CAHtalyst Pediatric
Trial question
What is the role of crinecerfont in pediatric patients with congenital adrenal hyperplasia?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
49.0% female
51.0% male
N = 103
103 patients (50 female, 53 male).
Inclusion criteria: pediatric patients with congenital adrenal hyperplasia.
Key exclusion criteria: any condition other than congenital adrenal hyperplasia; long-term corticosteroid therapy; clinically significant unstable medical condition or chronic disease other than congenital adrenal hyperplasia; history of cancer unless considered to be cured.
Interventions
N=69 crinecerfont (at a dose of 25-100 mg BID based on the body weight for 28 weeks).
N=34 placebo (matching placebo BID for 28 weeks).
Primary outcome
Reduction in androstenedione level at week 4
197 ng/dL
-71 ng/dL
197.0 ng/dL
147.8 ng/dL
98.5 ng/dL
49.3 ng/dL
0.0 ng/dL
-49.3 ng/dL
-98.5 ng/dL
Crinecerfont
Placebo
Significant
increase ▲
Significantly greater reduction in androstenedione level at week 4 (197 ng/dL vs. -71 ng/dL; LSMD 268, 95% CI 132 to 403).
Secondary outcomes
Significantly greater reduction in serum 17-hydroxyprogesterone at week 4 (5865 ng/dL vs. -556 ng/dL; LSMD 6421, 95% CI 4454 to 8387).
Significantly greater reduction in corticosteroid dose with androstenedione control at week 28 (18% vs. -5.6%; LSMD 23.5, 95% CI 17.2 to 29.9).
Significantly greater reduction in mean corticosteroid daily dose at week 28 (18.4% vs. -5.5%; LSMD 23.8, 95% CI 17.5 to 30.1).
Safety outcomes
No significant difference in adverse events.
Conclusion
In pediatric patients with congenital adrenal hyperplasia, crinecerfont was superior to placebo with respect to reduction in androstenedione level at week 4.
Reference
Kyriakie Sarafoglou, Mimi S Kim, Maya Lodish et al. Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):493-503.
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