Safety, Tolerability, and Efficacy of AXA1125 in NASH With Fibrosis

• Sponsor: Axcella Health, Inc (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04880187
• Collaborator: (none)
• Enrollment: 273
• Locations: 73
• Arms: 3
• Anticipated Duration (Months): 28.8
• Patients Per Site: 3.7
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Non Alcoholic Steatohepatitis (NASH)	Drug: AXA1125; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 273 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official 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)
• Actual Study Start Date: May 7, 2021
• Anticipated Primary Completion Date: Sep 1, 2023
• Anticipated Study Completion Date: Oct 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AXA1125 22.6g 22.6 g AXA1125 administered orally BID with or without food	Drug: AXA1125; AXA1125 administered BID with or without food
Experimental: AXA1125 33.9g 33.9 g AXA1125 administered orally BID with or without food	Drug: AXA1125; AXA1125 administered BID with or without food
Placebo Comparator: Placebo Matching Placebo administered orally BID with or without food	Drug: Placebo; Matching Placebo administered BID with or without food

Outcome Measures

Primary Outcome Measures

1. Improvement in steatohepatitis [Baseline to Week 48]

   2-point improvement from baseline to Week 48 in the non-alcoholic fatty liver disease (NAFLD) Activity Score (NAS) based on a scale from 0-8 with 0 being no NASH and 8 being the highest score

Secondary Outcome Measures

1. Resolution of NASH without worsening of fibrosis [Baseline to week 48]

   The proportion of subjects with resolution of NASH with no worsening of fibrosis defined as a post-treatment ballooning score of 0 and an inflammation score of 0 or 1 on liver biopsy.

2. Improvement of fibrosis by one stage without worsening of NASH [Baseline to week 48]

   The proportion of subjects who have at least a 1-stage improvement in fibrosis and no worsening of NASH based on liver biopsy 0 being no fibrosis and 4 being the worse.

3. Incidence of study drug emergent adverse events (AEs) and serious adverse events (SAEs) [Baseline to week 48]

4. Change from baseline in liver stiffness as measured by vibration controlled transient elastography (Fibroscan™) [Baseline to week 48]

5. Change from baseline in hepatic fat as measured by MRI [Baseline to week 48]

6. Change from baseline in measures of glucose control as determined by glycated hemoglobin (HbA1c) [Baseline to week 48]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cullman Clinical Trials	Cullman	Alabama	United States	35055
2	Adobe Clinical Research, LLC	Tucson	Arizona	United States	85712
3	Del Sol Research Management, LLC	Tucson	Arizona	United States	85715
4	San Fernando Valley Health Institute	Canoga Park	California	United States	91304
5	National Research Institute	Gardena	California	United States	90247
6	National Research Institute	Huntington Park	California	United States	90255
7	OM Research LLC	Lancaster	California	United States	93534
8	National Research Institute	Los Angeles	California	United States	90057
9	Inland Empire Liver Foundation	Rialto	California	United States	92377
10	Precision Research Institute, LLC	San Diego	California	United States	92114
11	Excel Medical Clinical Trials, LLC	Boca Raton	Florida	United States	33434
12	Synergy Healthcare	Bradenton	Florida	United States	34208
13	Tampa Bay Medical Research , Inc.	Clearwater	Florida	United States	33761
14	Evolution Clinical Trials, Inc.	Hialeah Gardens	Florida	United States	33016
15	ENCORE Borland Groover Clinical Research	Jacksonville	Florida	United States	32256
16	ClinCloud LLC	Maitland	Florida	United States	32751
17	Panax Clinical Research	Miami Lakes	Florida	United States	33014
18	Clinical Pharmacology of Miami, LLC	Miami	Florida	United States	33014-3616
19	La Salud Research	Miami	Florida	United States	33155
20	Genoma Research Group, Inc	Miami	Florida	United States	33173
21	Sensible Healthcare, LLC	Ocoee	Florida	United States	34761
22	Theia Clinical Research, LLC	Pinellas Park	Florida	United States	33709
23	Progressive Medical Research	Port Orange	Florida	United States	32124
24	ClinCloud, LLC	Viera	Florida	United States	32940
25	Metabolic Research Institute, Inc.	West Palm Beach	Florida	United States	33401
26	Conquest Research	Winter Park	Florida	United States	32789
27	Indiana University (IU) School of Medicine	Indianapolis	Indiana	United States	46202
28	Delta Research Partners	Bastrop	Louisiana	United States	71201
29	Tandem Clinical Research	Marrero	Louisiana	United States	70072
30	Tandem Clinical Research GI	Metairie	Louisiana	United States	70006
31	Louisiana Research Center, LLC	Shreveport	Louisiana	United States	71105
32	The National Diabetes & Obesity Research Institute	Biloxi	Mississippi	United States	39532
33	Jubilee Clinical Research, Inc.	Las Vegas	Nevada	United States	89106
34	Sierra Clinical Research	Las Vegas	Nevada	United States	89106
35	Icahn School of Medicine at Mount Sinai	New York	New York	United States	10029
36	Tandem Clinical Research GI	New York	New York	United States	10033
37	Lucas Research - Diabetes & Endocrinology Consultants, PC	Morehead City	North Carolina	United States	28557
38	Consultants for Clinical Research	Cincinnati	Ohio	United States	45219
39	Rapid City Medical Center	Rapid City	South Dakota	United States	57701
40	ClinSearch LLC	Chattanooga	Tennessee	United States	37421
41	Texas Clinical Research Institute	Arlington	Texas	United States	76012
42	American Research Corporation	Austin	Texas	United States	78757
43	Pinnacle Clinical Research	Austin	Texas	United States	78757
44	Pioneer Research Solutions Inc.	Houston	Texas	United States	77099
45	R & H Clinical Research	Katy	Texas	United States	77494
46	EPIC Medical Research	Red Oak	Texas	United States	75154
47	American Research Corporation	San Antonio	Texas	United States	78215
48	Endeavor Clinical Trials	San Antonio	Texas	United States	78229
49	Pinnacle Clinical Research	San Antonio	Texas	United States	78229
50	R & H Clinical Research	Stafford	Texas	United States	77477
51	Simcare Medical Research, LLC	Sugar Land	Texas	United States	77478
52	Impact Clinical Research	Waco	Texas	United States	76710
53	Velocity - Advanced Clinical Research - Salt Lake City of Gastroenterolog	Riverton	Utah	United States	84065
54	Manassas Clinical Research Center	Manassas	Virginia	United States	20110
55	Virginia Commonwealth University Medical College of Virginia	Richmond	Virginia	United States	23298
56	Flinders Medical Centre	Bedford Park		Australia
57	Royal Brisbane and Women's Hospital	Brisbane		Australia
58	Monash Medical Centre	Clayton		Australia
59	Concord Repatriation General Hospital	Concord		Australia
60	Fiona Stanley Hospital	Murdoch		Australia
61	University of Calgary Liver Unit	Calgary	Alberta	Canada
62	LMC Diabetes & Endocrinology Ltd	London	Ontario	Canada
63	Toronto Liver Centre	Toronto	Ontario	Canada	M6H 3M1
64	Nova Scotia Health Authority	Halifax		Canada
65	CHU Grenoble-Alpes - Hopital Michallon	La Tronche		France
66	Hôpital de la Croix Rousse - HCL	Lyon		France
67	CHU de Montpellier - Hopital Saint Eloi	Montpellier		France
68	AP-HP Hopital Saint Antoine	Paris		France
69	CHU Bordeaux - Hopital Haut-Leveque	Pessac		France
70	AP-HP Hopital Paul Brousse	Villejuif		France
71	ID Clinic	Myslowice	CET	Poland
72	Fundacion de Investigacion (FDI)	San Juan		Puerto Rico
73	King's College Hospital	London		United Kingdom

Sponsors and Collaborators

• Axcella Health, Inc

Investigators

• Principal Investigator: Stephen Harrison, MD, Pinnacle Clinical Research

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• EMMPACT Phase 2b Clinical Trial Homepage

Publications

None provided.

• Responsible Party: Axcella Health, Inc
• ClinicalTrials.gov Identifier: NCT04880187
• Other Study ID Numbers: AXA1125-101
• First Posted: May 10, 2021
• Last Update Posted: Jul 27, 2022
• Last Verified: May 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Axcella Health, Inc: Steatosis; Lobular inflammation; Ballooning; Liver biopsy; Liver fat; Liver stiffness; NASH; Aminio Acids; Fibrosis
• Additional relevant MeSH terms: Fatty Liver; Non-alcoholic Fatty Liver Disease; Fibrosis

Study Results

No Results Posted as of Jul 27, 2022