RESPONSE
Trial question
What is the role of seladelpar in patients with primary biliary cholangitis?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
95.0% female
5.0% male
N = 193
193 patients (183 female, 10 male).
Inclusion criteria: patients with primary biliary cholangitis with an inadequate response to or unacceptable side effects with UDCA.
Key exclusion criteria: advanced primary biliary cholangitis; hepatic decompensation; any other chronic liver disease.
Interventions
N=128 seladelpar (an oral dose of 10 mg daily).
N=65 placebo (matching placebo).
Primary outcome
Biochemical response
61.7%
20%
61.7 %
46.3 %
30.9 %
15.4 %
0.0 %
Seladelpar
Placebo
Significant
increase ▲
NNT = 2
Significant increase in biochemical response (61.7% vs. 20%; AD 41.7%, 95% CI 27.7 to 53.4).
Secondary outcomes
Significant increase in normalization of ALP level at 12 months (25% vs. 0%; AD 25%, 95% CI 18.3 to 33.2).
Significantly greater reduction in the score on the pruritus numerical rating scale at 6 months in patients with moderate-to-severe pruritus (3.2 points vs. 1.7 points; MD 1.5, 95% CI 0.5 to 2.5).
Significantly greater reduction in the score on the pruritus numerical rating scale at 6 months in overall population (1.3 points vs. 0.4 points; MD 0.9, 95% CI 0.5 to 1.4).
Safety outcomes
No significant differences in adverse events, serious adverse events.
Conclusion
In patients with primary biliary cholangitis with an inadequate response to or unacceptable side effects with UDCA, seladelpar was superior to placebo with respect to biochemical response.
Reference
Gideon M Hirschfield, Christopher L Bowlus, Marlyn J Mayo et al. A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis. N Engl J Med. 2024 Feb 29;390(9):783-794.
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