Hepatic EncephalopathyPhase 2EMMPOWERNCT04816916
Efficacy and Safety of AXA1665 in Cirrhotic Subjects With Prior Overt Hepatic Encephalopathy
A Phase 2 study testing AXA1665 in people with Hepatic Encephalopathy, run by Axcella Health, Inc at 40 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatic Encephalopathy
- What is being tested?
- AXA1665
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 40 sitesin 7 countries
- Study dates
- 29 Jun 2021to 30 Jun 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
40 sites in 7 countries.
40 sites in 7 countries
United States21 sites
Mercy Medical Center
Baltimore, Maryland, 21202
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425
University of Virginia School of Medicine
Charlottesville, Virginia, 22903
OMEGA Research Consultants
DeBary, Florida, 32713
Optimed Research
Edinburg, Texas, 78359
Cumberland Research Associates
Fayetteville, North Carolina, 28304
Baylor Scott & White Research Institute
Fort Worth, Texas, 75246
UF Hepatology Research at CTRB
Gainesville, Florida, 32610
Advanced Liver Therapies at St. Luke's Episcopal Hospital
Houston, Texas, 77030
Indiana University School of Medicine
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
Homestead Associates in Research
Miami, Florida, 33032
University of Miami
Miami, Florida, 33136
New York University (NYU) School of Medicine
New York, New York, 10016
Weill Medical College of Cornell University
New York, New York, 10021
Bon Secours Liver Institute
Newport News, Virginia, 23602
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Virginia Commonwealth University
Richmond, Virginia, 23114
McGuire Research Institute, Inc., McGuire VA Medical Center
Richmond, Virginia, 23249
Pinnacle Clinical Research
San Antonio, Texas, 78229
University of California, San Francisco (UCSF)
San Francisco, California, 94143
Spain6 sites
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Barcelona
Hospital Universitario Vall d'Hebron
Barcelona
Hospital Universitario Puerta de Hierro
Majadahonda, 28222
Hospital Universitario Marques de Valdecilla
Santander
Hospital Clinico Universitario de Santiago
Santiago de Compostela
Hospital Universitario Virgen del Rocio
Seville
Poland5 sites
KO - MED Centra Kliniczne Lublin II
Lublin
ID Clinic
Mysłowice
SONOMED Sp. z o.o.
Szczecin
Prywatna Specjalistyczna Praktyka Lekarska Jan Gietka
Warsaw
Centrum Badan Klinicznych Piotr Napora Lekarze Sp. p.
Wroclaw
Canada3 sites
University of Calgary
Calgary, Alberta, T2N 4Z6
LMC Diabetes & Endocrinology Ltd. - London
London, Ontario, N6A 2C2
The Ottawa Hospital
Ottawa
Hungary2 sites
Debreceni Egyetem Klinikai Kozpont
Debrecen
Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz
Székesfehérvár
Italy2 sites
Azienda Ospedaliera Universitaria Federico II
Naples, 80131
Azienda sanitaria universitaria Friuli Centrale (ASU FC)
Udine, 33100
About this study
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)
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
- Being tested
- AXA1665, Placebo
- Keywords
- liver cirrhosis, end stage liver disease, sarcopenia, amino acids
- Medical subject headings
- Hepatic Encephalopathy, Liver Cirrhosis, End Stage Liver Disease, Sarcopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AXA1665Drug
AXA1665 administered TID with food
PlaceboDrug
Matching placebo administered TID with food
| Study group | Role | What they receive |
|---|---|---|
| AXA1665 53.8 g per day | Experimental | AXA1665 administered orally TID Receives: Drug: AXA1665 |
| Matching placebo | Placebo comparator | Placebo administered orally TID Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in neurocognitive function by Psychometric Hepatic Encephalopathy Score (PHES)
Measured: Baseline to week 24
Secondary outcomes (3)
Time to and frequency of recurrent overt hepatic encephalopathy
Measured: 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)
Measured: Baseline to week 24
Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)
Measured: Baseline to week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2021
- Study start29 Jun 2021
- Primary completion30 Jun 2022
- Study completion30 Jun 2022
Why it stopped: Sponsor is stopping the study to focus company resources on other development programs.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatic EncephalopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Axcella Health, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andres Duarte-Rojo, M.D., Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04816916
- Sponsor's protocol ID
- AXA1665-101
- Acronym
- EMMPOWER
- Status last verified
- Jul 2022
- First submitted
- 19 Mar 2021
- Last updated
- 27 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04816916
- Acronym / sponsor ID
- EMMPOWER / AXA1665-101
- Conditions
- Hepatic Encephalopathy
- Keywords
- liver cirrhosis, end stage liver disease, sarcopenia, amino acids
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor is stopping the study to focus company resources on other development programs.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Axcella Health, IncIndustry (pharma, biotech, devices)
- Study start
- 29 Jun 2021 (actual)
- Primary completion
- 30 Jun 2022 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 25 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AXA1665 53.8 g per day | Experimental | Drug: AXA1665AXA1665 administered orally TID |
| Matching placebo | Placebo comparator | Drug: PlaceboPlacebo administered orally TID |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AXA1665AXA1665 administered TID with food | Drug | AXA1665 53.8 g per day |
| PlaceboMatching placebo administered TID with food | Drug | Matching placebo |
Primary outcome
Change in neurocognitive function by Psychometric Hepatic Encephalopathy Score (PHES)
Time frame: Baseline to week 24
3 secondary outcomes
Time to and frequency of recurrent overt hepatic encephalopathy
Time frame: 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)
Time frame: Baseline to week 24
Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)
Time frame: Baseline to week 24
Results and safety
The study was stopped early (reason given: Sponsor is stopping the study to focus company resources on other development programs.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2022.
- EMMPOWER Phase 2 Clinical Trial HomepageLink provided by the sponsor.
Related trials for Hepatic Encephalopathy
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Equivalence of Electronic vs Paper-based PHES in Diagnosing Hepatic Encephalopathy
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andres Duarte-Rojo, M.D. principal investigator
University of Pittsburgh
40 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AXA1665 in people with Hepatic Encephalopathy, run by Axcella Health, Inc at 40 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 27 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, Spain, Poland, Canada, Hungary and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor is stopping the study to focus company resources on other development programs.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in neurocognitive function by Psychometric Hepatic Encephalopathy Score (PHES) (time frame: Baseline to week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2022.