Human RCTHumanRandomizedBlindedControlled
Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
Ravi Savarirayan, Louise Tofts, Melita Irving, William Wilcox, Carlos A. Bacino, et al.
Study design
Methods snapshot
- Population
- Ambulatory children aged 5 to <18 years with achondroplasia
- Sample size
- 121
- Intervention
- Vosoritide
- Route
- Subcutaneous
- Dose reported
- 15.0 µg/kg
- Frequency
- Once daily
- Duration
- 52 weeks
- Control / placebo
- Controlled study
Outcomes
Primary
- Change from baseline in annualized growth velocity at 52 weeks
Results
The adjusted mean difference in annualized growth velocity favored vosoritide by 1.57 cm/year (95% CI 1.22–1.93).
Interpretation
Provides phase 3 evidence for increased annualized growth velocity in the studied pediatric achondroplasia population.
Adverse events
- 119 participants had at least one adverse event.
- No serious adverse event was considered treatment-related in the report.
Limitations
- Final adult height was not established
- Long-term harms were not established by the 52-week trial
Connected records
Original source
Sempre consulte o artigo original para métodos completos, análises e contexto editorial.
Open PubMed record ↗