Trial Outcomes & Findings for Comparing Cyclophosphamide and Abatacept With Standard of Care Treatment Following Stem Cell Transplantation (NCT NCT03680092)
NCT ID: NCT03680092
Last Updated: 2025-10-02
Results Overview
The occurrence of moderate and severe chronic GVHD at one year post Chronic GVHD will be diagnosed and staged according to the previously published and widely accepted National Institutes of Health consensus criteria
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
43 participants
Primary outcome timeframe
through study completion, an average of 1 year
Results posted on
2025-10-02
Participant Flow
Participant milestones
| Measure |
Cyclophosphamide and Abatacept
The GVHD prophylaxis regimen on the experimental arm will consist of high dose Cyclophosphamide on Days +3 and +4 followed by abatacept for 6 months. Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
Cyclophosphamide: Day 3 and day 4 following transplant. Dosing will be based on patients' actual weight up to 120% of ideal body weight, above which it will be based on adjusted ideal body weight (ideal weight plus 50% of the difference between ideal and actual weight).
abatacept: Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
|
Methotrexate and Tacrolimus
The GVHD prophylaxis regimen on the standard of care arm will consist of methotrexate on Days +1,+3, +6 and +11 and tacrolimus
Methotrexate: standard of care Methotrexate 5 mg/m2 on Days 1, 3, 6 and 11
Tacrolimus: Tacrolimus per institutional guidelines
|
|---|---|---|
|
Overall Study
STARTED
|
28
|
15
|
|
Overall Study
COMPLETED
|
25
|
15
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Comparing Cyclophosphamide and Abatacept With Standard of Care Treatment Following Stem Cell Transplantation
Baseline characteristics by cohort
| Measure |
Cyclophosphamide and Abatacept
n=25 Participants
The GVHD prophylaxis regimen on the experimental arm will consist of high dose Cyclophosphamide on Days +3 and +4 followed by abatacept for 6 months. Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
Cyclophosphamide: Day 3 and day 4 following transplant. Dosing will be based on patients' actual weight up to 120% of ideal body weight, above which it will be based on adjusted ideal body weight (ideal weight plus 50% of the difference between ideal and actual weight).
abatacept: Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
|
Methotrexate and Tacrolimus
n=15 Participants
The GVHD prophylaxis regimen on the standard of care arm will consist of methotrexate on Days +1,+3, +6 and +11 and tacrolimus
Methotrexate: standard of care Methotrexate 5 mg/m2 on Days 1, 3, 6 and 11
Tacrolimus: Tacrolimus per institutional guidelines
|
Total
n=40 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
21 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
34 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
25 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
40 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: through study completion, an average of 1 yearThe occurrence of moderate and severe chronic GVHD at one year post Chronic GVHD will be diagnosed and staged according to the previously published and widely accepted National Institutes of Health consensus criteria
Outcome measures
| Measure |
Cyclophosphamide and Abatacept
n=24 Participants
The GVHD prophylaxis regimen on the experimental arm will consist of high dose Cyclophosphamide on Days +3 and +4 followed by abatacept for 6 months. Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
Cyclophosphamide: Day 3 and day 4 following transplant. Dosing will be based on patients' actual weight up to 120% of ideal body weight, above which it will be based on adjusted ideal body weight (ideal weight plus 50% of the difference between ideal and actual weight).
abatacept: Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
|
Methotrexate and Tacrolimus
n=14 Participants
The GVHD prophylaxis regimen on the standard of care arm will consist of methotrexate on Days +1,+3, +6 and +11 and tacrolimus
Methotrexate: standard of care Methotrexate 5 mg/m2 on Days 1, 3, 6 and 11
Tacrolimus: Tacrolimus per institutional guidelines
|
|---|---|---|
|
Occurrence of Moderate and Severe Chronic GVHD at One Year Post Transplant
|
0 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: through study completion, an average of 1 yearGVHD- and relapse- free survival will be defined as the absence of acute GVHD Grade III or IV or moderate or severe chronic GVHD or relapse or non-relapse mortality by one year post transplant. Acute GVHD will be diagnosed and graded according to Glucksberg criteria
Outcome measures
| Measure |
Cyclophosphamide and Abatacept
n=24 Participants
The GVHD prophylaxis regimen on the experimental arm will consist of high dose Cyclophosphamide on Days +3 and +4 followed by abatacept for 6 months. Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
Cyclophosphamide: Day 3 and day 4 following transplant. Dosing will be based on patients' actual weight up to 120% of ideal body weight, above which it will be based on adjusted ideal body weight (ideal weight plus 50% of the difference between ideal and actual weight).
abatacept: Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
|
Methotrexate and Tacrolimus
n=14 Participants
The GVHD prophylaxis regimen on the standard of care arm will consist of methotrexate on Days +1,+3, +6 and +11 and tacrolimus
Methotrexate: standard of care Methotrexate 5 mg/m2 on Days 1, 3, 6 and 11
Tacrolimus: Tacrolimus per institutional guidelines
|
|---|---|---|
|
GVHD- and Relapse- Free Survival by One Year Post Transplant
|
15 Participants
|
4 Participants
|
Adverse Events
Cyclophosphamide and Abatacept
Serious events: 9 serious events
Other events: 5 other events
Deaths: 2 deaths
Methotrexate and Tacrolimus
Serious events: 13 serious events
Other events: 10 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Cyclophosphamide and Abatacept
n=25 participants at risk
The GVHD prophylaxis regimen on the experimental arm will consist of high dose Cyclophosphamide on Days +3 and +4 followed by abatacept for 6 months. Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
Cyclophosphamide: Day 3 and day 4 following transplant. Dosing will be based on patients' actual weight up to 120% of ideal body weight, above which it will be based on adjusted ideal body weight (ideal weight plus 50% of the difference between ideal and actual weight).
abatacept: Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
|
Methotrexate and Tacrolimus
n=15 participants at risk
The GVHD prophylaxis regimen on the standard of care arm will consist of methotrexate on Days +1,+3, +6 and +11 and tacrolimus
Methotrexate: standard of care Methotrexate 5 mg/m2 on Days 1, 3, 6 and 11
Tacrolimus: Tacrolimus per institutional guidelines
|
|---|---|---|
|
Infections and infestations
Febrile Neutropenia
|
16.0%
4/25 • Number of events 5 • 1 year
|
66.7%
10/15 • Number of events 11 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
4.0%
1/25 • Number of events 2 • 1 year
|
0.00%
0/15 • 1 year
|
|
Infections and infestations
CMV viremia
|
4.0%
1/25 • Number of events 1 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Cardiac disorders
Heart failure
|
4.0%
1/25 • Number of events 2 • 1 year
|
0.00%
0/15 • 1 year
|
|
Cardiac disorders
hypotension
|
4.0%
1/25 • Number of events 1 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Lung infection
|
4.0%
1/25 • Number of events 1 • 1 year
|
20.0%
3/15 • Number of events 5 • 1 year
|
|
Gastrointestinal disorders
Oral mucositis
|
8.0%
2/25 • Number of events 3 • 1 year
|
13.3%
2/15 • Number of events 3 • 1 year
|
|
Cardiac disorders
pericardial effusion
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/15 • 1 year
|
|
Infections and infestations
sepsis
|
24.0%
6/25 • Number of events 10 • 1 year
|
46.7%
7/15 • Number of events 11 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin rash
|
4.0%
1/25 • Number of events 1 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
hemorrhagic cystitis
|
4.0%
1/25 • Number of events 1 • 1 year
|
6.7%
1/15 • Number of events 2 • 1 year
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
C diff infection
|
0.00%
0/25 • 1 year
|
20.0%
3/15 • Number of events 3 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
Blood and lymphatic system disorders
engraftment syndrome
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Nervous system disorders
Headache
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 2 • 1 year
|
|
Blood and lymphatic system disorders
HLH
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Hepatobiliary disorders
hyperbilirubinemiα
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 5 • 1 year
|
|
Endocrine disorders
hyperglycemia
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
hypogammaglobulinemia
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
hypokalemia
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
ischemic colitis and perforation
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
cellulitis
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
PTLD
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Eye disorders
retinal detachment
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
volume overload
|
0.00%
0/25 • 1 year
|
6.7%
1/15 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Cyclophosphamide and Abatacept
n=25 participants at risk
The GVHD prophylaxis regimen on the experimental arm will consist of high dose Cyclophosphamide on Days +3 and +4 followed by abatacept for 6 months. Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
Cyclophosphamide: Day 3 and day 4 following transplant. Dosing will be based on patients' actual weight up to 120% of ideal body weight, above which it will be based on adjusted ideal body weight (ideal weight plus 50% of the difference between ideal and actual weight).
abatacept: Abatacept at a dose of 10mg/kg will be administered on days +5, +14 and +28, +56, +84, +112, +140, +168
|
Methotrexate and Tacrolimus
n=15 participants at risk
The GVHD prophylaxis regimen on the standard of care arm will consist of methotrexate on Days +1,+3, +6 and +11 and tacrolimus
Methotrexate: standard of care Methotrexate 5 mg/m2 on Days 1, 3, 6 and 11
Tacrolimus: Tacrolimus per institutional guidelines
|
|---|---|---|
|
Infections and infestations
CMV Viremia
|
20.0%
5/25 • Number of events 5 • 1 year
|
26.7%
4/15 • Number of events 6 • 1 year
|
|
Gastrointestinal disorders
oral mucositis
|
12.0%
3/25 • Number of events 8 • 1 year
|
60.0%
9/15 • Number of events 13 • 1 year
|
|
Eye disorders
dry eyes
|
8.0%
2/25 • Number of events 2 • 1 year
|
0.00%
0/15 • 1 year
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/25 • 1 year
|
40.0%
6/15 • Number of events 9 • 1 year
|
|
Infections and infestations
BK viremia
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
colitis
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
General disorders
fatigue
|
0.00%
0/25 • 1 year
|
33.3%
5/15 • Number of events 5 • 1 year
|
|
Infections and infestations
fever
|
0.00%
0/25 • 1 year
|
20.0%
3/15 • Number of events 3 • 1 year
|
|
Immune system disorders
EBV viremia
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
Nervous system disorders
headache
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 3 • 1 year
|
|
Gastrointestinal disorders
nausea
|
0.00%
0/25 • 1 year
|
26.7%
4/15 • Number of events 4 • 1 year
|
|
Blood and lymphatic system disorders
TTP
|
0.00%
0/25 • 1 year
|
13.3%
2/15 • Number of events 2 • 1 year
|
Additional Information
Dr. Dimitrios Tzachanis
University of California, San Diego
Phone: (858) 822-6600
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place