Compliance Behavior
Conditions
Brief summary
Oral administration of betaglucosylceramide was shown effective in reducing inflammation in animal models and was found safe in humans.
Detailed description
Patients with NASH to receive the treatment versus placebo for 40 weeks followed by a liver biopsy.
Interventions
DRUGBeta Glucosylceramide
Beta Glucosylceramide
DRUGPlacebo
normal saline
Sponsors
Hadassah Medical Organization
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Biopsy proven NASH
Exclusion criteria
* Other therapies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With a Decrease of 2 Points in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) Analysis is Per Protocol | Total score from baseline compared with week 40. | Number of Participants who had a decrease of 2 points in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) from baseline at 40 weeks per protocol analysis. The score is performed on the liver biopsy before and after treatment. The total score is used with a range from 4-16. A decrease in the total score by 2 points or more is considered an improvement, while an increase in the total score by 2 points or more is considered deterioration. No use of subscales for the data analysis was made. Allparticipants in both arms who showed a decrease of 2 points or more in the Non-Alcoholic Fatty Liver Disease Activity Score are conisdered as responders. Only values quantifying data that were actually measured and analyzed are included. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Placebo Placebo
Beta Glucosylceramide: Beta Glucosylceramide
placebo: placebo | 10 |
| Beta Glucosylceramide Beta Glucosylceramide
Beta Glucosylceramide: Beta Glucosylceramide
placebo: placebo | 13 |
| Total | 23 |
Baseline characteristics
| Characteristic | Placebo | Beta Glucosylceramide | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants | 13 Participants | 23 Participants |
| Region of Enrollment Israel | 10 participants | 13 participants | 23 participants |
| Sex: Female, Male Female | 3 Participants | 3 Participants | 6 Participants |
| Sex: Female, Male Male | 7 Participants | 10 Participants | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 10 | 0 / 13 |
| serious Total, serious adverse events | 0 / 10 | 0 / 13 |
Outcome results
Primary
Number of Participants With a Decrease of 2 Points in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) Analysis is Per Protocol
Number of Participants who had a decrease of 2 points in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) from baseline at 40 weeks per protocol analysis. The score is performed on the liver biopsy before and after treatment. The total score is used with a range from 4-16. A decrease in the total score by 2 points or more is considered an improvement, while an increase in the total score by 2 points or more is considered deterioration. No use of subscales for the data analysis was made. Allparticipants in both arms who showed a decrease of 2 points or more in the Non-Alcoholic Fatty Liver Disease Activity Score are conisdered as responders. Only values quantifying data that were actually measured and analyzed are included.
Time frame: Total score from baseline compared with week 40.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Number of Participants With a Decrease of 2 Points in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) Analysis is Per Protocol | 10 participants |
| Beta Glucosylceramide | Number of Participants With a Decrease of 2 Points in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS) Analysis is Per Protocol | 13 participants |