Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia
Primary result · Change in androstenedioneResult — go to the resultLeast-squares mean difference −268 ng per deciliterAuthors' conclusion
In this phase 3 trial, crinecerfont was superior to placebo in reducing elevated androstenedione levels in pediatric participants with CAH and was also associated with a decrease in the glucocorticoid dose from supraphysiologic to physiologic levels while androstenedione control was maintained.
Study context
- Population
- Pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency; baseline mean glucocorticoid dose 16.4 mg/m²/day, mean androstenedione 431 ng/dLrandomized 2:1
- Participants
- 103 participants
- Comparison
- Crinecerfont vs placebo for 28 weeks
- Crinecerfontn = 69
- Placebon = 34reference
- Primary endpoint
- Change in androstenedione level from baseline
- Follow-up
- Week 4
Methodological detailsphase 3, multinational, placebo-controlled, 100 (97%) remained in the trial at 28 weeks
- Phase 3
- Multinational
- Placebo-controlled
- 100 (97%) remained in the trial at 28 weeks
Primary result
Change in androstenedione
Least-squares mean difference
−268 ng per deciliter
P < 0.001
Change by group
Crinecerfont
−197
ng per deciliter
Placebo
71
ng per deciliter
As reported in the abstract
Secondary outcomes
Observed mean androstenedione (before morning glucocorticoid dose) · week 4
| Outcome | Crinecerfont | Placebo | LSMD (percentage points) | P value |
|---|---|---|---|---|
| Key secondary: percent change in glucocorticoid dose with androstenedione control maintained · week 28 | mean decreased by 18.0% | mean increased by 5.6% | −23.5 | P < 0.001 |
Safety
Adverse events
- Headache, pyrexia, and vomiting were the most common AEs
Original publication
Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia. N Engl J Med. Published 2 June 2024. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →