Trial Outcomes & Findings for Treatment of Type I Hepatorenal Syndrome (HRS) With Pentoxyfylline (NCT NCT02123576)

NCT ID: NCT02123576

Last Updated: 2019-05-29

Results Overview

We define this as a decrease in serum creatinine level to \<1.5 mg/dL without dialysis or death

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 12 participants
• Primary outcome timeframe: 14 days
• Results posted on: 2019-05-29

Participant Flow

Participant milestones

Measure	Treatment Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Overall Study STARTED	6	6
Overall Study COMPLETED	5	6
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	Treatment Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Overall Study Adverse Event	1	0

Baseline Characteristics

Treatment of Type I Hepatorenal Syndrome (HRS) With Pentoxyfylline

Baseline characteristics by cohort

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Total n=12 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
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	57 years n=5 Participants	60.8 years n=7 Participants	58.9 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants

PRIMARY outcome

Timeframe: 14 days

We define this as a decrease in serum creatinine level to <1.5 mg/dL without dialysis or death

Outcome measures

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Number of Participants With Treatment Success	1 Participants	1 Participants

SECONDARY outcome

Timeframe: baseline and 14 days

Outcome measures

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Change in Serum Creatinine From Baseline	0.48 g/dL Interval -0.49 to 1.46	0.03 g/dL Interval -0.64 to 0.7

SECONDARY outcome

Timeframe: up to day 14

Defined as creatinine level above baseline value after day 7, dialysis or death

Outcome measures

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Incidence of Treatment Failure	3 Participants	3 Participants

SECONDARY outcome

Timeframe: 14 days

We define as serum creatinine level decreased by >50% from baseline but not to <1.5 mg/dL, without dialysis or HRS recurrence

Outcome measures

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Number of Participants With Combined Outcome of Treatment Success and Partial Response	1 Participants	2 Participants

SECONDARY outcome

Timeframe: day 30 and 180

Outcome measures

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Transplant Free Survival	80 days Interval 2.0 to 163.0	36 days Interval 4.0 to 67.0

SECONDARY outcome

Timeframe: up to 1 year

This will be the combination of transplant free survival and those patients who received liver transplant

Outcome measures

Measure	Treatment n=6 Participants Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 Participants This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
Overall Survival	102 days Interval 12.0 to 192.0	59 days Interval 11.0 to 129.0

Adverse Events

Treatment

Serious events: 1 serious events

Other events: 0 other events

Deaths: 5 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 3 deaths

Serious adverse events

Measure	Treatment n=6 participants at risk Pentoxyfylline 400mg three times a day or 400mg twice a day for eGFR 10-50 and 400mg once a day for eGFR \<10 for 90 days in addition to standard AMO therapy Pentoxyfylline AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)	Placebo n=6 participants at risk This is a standard placebo pill. Placebo AMO Therapy: Albumin, midodrine and octreotide therapy (standard of care for HRS)
General disorders Discontinued intervention	16.7% 1/6 • Number of events 1 • adverse event data was collected for up to 14 days while subject was on placebo or AMO therapy.	0.00% 0/6 • adverse event data was collected for up to 14 days while subject was on placebo or AMO therapy.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Jonathan G. Stine

The Pennsylvania State University Division of Gastroenterology and Hepatology Milton S. Herhsey Medical Center

Phone: 717-531-0003

Email: jstine@pennstatehealth.psu.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place