NAFLD
Conditions
Brief summary
This is a randomized, single blind study to determine whether AXA1957, a novel composition of amino acids, is safe and well tolerated. Subjects will be adolescents with non-alcoholic fatty liver disease (NAFLD), and it will also examine liver biology using blood tests and magnetic resonance imaging (MRI).
Interventions
DIETARY_SUPPLEMENTAXA1957
Amino acids, food study
DIETARY_SUPPLEMENTPlacebo
placebo
Sponsors
Axcella Health, Inc
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Willing to participate in the study and provide written informed consent. * Male and female adolescent subjects aged 12 to 17 years * Subjects must not have participated in any diet/lifestyle intervention or observational studies, or engaged in any body weight altering regimens that resulted in body weight fluctuations (i.e. body weight loss or gain by 5%) in the preceding 30 days prior to Screening. * Non alcoholic fatty liver disease * Diabetes or prediabetes * A screening MRI consistent with liver inflammation and fibrosis Key
Exclusion criteria
* Current or history of significant alcohol consumption * History or presence of liver disease (other than NAFLD/NASH) * History or presence of cirrhosis and/or history or presence of hepatic decompensation * Any diabetes other than Type 2 * Other poorly controlled medical condition (for example, uncontrolled hypertension with a systolic blood pressure \> 100 mmHg) * Known sensitivity and/or history of clinically significant food intolerance/allergies to proteins (including whey, soy, casein, amino acids, etc.), * Unable or unwilling to adhere to contraception requirements * Any contraindications to a MRI scan * Any other condition that, in the opinion of the Investigator, renders the subject at risk for compliance, compromises the well-being of the subject, or hinders study completion
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects With Adverse Events (AEs). | AEs Baseline to week 13 - Part 1 | Study food product emergent adverse events (AEs). Subjects reporting \>or equal to 1 study food product-emergent AE. |
Countries
United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| AXA1957 AXA1957 20.4g
AXA1957: Amino acids, food study | 6 |
| Placebo Placebo 24g
Placebo: placebo | 3 |
| Total | 9 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part 1 | Subjects discontinued in response to restrictions on the study due to impact of COVID-19 pandemic | 4 | 1 |
| Part 1 | Withdrawal by Subject | 1 | 0 |
| Part 2 | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | Placebo | Total | AXA1957 |
|---|---|---|---|
| Age, Categorical <=18 years | 3 Participants | 9 Participants | 6 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 13.7 years STANDARD_DEVIATION 1.15 | 13.6 years STANDARD_DEVIATION 1.24 | 13.5 years STANDARD_DEVIATION 1.38 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 2 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 2 Participants | 7 Participants | 5 Participants |
| Region of Enrollment United Kingdom | 3 Participants | 9 Participants | 6 Participants |
| Sex: Female, Male Female | 1 Participants | 4 Participants | 3 Participants |
| Sex: Female, Male Male | 2 Participants | 5 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 3 |
| other Total, other adverse events | 2 / 6 | 1 / 3 |
| serious Total, serious adverse events | 0 / 6 | 0 / 3 |
Outcome results
Primary
Number of Subjects With Adverse Events (AEs).
Study food product emergent adverse events (AEs). Subjects reporting \>or equal to 1 study food product-emergent AE.
Time frame: AEs Baseline to week 13 - Part 1
Population: Safety analysis for Part 1 Baseline to week 13
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| AXA1957 | Number of Subjects With Adverse Events (AEs). | 2 Participants |
| Placebo | Number of Subjects With Adverse Events (AEs). | 1 Participants |