Status:
COMPLETED
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
Lead Sponsor:
Amgen
Collaborating Sponsors:
Ucyclyd Pharma, Inc.
Conditions:
Hepatic Encephalopathy
Urea Cycle Disorders
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief 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 cir...
Detailed Description
Study acquired from Horizon in 2024.
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2007
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT00986895
Start Date
September 1 2006
End Date
June 1 2007
Last Update
June 20 2024
Active Locations (2)
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1
National University of Pharmacy
Kharkiv, Ukraine, 61057
2
Department of General Surgery #2; Kharkiv State Medical University
Kharkiv, Ukraine, 61128