Efficacy and Safety of AXA1665 in Cirrhotic Subjects With Prior Overt Hepatic Encephalopathy

NCT04816916 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: AXA1665-101
• Study Type: interventional
• Target: 12
• Locations: 7 countries
• Sites: 40

Brief Summary

This is a global study to compare the effects of AXA1665, an orally active mixture of amino acids, compared to placebo, on cognitive and physical function, as well as the safety and tolerability of AXA1665.

Conditions

Hepatic Encephalopathy

Keywords

liver cirrhosis; end stage liver disease; sarcopenia; amino acids

Outcome Measures

Primary Outcomes (1)

• Change in neurocognitive function by Psychometric Hepatic Encephalopathy Score (PHES)

  Baseline to week 24

Secondary Outcomes (3)

• Time to and frequency of recurrent overt hepatic encephalopathy

  Baseline to week 24

• Impact on physical function assessed by Liver Frailty Index (a composite measure of hand grip strength, balance and ability to stand up from a chair)

  Baseline to week 24

• Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)

  Baseline to week 24

Study Arms (2)

AXA1665 53.8 g per day

EXPERIMENTAL

AXA1665 administered orally TID

Drug: AXA1665

Matching placebo

PLACEBO COMPARATOR

Placebo administered orally TID

Drug: Placebo

Interventions

AXA1665 DRUG

AXA1665 administered TID with food

AXA1665 53.8 g per day

Placebo DRUG

Matching placebo administered TID with food

Matching placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Willing to participate in the study and provide written informed consent.
• Male and female adults aged ≥18 years.
• History of cirrhosis and at least 1 prior episode of overt hepatic encephalopathy (OHE) prior to Screening.
• A PHES of ≤ -4 during Screening.
• MELD score of ≤ 22 at Screening.
• Support of a primary caregiver who is able and willing to give written informed consent.

You may not qualify if:

• Hospitalization or serious medical condition.
• Presence of Child's-Pugh score ≥12, hepato-renal syndrome(s), refractory ascites, or spontaneous bacterial peritonitis (SBP).
• History of a surgical portosystemic or a transjugular intrahepatic portosystemic shunt (TIPS) placement.
• Expectation of a liver transplant during the study.
• Use of marijuana or tetrahydrocannabinol (THC) within 1 week prior to Screening and during the study.

Sponsors & Collaborators

• Axcella Health, Inc: lead

Study Sites (40)

University of California, San Francisco (UCSF)

San Francisco, California, 94143, United States

Location

OMEGA Research Consultants

DeBary, Florida, 32713, United States

Location

UF Hepatology Research at CTRB

Gainesville, Florida, 32610, United States

Location

Homestead Associates in Research

Miami, Florida, 33032, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Mercy Medical Center

Baltimore, Maryland, 21202, United States

Location

New York University (NYU) School of Medicine

New York, New York, 10016, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Cumberland Research Associates

Fayetteville, North Carolina, 28304, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States

Location

Optimed Research

Edinburg, Texas, 78359, United States

Location

Baylor Scott & White Research Institute

Fort Worth, Texas, 75246, United States

Location

Advanced Liver Therapies at St. Luke's Episcopal Hospital

Houston, Texas, 77030, United States

Location

Pinnacle Clinical Research

San Antonio, Texas, 78229, United States

Location

University of Virginia School of Medicine

Charlottesville, Virginia, 22903, United States

Location

Bon Secours Liver Institute

Newport News, Virginia, 23602, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23114, United States

Location

McGuire Research Institute, Inc., McGuire VA Medical Center

Richmond, Virginia, 23249, United States

Location

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

Location

LMC Diabetes & Endocrinology Ltd. - London

London, Ontario, N6A 2C2, Canada

Location

The Ottawa Hospital

Ottawa, Canada

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, Hungary

Location

Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz

Székesfehérvár, Hungary

Location

Azienda Ospedaliera Universitaria Federico II

Naples, 80131, Italy

Location

Azienda sanitaria universitaria Friuli Centrale (ASU FC)

Udine, 33100, Italy

Location

KO - MED Centra Kliniczne Lublin II

Lublin, Poland

Location

ID Clinic

Mysłowice, Poland

Location

SONOMED Sp. z o.o.

Szczecin, Poland

Location

Prywatna Specjalistyczna Praktyka Lekarska Jan Gietka

Warsaw, Poland

Location

Centrum Badan Klinicznych Piotr Napora Lekarze Sp. p.

Wroclaw, Poland

Location

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

Barcelona, Spain

Location

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Location

Hospital Universitario Puerta de Hierro

Majadahonda, 28222, Spain

Location

Hospital Universitario Marques de Valdecilla

Santander, Spain

Location

Hospital Clinico Universitario de Santiago

Santiago de Compostela, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, Spain

Location

The Royal Infirmary of Edinburgh

Edinburgh, United Kingdom

Location

Related Links

• EMMPOWER Phase 2 Clinical Trial Homepage

MeSH Terms

Conditions

Hepatic Encephalopathy; Liver Cirrhosis; End Stage Liver Disease; Sarcopenia

Condition Hierarchy (Ancestors)

Liver Failure; Hepatic Insufficiency; Liver Diseases; Digestive System Diseases; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Muscular Atrophy; Neuromuscular Manifestations; Neurologic Manifestations; Atrophy; Pathological Conditions, Anatomical; Signs and Symptoms

Study Officials

• Andres Duarte-Rojo, M.D.

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2021
• First Posted: March 25, 2021
• Study Start: June 29, 2021
• Primary Completion: June 30, 2022
• Study Completion: June 30, 2022
• Last Updated: July 27, 2022

Record last verified: 2022-07

Locations

ES (6); CA (3); IT (2); US (21); HU (2); PL (5); GB (1)