Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia
Primary result · Change from baseline in annualized height velocityResult — go to the resultDifference in least-squares mean change 1.74 cm per year 95% CI 1.31–2.17Authors' conclusion
In children with achondroplasia, treatment with once-daily oral infigratinib for 52 weeks resulted in a significantly greater increase from baseline in the annualized height velocity than placebo.
Study context
- Population
- Children with achondroplasia, 3 to 17 years of agerandomized 2:1
- Participants
- 114 children
- Comparison
- Oral infigratinib 0.25 mg/kg once daily vs placebo for 52 weeks
- Infigratinibn = 75
- Placebon = 39reference
- Primary endpoint
- Change from baseline in annualized height velocity, infigratinib vs placebo
- Follow-up
- Week 52
Methodological detailsphase 3, multicenter, double-blind, placebo-controlled
- Phase 3
- Multicenter
- Double-blind
- Placebo-controlled
Primary result
As reported in the abstract
Change from baseline in annualized height velocity
Difference in least-squares mean change
1.74 cm per year
95% CI 1.31–2.17
P < 0.001
Infigratinibvs Placebo
difference in least-squares mean change 1.74 cm per year (95% CI 1.31–2.17); P < 0.001
Secondary outcomes
| Outcome | Difference in least-squares mean change (96% CI) | P value | |
|---|---|---|---|
| Key secondary: change from baseline in height z score · week 52 | 0.32 (0.23–0.41) | P < 0.001 | |
| Key secondary: change from baseline in upper-to-lower body segment ratio · week 52 | −0.02 (−0.06 to 0.01) | ||
| −0.200.20.40.6Difference (linear scale) |
Safety
Adverse events
Serious adverse events
- Adverse events: infigratinib denominator 74
Serious adverse events
- No serious AEs or AEs leading to discontinuation considered by the investigator related to infigratinib or placebo
Original publication
Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia. N Engl J Med. Published 28 June 2026. doi:10.
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