Healthy
Conditions
Keywords
Ammonul, Sodium phenylacetate, sodium benzoate, Buphenyl, sodium phenylbutyrate, HPN-100, GT4P, Glyceryl tri-(4-phenylbutyrate), Healthy Volunteers
Brief summary
To determine the safety and tolerability of single oral doses of HPN-100 as a formulation (GT4P-F) and GT4P as the active pharmaceutical ingredient (GT4P-API) administered to healthy male subjects.
Detailed description
A randomized, open-label, four-treatment, four-period crossover study in which healthy male subjects received a single dose of each of the following four treatments on four separate dosing days, 7 days apart: * Oral sodium phenylbutyrate (Buphenyl®) equivalent to 3 g/m2 of 4-phenylbutyric acid (PBA) per dose * Oral GT4P-F mole equivalents to 3 g/m2 of PBA per dose * Oral GT4P-API mole equivalents to 3 g/m2 of PBA per dose * Intravenous 10% sodium phenylacetate plus 10% sodium benzoate (Ammonul®) 2.75 g/m2 Study acquired from Horizon in 2024.
Interventions
DRUGHPN-100
HPN-100 is a triglyceride that has a similar mechanism of action as Buphenyl. It is a liquid with minimal taste and odor. HPN-100 is broken down to phenylbutyric acid (PBA). PBA is converted to phenyl acetic acid (PAA) that is the active metabolite. Three teaspoons of HPN-100 (\ 17.4mL) delivers equivalent amount of PBA that 40 tablets of NaPBA do.
DRUGAmmonul
DRUGBuphenyl
Sponsors
Ucyclyd Pharma, Inc.
Amgen
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
Subjects were required to fulfill the following criteria in order to participate in the study: * Males aged 18 to 45 years of age * Ability 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 * Subjects who were in good health as determined by a medical history, physical examination, serum chemistry, hematology, urinalysis, 12 lead ECG, and vital signs * Weight within the range of 60-120 kg
Exclusion criteria
Subjects who fulfilled any of the following criteria were excluded from the study: * Clinically significant history or evidence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, neurologic, immunologic, or psychiatric disorder(s), as determined by the investigator * Clinically significant abnormal laboratory values (as determined by the investigator) * Significant illness within 14 days prior to screening * Any disorder that might significantly interfere with the absorption, distribution, metabolism, or excretion of any drug * Use of any prescription medication within 14 days prior to screening * Use of dietary supplements, herbal medicines, vitamins, or over-the-counter medication(s) (with the exception of acetaminophen ≤ 500 mg/day) within 10 days prior to first dosing * Positive drugs of abuse urine test at screening or pre-dose day (cocaine, amphetamines, barbiturates, opiates, benzodiazepines, cannabinoids, methadone) * Positive alcohol breath test at screening or pre-dose day * Donation or loss of blood (500 ml or more) within 30 days prior to first dosing, or during the study * Donation or loss of plasma within 7 days prior to first dosing, or during the study * History of or current hepatitis or carriers of hepatitis B surface antigen (HBsAg) and/or hepatitis C antibodies (anti-HC) * History of acquired immunodeficiency syndrome (AIDS) or determined HIV positive at screening * Use of any investigational drug within 12 weeks prior to first dosing * Known hypersensitivity to sodium phenylbutyrate or similar drugs * Previous exposure to sodium phenylbutyrate
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The rate of adverse events | 33 Days |
Countries
Ukraine
Outcome results
None listed