Oral Infigratinib Therapy in Children with Achondroplasia
Authors' conclusion
The administration of oral infigratinib did not result in any apparent major safety signal and increased the annualized height velocity and z score and decreased the upper-to-lower body segment ratio at 18 months of treatment in cohort 5.
Study context
- Population
- Children with achondroplasia aged 3 to 11 yearsenrolled
- Participants
- 72 children
- Comparison
- Five daily oral infigratinib doses
- Outcome
- Primary efficacy outcome: change from baseline in the annualized height velocity; primary safety outcome: incidence of adverse events that led to a decrease in the dose or discontinuation of infigratinib
Methodological detailsphase 2, five sequential cohorts, dose escalation, 6 months of treatment followed by 12 months of extended treatment; cohorts 1 and 2 could escalate to the next dose level at months 6 and 12
- Phase 2
- Five sequential cohorts
- Dose escalation
- 6 months of treatment followed by 12 months of extended treatment; cohorts 1 and 2 could escalate to the next dose level at months 6 and 12
Main finding
As reported in the abstract
Change from baseline in annualized height velocity · 18 months
Cohort 5
2.50
mean · 95% CI 1.22–3.79 · cm per year
- P value, cohort 5 change from baseline
- P = 0.001
Further findings
| Outcome | Values |
|---|---|
| Change from baseline in height z score vs untreated achondroplasia reference population · 18 months | Cohort 5 mean 0.54 (95% CI 0.35–0.72) |
| Change from baseline in upper-to-lower body segment ratio · 18 months | Cohort 5 mean −0.12 (95% CI −0.18–-0.06) |
Safety
Adverse events · during treatment
- Adverse events resulting in treatment discontinuation
- 0
- All children had at least one adverse event, mostly mild or moderate
Original publication
Oral Infigratinib Therapy in Children with Achondroplasia. N Engl J Med. Published 18 November 2024. doi:10.
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