ELATIVE
Trial question
What is the role of elafibranor in patients with primary biliary cholangitis?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
96.0% female
4.0% male
N = 161
161 patients (154 female, 7 male).
Inclusion criteria: patients with primary biliary cholangitis who had an inadequate response to or unacceptable side effects with UDCA.
Key exclusion criteria: AIH or primary biliary cirrhosis-AIH overlap; evidence of clinically significant hepatic decompensation; history of alcohol abuse; evidence of any other unstable or untreated clinically significant disease.
Interventions
N=108 elafibranor (at a dose of 80 mg/day).
N=53 placebo (matching placebo).
Primary outcome
Biochemical response at week 52
51%
4%
51.0 %
38.3 %
25.5 %
12.8 %
0.0 %
Elafibranor
Placebo
Significant
increase ▲
NNT = 2
Significant increase in biochemical response at week 52 (51% vs. 4%; AD 47%, 95% CI 32 to 57).
Secondary outcomes
Significant increase in normalization of ALP level at week 52 (15% vs. 0%; AD 15%, 95% CI 6 to 23).
No significant difference in reduction in least-squares mean of Worst Itch NRS score at week 52 (1.93 points vs. 1.15 points; AD 0.78 points, 95% CI -0.42 to 1.99).
Significant increase in least square mean reduction in itch domain of the Primary Biliary Cirrhosis-40 quality-of-life questionnaire (2.5 points vs. 0.1 points; MD 2.3, 95% CI 0.7 to 4).
Safety outcomes
No significant differences in adverse events, severe or serious adverse events, and adverse events leading to treatment discontinuation.
Conclusion
In patients with primary biliary cholangitis who had an inadequate response to or unacceptable side effects with UDCA, elafibranor was superior to placebo with respect to biochemical response at week 52.
Reference
Kris V Kowdley, Christopher L Bowlus, Cynthia Levy et al. Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis. N Engl J Med. 2024 Feb 29;390(9):795-805.
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