a study of glyceryl tri-(4-phenylbutyrate) administered orally as a single dose, and twice daily for seven consecutive days to subjects with hepatic impairment with cirrhosis and to a control group

Conditions: Hepatic Encephalopathy; Urea Cycle Disorders

Summary: the purpose of this study is to determine the safety and tolerability of gt4p administered orally as a single dose, and twice daily for 7 consecutive days, to subjects with hepatic impairment with cirrhosis (child-pugh scores of a, b, or c) and to a gender matched and similar age control group with normal hepatic function.

study acquired from horizon in 2024.

Eligibility criteria:
inclusion criteria: subjects were required to fulfill the following criteria in order to participate in the study: screening: * males or females aged ≥ 18 years of age * able to provide written informed consent before any study-related procedures, and ability, in the opinion of the investigator, to comply with all the requirements of the study * classification to one of the following: * current diagnosis of hepatic impairment with cirrhosis * healthy subject * subjects with hepatic impairment with cirrhosis were classifiable to one of the following groups: * child-pugh score a * child-pugh score b * child-pugh score c * subjects with hepatic impairment with cirrhosis who were on a therapeutic regimen of lactulose must have been on a stable dose for ≥ 30 days prior to screening * if female, a negative pregnancy test at screening and pre-dose on day 0, or a documented sterilization procedure; a female of child-bearing potential must have been using a medically approved birth control method and must have agreed to use the same method of contraception during the full course of the study (on pre-dose day 0 as well as at screening) * weight within the range of 60-100 kg (at screening and pre-dose on day 0) * willing to stop taking any medication that the sponsor and the investigator felt was not appropriate for use during the study, beginning 2 days before dosing and throughout the study exclusion criteria: subjects who fulfilled any of the following criteria were excluded from the study: screening: * clinically significant history or evidence of cardiovascular, respiratory, renal, gastrointestinal, endocrine, neurological, immunological, or psychiatric disorder(s), as determined by the investigator * serum sodium \< 120 meq/l * serum creatinine ≥ 1.5 upper limit of normal * potassium ≤ 3.5 meq/l * other laboratory values outside the normal range which were determined to be clinically significant by the investigator * significant illness within the last 14 days * oral temperature \> 38.5°c or \< 36°c and/or a suspected site of active infection * inflammatory bowel disease or malabsorption defined with steatorrhea * active gastrointestinal bleeding, defined as melena, hematochezia, or hematemesis requiring hospitalization within the last 30 days * use of probenecid, valproate, or corticosteroids within the last 24 hours * use of any medication, other than those approved by the sponsor and investigator, in the last 48 hours * history of seizures within the last 72 hours * positive drugs of abuse urine test * positive alcohol breath test * donation or loss of blood (500 ml or more) within the last 30 days * donation or loss of plasma within the last 7 days * history of acquired immunodeficiency syndrome (aids) or determined human immunodeficiency virus (hiv) positive * hepatitis b or c (hbv; hcv) positive (healthy volunteers only) * use of any investigational drug within the last 30 days * known hypersensitivity to sodium phenylbutyrate or similar drugs * emergency hospitalization within the last 90 days * intake of alcohol in the last 7 days pre-dose (days 0 and 7): * significant illness or emergency hospitalization since the last study visit * oral temperature \> 38.5°c or \< 36°c and/or a suspected site of active infection * use of probenecid, valproate, or corticosteroids within the last 24 hours * use of any non-approved medication (by the sponsor/investigator) within the 48 hours before dosing * history of seizures within the last 72 hours * positive drugs of abuse urine test * positive alcohol breath test * donation or loss of blood (500 ml or more) or plasma since the last study visit * use of any investigational drug since the last study visit * intake of alcohol in the last 7 days

Keywords: hpn-100 gt4p glyceryl tri-(4-phenylbutyrate)