Clinical trial NCT04147936: Study of the Safety and Tolerability of AXA1665 in Subjects With Mild and Moderate Hepatic Insufficiency
Official title: A 12-Week, Single-Blind, Placebo-Controlled, Randomized Study to Evaluate the Safety, Tolerability, and Physiological Regulation of an Amino Acid Food Product, AXA1665, in Subjects With Mild and Moderate Hepatic Insufficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Hepatic Insufficiency
Keywords: Amino acids, food study
Lead sponsor: Axcella Health, Inc
Sponsor class: INDUSTRY
First posted: 2019-11-01
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

Brief summary: This is a randomized, single blind study to determine whether AXA1665, a composition of naturally occuring amino acids, is well tolerated in subjects with mild and moderate hepatic insufficiency. Study will also examine how the food product may influence the biology in muscle which will be assessed using magnetic resonance imaging (MRI) and other functional assessments such as strength, balance and cognition as part of a comprehensive physical/neurological exam. Changes in blood biomarkers of inflammation will also be assessed.


Eligibility criteria: Inclusion Criteria:

* Willing to participate in the study and provide written informed consent
* Male and female adults aged \> 18 years
* Child-Pugh score ≤9 (i.e. Child-Pugh class A or B)
* Liver Frailty Index (LFI) of ≥3.6
* Willing and able to engage in 30 minutes of walking/physical activity at least 3 days per week

Exclusion Criteria:

* Hospitalization for any complication of cirrhosis or taking new medications intended to treat hepatic encephalopathy within 2 months prior to Screening or any hospitalization for any cause/reason within 30 days prior to Screening
* Prior history or presence of a transjugular intrahepatic portal systemic shunt (TIPS)
* Current or history of significant alcohol consumption
* Other poorly controlled medical condition \[e.g., renal disease with an estimated glomerular filtration rate (GFR) \<60 mL/min/1.73m2)
* Known sensitivity and/or history of clinically significant food intolerance/allergies to proteins (including whey, soy, casein, amino acids, etc.)
* Any extreme or unbalanced diet such as Ketogenic, Atkins, Paleo, Vegan, etc.
* Unable or unwilling to adhere to contraception requirements
* Any contraindications to a MRI scan
* Any other condition that, in the opinion of the Investigator, renders the subject at risk for compliance, compromises the well-being of the subject, or hinders study completion

Primary outcomes:
measure: Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)
timeFrame: Baseline to Week 12
Secondary outcomes:
measure: Change in muscle mass by MRI
timeFrame: Baseline to Week 12
measure: Change in Fischer's ratio [measured by ratio of branched-chain amino acids (leucine, valine, isoleucine) to aromatic amino acids (phenylalanine, tyrosine)]
timeFrame: Baseline to Week 12
measure: Change in plasma ammonia
timeFrame: Baseline to Week 12
measure: Change in blood urea nitrogen concentration
timeFrame: Baseline to Week 12
measure: Change in creatinine concentration
timeFrame: Baseline to Week 12
measure: Change in gait speed
timeFrame: Baseline to Week 12
measure: Change in Liver Frailty Index
timeFrame: Baseline to Week 12
measure: Change in overall physical activity (measured by actigraphy watch)
timeFrame: Baseline to Week 12
measure: Change in cognitive function measured by the Psychometric Hepatic Encephalopathy Score (PHES)
timeFrame: Baseline to Week 12
Other outcomes:
Locations:
city: Montclair
country: United States
facility: Catalina Research Institute, LLC
state: California
city: Tustin
country: United States
facility: Orange County Research Center
state: California
city: Miami Lakes
country: United States
facility: Panax Clinical Research
state: Florida
city: Orlando
country: United States
facility: OMEGA Research Maitland, LCC
state: Florida
city: Tampa
country: United States
facility: Avita Clinical Research
state: Florida
city: Atlanta
country: United States
facility: Atlanta Center for Medical Research
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University Health
state: Indiana
city: Bastrop
country: United States
facility: Delta Research Partners
state: Louisiana
city: Pittsburgh
country: United States
facility: UPMC Center for Liver Disease
state: Pennsylvania
city: San Antonio
country: United States
facility: Texas Liver Institute
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
Study officials:
affiliation: Virginia Commonwealth University
name: Arun J Sanyal, MD
role: PRINCIPAL_INVESTIGATOR
References: