Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04880187




Registration number
NCT04880187
Ethics application status
Date submitted
3/05/2021
Date registered
10/05/2021
Date last updated
8/09/2022

Titles & IDs
Public title
Safety, Tolerability, and Efficacy of AXA1125 in NASH With Fibrosis
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Dose Ranging Study to Evaluate the Safety, Tolerability, and Efficacy of AXA1125 in Subjects With Non Cirrhotic, Non Alcoholic Steatohepatitis and Fibrosis (EMMPACT)
Secondary ID [1] 0 0
AXA1125-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Non Alcoholic Steatohepatitis (NASH) 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Diet and Nutrition 0 0 0 0
Obesity
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AXA1125
Treatment: Drugs - Placebo

Experimental: AXA1125 22.6g - 22.6 g AXA1125 administered orally BID with or without food

Experimental: AXA1125 33.9g - 33.9 g AXA1125 administered orally BID with or without food

Placebo Comparator: Placebo - Matching Placebo administered orally BID with or without food


Treatment: Drugs: AXA1125
AXA1125 administered BID with or without food

Treatment: Drugs: Placebo
Matching Placebo administered BID with or without food
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Improvement in steatohepatitis
Timepoint [1] 0 0
Baseline to Week 48
Secondary outcome [1] 0 0
Resolution of NASH without worsening of fibrosis
Timepoint [1] 0 0
Baseline to week 48
Secondary outcome [2] 0 0
Improvement of fibrosis by one stage without worsening of NASH
Timepoint [2] 0 0
Baseline to week 48
Secondary outcome [3] 0 0
Incidence of study drug emergent adverse events (AEs) and serious adverse events (SAEs)
Timepoint [3] 0 0
Baseline to week 48
Secondary outcome [4] 0 0
Change from baseline in liver stiffness as measured by vibration controlled transient elastography (Fibroscan™)
Timepoint [4] 0 0
Baseline to week 48
Secondary outcome [5] 0 0
Change from baseline in hepatic fat as measured by MRI
Timepoint [5] 0 0
Baseline to week 48
Secondary outcome [6] 0 0
Change from baseline in measures of glucose control as determined by glycated hemoglobin (HbA1c)
Timepoint [6] 0 0
Baseline to week 48

Eligibility
Key inclusion criteria
- Willing to participate in the study and provide written informed consent.

- Male and female adults aged > 18 years.

- Must have NASH and fibrosis on a liver biopsy sample

- If a historical liver biopsy is used for Screening, obtained within 6 months prior to
Screening;

- Subjects may have a diagnosis of T2DM
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- History or presence of liver disease (other than NAFLD or NASH)

- History or presence of cirrhosis and/or history or presence of hepatic decompensation

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Unknown status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/05/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2023
Actual
Sample size
Target
273
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Brisbane
Recruitment hospital [3] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [4] 0 0
Concord Repatriation General Hospital - Concord
Recruitment hospital [5] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
- Bedford Park
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Clayton
Recruitment postcode(s) [4] 0 0
- Concord
Recruitment postcode(s) [5] 0 0
- Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Mississippi
Country [8] 0 0
United States of America
State/province [8] 0 0
Nevada
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
South Dakota
Country [13] 0 0
United States of America
State/province [13] 0 0
Tennessee
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
Canada
State/province [17] 0 0
Alberta
Country [18] 0 0
Canada
State/province [18] 0 0
Ontario
Country [19] 0 0
Canada
State/province [19] 0 0
Halifax
Country [20] 0 0
France
State/province [20] 0 0
La Tronche
Country [21] 0 0
France
State/province [21] 0 0
Lyon
Country [22] 0 0
France
State/province [22] 0 0
Montpellier
Country [23] 0 0
France
State/province [23] 0 0
Paris
Country [24] 0 0
France
State/province [24] 0 0
Pessac
Country [25] 0 0
France
State/province [25] 0 0
Villejuif
Country [26] 0 0
Poland
State/province [26] 0 0
CET
Country [27] 0 0
Puerto Rico
State/province [27] 0 0
San Juan
Country [28] 0 0
United Kingdom
State/province [28] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Axcella Health, Inc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will compare the effects of AXA1125, an orally active mixture of amino acids,
compared to placebo, on improving fat and inflammation (steatohepatitis) as well as fibrosis
in subjects with non alcoholic steatohepatitis (NASH). as well as the safety and tolerability
of AXA1125. Subjects will take one of two different doses of AXA1125 or a placebo twice
daily, and a liver biopsy will be done at the beginning and end of the 48-week study.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04880187
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Stephen Harrison, MD
Address 0 0
Pinnacle Clinical Research
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Margaret Koziel, MD
Address 0 0
Country 0 0
Phone 0 0
(857) 320-3200
Fax 0 0
Email 0 0
clinicaltrials@axcellahealth.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04880187