| Measure |
Cyclophosphamide and Sirolimus Only
Cyclophosphamide = 4g/m2 IV Sirolimus = 6 mg PO x 1; 2 mg PO
No patients were enrolled in the Cyclophosphamide, Sirolimus, IL-2 arm or the Cyclophosphamide, Sirolimus, IL-2, azacitidine arm
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Cyclophosphamide and Sirolimus Only
Cyclophosphamide = 4g/m2 IV Sirolimus = 6 mg PO x 1; 2 mg PO
No patients were enrolled in the Cyclophosphamide, Sirolimus, IL-2 arm or the Cyclophosphamide, Sirolimus, IL-2, azacitidine arm
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
Baseline Characteristics
In-vivo Regulatory T Cell Enhancement With Cyclophosphamide and Sirolimus
Baseline characteristics by cohort
| Measure |
Cyclophosphamide and Sirolimus
n=3 Participants
Patients will be treated in sequential cohorts of 5. In cohort A, the first 5 enrolled patients will be receive cyclophosphamide and sirolimus only
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 28 days to 100 days post transplantPopulation: No patients were enrolled in the Low dose IL-2 with Cytoxan + Sirolimus or Low dose IL-2, Vidaza, Cytoxan \& Sirolimus arms
The primary objective of this study is to determine the response rate of patients treated steroid-refractory graft-versus-host disease (GVHD) using cyclophospahmide and sirolimus combined with 3 variations of low-dose IL 2 and low-dose Vidaza with an outcome goal of promoting CD4+CD25+FoxP3+ Tregs.
Outcome measures
| Measure |
Cyclophosphamide and Sirolimus
n=3 Participants
Patients will be treated in sequential cohorts of 5. In cohort A, the first 5 enrolled patients will be receive cyclophosphamide and sirolimus only
|
Low Dose IL-2 With Cytoxan + Sirolimus
Patients in treatment arm B will be receiving low-dose IL-2 in conjunction with the cyclophosphamide and sirolimus.
|
Low Dose IL-2, Vidaza, Cytoxan & Sirolimus
Patients in treatment arm C will be receiving low-dose azacitidine (Vidaza)
|
|---|---|---|---|
|
Response Rate of Patients With Steroid-refractory Graft-versus-host Disease (GVHD) Using Cyclophospahmide and Sirolimus Combined With 3 Variations of Low-dose IL 2 and Low-dose Vidaza.
|
0 Participants
|
—
|
—
|
Adverse Events
Cyclophosphamide and Sirolimus
Serious events: 3 serious events
Other events: 0 other events
Deaths: 0 deaths
Low Dose IL-2 With Cytoxan + Sirolimus
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Low Dose IL-2, Vidaza, Cytoxan & Sirolimus
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cyclophosphamide and Sirolimus
n=3 participants at risk
Cyclophosphamide and Sirolimus - Patients will be treated in sequential cohorts of 5. In cohort A, the first 5 enrolled patients will be receive cyclophosphamide and sirolimus only
|
Low Dose IL-2 With Cytoxan + Sirolimus
Low dose IL-2 with Cyclophosphamide + Sirolimus - In cohort B, the next 5 patients will additionally receive cyclophosphamide and sirolimus \& low-dose IL-2 Low dose IL-2, low dose Vidaza, Cyclophosphamide \& Sirolimus
|
Low Dose IL-2, Vidaza, Cytoxan & Sirolimus
Low dose IL-2, Vidaza, Cytoxan \& Sirolimus - In cohort C the next 5 enrolled patients will additionally receive cyclophosphamide and Sirolimus, low-dose IL-2 and low-dose Vidaza
|
|---|---|---|---|
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Blood and lymphatic system disorders
SAE 5379 - Anemia
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Nervous system disorders
SAE 5917-neurological
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Immune system disorders
SAE 6031- Post-transplant lymphoproliferative disorder (PTLD)
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Infections and infestations
SAE 5063 - C. difficile Infection
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Hepatobiliary disorders
SAE 5878- Graft Versus Host Disease (GVHD)
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
SAE 5856-brain
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
SAE 5055- Deep vein thrombosis (DVT)
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
|
Infections and infestations
Deep Vein Thrombosis, Cytomegalovirus infection and Graft Versus Host Disease Complications
|
33.3%
1/3 • Number of events 1 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
—
0/0 • SAE data was collected over a period of 6 months for the three patients enrolled in the study
Please see SAE # 5045 and 5063 SAE # 5633 and 5878 and SAE 5055 for h/o DVT, CMV+ -dated 10-18-11 SAE 5247 for diarrhea and fever - dated 12-13-11 SAE 5379 for severe anemia dated 1-10-12 SAE 5899 for new brain masses vs abscess dated 4-6-12 SAE 5917 for confusion,malaise weakness dated 4-23-12
|
Other adverse events
Adverse event data not reported
Additional Information
Dr. Michele Donato, MD
Hackensack University Medical Center
Phone: 551-996-2000
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place