Trial Outcomes & Findings for A Safety and Efficacy Study of Mycophenolate Mofetil and Rilonacept in Patients With Alcoholic Hepatitis (NCT NCT01903798)
NCT ID: NCT01903798
Last Updated: 2025-06-12
Results Overview
To determine whether treatment with prednisolone + mycophenolate mofetil is better than standard of care treatment among patients with alcoholic hepatitis who fail to respond to 1 week of prednisolone (i.e., Lille score of ≥0.45). Primary outcome is survival at Day 29. All study participants received the Standard of care (prednisolone) with or without experimental drug at Day 1 (based on randomization). Response to the treatment was determined at Day 8. Data was collected for both responders and non-responders.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
Day 8 to Day 29
Results posted on
2025-06-12
Participant Flow
4 participants were randomized. 3 in the standard of care (with Prednisolone) and 1 in mycophenolate + prednisolone arm. Out of 3 randomized to the SOC arm, 2 had a Lille score of \<.45 and one had a Lille score of \>.45. The participant who was randomized to mycophenolate + prednisolone had a Lille score of \>.45.
Participant milestones
| Measure |
Continue Prednisolone (Lille <0.45)
Continue prednisolone 40 mg/day (current standard of care) for 21 days.
Prednisolone: Corticosteroid
|
Rilonacept + Prednisolone (Lille <0.45)
Prednisolone (40mg/day) and rilonacept (Arcalyst®) subcutaneously once a week for 21 days (320 mg on study Day 8 and 160 mg on study Day 15 and study Day 22).
Rilonacept: Interleukin-1 blocker
Prednisolone: Corticosteroid
|
Standard of Care (Lille ≥ 0.45)
Standard of care therapy (continue prednisolone, stop all therapy and/or offer palliative care)
|
Mycophenolate + Prednisolone (Lille ≥ 0.45)
Prednisolone (40 mg/day) and mycophenolate mofetil for 21 days (500 mg BID for first 4 days followed by 1000 mg BID for the next 17 days).
Mycophenolate mofetil: Immunosuppressive agent
Prednisolone: Corticosteroid
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
2
|
0
|
1
|
1
|
|
Overall Study
COMPLETED
|
2
|
0
|
1
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Safety and Efficacy Study of Mycophenolate Mofetil and Rilonacept in Patients With Alcoholic Hepatitis
Baseline characteristics by cohort
| Measure |
Continue Prednisolone (Lille <0.45)
n=2 Participants
Continue prednisolone 40 mg/day (current standard of care) for 21 days.
Prednisolone: Corticosteroid
|
Rilonacept + Prednisolone (Lille <0.45)
Prednisolone (40mg/day) and rilonacept (Arcalyst®) subcutaneously once a week for 21 days (320 mg on study Day 8 and 160 mg on study Day 15 and study Day 22).
Rilonacept: Interleukin-1 blocker
Prednisolone: Corticosteroid
|
Standard of Care (Lille ≥ 0.45)
n=1 Participants
Standard of care therapy (continue prednisolone, stop all therapy and/or offer palliative care)
|
Mycophenolate + Prednisolone (Lille ≥ 0.45)
n=1 Participants
Prednisolone (40 mg/day) and mycophenolate mofetil for 21 days (500 mg BID for first 4 days followed by 1000 mg BID for the next 17 days).
Mycophenolate mofetil: Immunosuppressive agent
Prednisolone: Corticosteroid
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Age, Continuous
|
45.6 years
STANDARD_DEVIATION 9.7 • n=5 Participants
|
—
|
71.2 years
STANDARD_DEVIATION 0 • n=5 Participants
|
58 years
STANDARD_DEVIATION 0 • n=4 Participants
|
52 years
STANDARD_DEVIATION 13.6 • n=21 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
—
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
4 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Day 8 to Day 29Population: There was no participant randomized to rilonocept +prednisolone arm
To determine whether treatment with prednisolone + mycophenolate mofetil is better than standard of care treatment among patients with alcoholic hepatitis who fail to respond to 1 week of prednisolone (i.e., Lille score of ≥0.45). Primary outcome is survival at Day 29. All study participants received the Standard of care (prednisolone) with or without experimental drug at Day 1 (based on randomization). Response to the treatment was determined at Day 8. Data was collected for both responders and non-responders.
Outcome measures
| Measure |
Continue Prednisolone (Lille <0.45)
n=2 Participants
Continue prednisolone 40 mg/day (current standard of care) for 21 days.
Prednisolone: Corticosteroid
|
Rilonacept + Prednisolone (Lille <0.45)
Prednisolone (40mg/day) and rilonacept (Arcalyst®) subcutaneously once a week for 21 days (320 mg on study Day 8 and 160 mg on study Day 15 and study Day 22).
Rilonacept: Interleukin-1 blocker
Prednisolone: Corticosteroid
|
Standard of Care (Lille ≥ 0.45)
n=1 Participants
Standard of care therapy (continue prednisolone, stop all therapy and/or offer palliative care)
|
Mycophenolate + Prednisolone (Lille ≥ 0.45)
n=1 Participants
Prednisolone (40 mg/day) and mycophenolate mofetil for 21 days (500 mg BID for first 4 days followed by 1000 mg BID for the next 17 days).
Mycophenolate mofetil: Immunosuppressive agent
Prednisolone: Corticosteroid
|
|---|---|---|---|---|
|
Survival at Day 29 of the Assigned Treatment
|
2 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Week 24Population: No patient was randomized to Rilonacept +prednisolone arm
Outcome measures
| Measure |
Continue Prednisolone (Lille <0.45)
n=2 Participants
Continue prednisolone 40 mg/day (current standard of care) for 21 days.
Prednisolone: Corticosteroid
|
Rilonacept + Prednisolone (Lille <0.45)
Prednisolone (40mg/day) and rilonacept (Arcalyst®) subcutaneously once a week for 21 days (320 mg on study Day 8 and 160 mg on study Day 15 and study Day 22).
Rilonacept: Interleukin-1 blocker
Prednisolone: Corticosteroid
|
Standard of Care (Lille ≥ 0.45)
n=1 Participants
Standard of care therapy (continue prednisolone, stop all therapy and/or offer palliative care)
|
Mycophenolate + Prednisolone (Lille ≥ 0.45)
n=1 Participants
Prednisolone (40 mg/day) and mycophenolate mofetil for 21 days (500 mg BID for first 4 days followed by 1000 mg BID for the next 17 days).
Mycophenolate mofetil: Immunosuppressive agent
Prednisolone: Corticosteroid
|
|---|---|---|---|---|
|
Number of Patients Reported Ascites
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
Adverse Events
Continue Prednisolone (Lille <0.45)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Rilonacept + Prednisolone (Lille <0.45)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Standard of Care (Lille ≥ 0.45)
Serious events: 1 serious events
Other events: 1 other events
Deaths: 1 deaths
Mycophenolate + Prednisolone (Lille ≥ 0.45)
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Continue Prednisolone (Lille <0.45)
n=2 participants at risk
Continue prednisolone 40 mg/day (current standard of care) for 21 days.
Prednisolone: Corticosteroid
|
Rilonacept + Prednisolone (Lille <0.45)
Prednisolone (40mg/day) and rilonacept (Arcalyst®) subcutaneously once a week for 21 days (320 mg on study Day 8 and 160 mg on study Day 15 and study Day 22).
Rilonacept: Interleukin-1 blocker
Prednisolone: Corticosteroid
|
Standard of Care (Lille ≥ 0.45)
n=1 participants at risk
Standard of care therapy (continue prednisolone, stop all therapy and/or offer palliative care)
|
Mycophenolate + Prednisolone (Lille ≥ 0.45)
n=1 participants at risk
Prednisolone (40 mg/day) and mycophenolate mofetil for 21 days (500 mg BID for first 4 days followed by 1000 mg BID for the next 17 days).
Mycophenolate mofetil: Immunosuppressive agent
Prednisolone: Corticosteroid
|
|---|---|---|---|---|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/2
|
—
0/0
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
|
Nervous system disorders
Hepatic Encephalopathy
|
0.00%
0/2
|
—
0/0
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
Other adverse events
| Measure |
Continue Prednisolone (Lille <0.45)
n=2 participants at risk
Continue prednisolone 40 mg/day (current standard of care) for 21 days.
Prednisolone: Corticosteroid
|
Rilonacept + Prednisolone (Lille <0.45)
Prednisolone (40mg/day) and rilonacept (Arcalyst®) subcutaneously once a week for 21 days (320 mg on study Day 8 and 160 mg on study Day 15 and study Day 22).
Rilonacept: Interleukin-1 blocker
Prednisolone: Corticosteroid
|
Standard of Care (Lille ≥ 0.45)
n=1 participants at risk
Standard of care therapy (continue prednisolone, stop all therapy and/or offer palliative care)
|
Mycophenolate + Prednisolone (Lille ≥ 0.45)
n=1 participants at risk
Prednisolone (40 mg/day) and mycophenolate mofetil for 21 days (500 mg BID for first 4 days followed by 1000 mg BID for the next 17 days).
Mycophenolate mofetil: Immunosuppressive agent
Prednisolone: Corticosteroid
|
|---|---|---|---|---|
|
Gastrointestinal disorders
ascites
|
0.00%
0/2
|
—
0/0
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
|
Gastrointestinal disorders
Rectal Bleeding
|
0.00%
0/2
|
—
0/0
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
Additional Information
Executive Director, Southern California Institute for Research and Education
SCIRE
Phone: 562-826-8000
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER