Clinical trial NCT04816916: Efficacy and Safety of AXA1665 in Cirrhotic Subjects With Prior Overt Hepatic Encephalopathy
Official title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of AXA1665 in Subjects With Liver Cirrhosis and Prior Overt Hepatic Encephalopathy (EMMPOWER)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Hepatic Encephalopathy
Keywords: liver cirrhosis
end stage liver disease
sarcopenia
amino acids
Lead sponsor: Axcella Health, Inc
Sponsor class: INDUSTRY
First posted: 2021-03-25
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Change in neurocognitive function by Psychometric Hepatic Encephalopathy Score (PHES)
timeFrame: Baseline to week 24
Secondary outcomes:
measure: Time to and frequency of recurrent overt hepatic encephalopathy
timeFrame: Baseline to week 24
measure: 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)
timeFrame: Baseline to week 24
measure: Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)
timeFrame: Baseline to week 24
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California, San Francisco (UCSF)
state: California
city: DeBary
country: United States
facility: OMEGA Research Consultants
state: Florida
city: Gainesville
country: United States
facility: UF Hepatology Research at CTRB
state: Florida
city: Miami
country: United States
facility: Homestead Associates in Research
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Baltimore
country: United States
facility: Mercy Medical Center
state: Maryland
city: New York
country: United States
facility: New York University (NYU) School of Medicine
state: New York
city: New York
country: United States
facility: Weill Medical College of Cornell University
state: New York
city: Fayetteville
country: United States
facility: Cumberland Research Associates
state: North Carolina
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina (MUSC)
state: South Carolina
city: Edinburg
country: United States
facility: Optimed Research
state: Texas
city: Fort Worth
country: United States
facility: Baylor Scott & White Research Institute
state: Texas
city: Houston
country: United States
facility: Advanced Liver Therapies at St. Luke's Episcopal Hospital
state: Texas
city: San Antonio
country: United States
facility: Pinnacle Clinical Research
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia School of Medicine
state: Virginia
city: Newport News
country: United States
facility: Bon Secours Liver Institute
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Richmond
country: United States
facility: McGuire Research Institute, Inc., McGuire VA Medical Center
state: Virginia
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: London
country: Canada
facility: LMC Diabetes & Endocrinology Ltd. - London
state: Ontario
city: Ottawa
country: Canada
facility: The Ottawa Hospital
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Kozpont
city: Székesfehérvár
country: Hungary
facility: Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
city: Udine
country: Italy
facility: Azienda sanitaria universitaria Friuli Centrale (ASU FC)
city: Lublin
country: Poland
facility: KO - MED Centra Kliniczne Lublin II
city: Mysłowice
country: Poland
facility: ID Clinic
city: Szczecin
country: Poland
facility: SONOMED Sp. z o.o.
city: Warszawa
country: Poland
facility: Prywatna Specjalistyczna Praktyka Lekarska Jan Gietka
city: Wrocław
country: Poland
facility: Centrum Badan Klinicznych Piotr Napora Lekarze Sp. p.
city: Barcelona
country: Spain
facility: Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta de Hierro
city: Santander
country: Spain
facility: Hospital Universitario Marques de Valdecilla
city: Santiago De Compostela
country: Spain
facility: Hospital Clinico Universitario de Santiago
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Edinburgh
country: United Kingdom
facility: The Royal Infirmary of Edinburgh
Study officials:
affiliation: University of Pittsburgh
name: Andres Duarte-Rojo, M.D.
role: PRINCIPAL_INVESTIGATOR
References: