Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE)

Study Overview
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Brief Summary
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The purpose of this study is to determine whether ornithine phenylacetate can speed recovery from an acute hepatic encephalopathy episode requiring hospitalization in cirrhotic patients.

Detailed Description
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The primary objectives of the study were to evaluate the efficacy of OCR-002 for treatment of an acute hepatic encephalopathy (HE) episode in cirrhotic patients requiring hospitalization and the safety and tolerability of OCR-002 in hospitalized cirrhotic patients with an acute episode of HE.

Official Title
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Multicenter, Randomized Phase 2B Study to Evaluate the Efficacy, Safety and Tolerability of OCR-002 (Ornithine Phenylacetate) in Hospitalized Patients With Cirrhosis and Associated Hyperammonemia With an Episode of Hepatic Encephalopathy (STOP-HE Study)

Conditions
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Acute Episode of Overt Hepatic Encephalopathy, Hepatic Encephalopathy

Intervention / Treatment
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* Drug: ornithine phenylacetate
* Drug: placebo intravenous infusion


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Hospitalized with an acute episode of hepatic encephalopathy as complication of cirrhosis Elevated venous ammonia Exclusion Criteria: Renal failure with serum creatinine > 3 mg/dL or need for dialysis Molecular Adsorbent Recirculation System utilized Pregnancy or breastfeeding

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Ornithine phenylacetate<br>Participants receive ornithine phenylacetate for up to 5 days via continuous IV infusion in addition to standard of care (SOC) | Drug: ornithine phenylacetate<br>* Ornithine phenylacetate for continuous IV infusion at dose levels predicated on level of hepatic decompensation<br>* Other names: OCR-002;|
| Placebo Comparator: Placebo<br>Participants receive matching placebo up to 5 days via continuous IV infusion in addition to SOC | Drug: placebo intravenous infusion<br>* Placebo for continuous IV infusion that is visually identical to the experimental product<br>* Other names: Matching Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants in Each HE Stage | To support the primary endpoint of confirmed clinical response, the investigator rated participants on a 4-point staging scale, where higher scores are worse. The stages are described as: Stage 0/1 = Participant has no tremor of the hand when the wrist is extended (asterixis) and no confusion, even about where they are (disorientation) Stage 2 = Participant has tremor of the hand when the wrist is extended (asterixis) and is confused, not knowing where they are (disorientation) Stage 3 = Participant is tired, falling asleep, answers questions but is confused, and doesn't know where they are (Stupor, arousable but falls asleep, responsive to verbal stimuli, Obvious confusion, Gross disorientation) Stage 4 = Participant is unconscious (in a coma) | Baseline to End of Study (through 3 hours post end-of-infusion) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hepatic encephalopathy, hyperammonemia, cirrhosis, ornithine phenylacetate, OCR-002