Vosoritide official-source update · version 1
This page preserves this version’s findings and source pins. Open the current profile →
Official-source update
Reviewed 2026-09-19 · Preserved version 1
Seventy-five enrolled does not mean seventy-five randomized
NCT03583697 enrolled 75 children younger than five: 11 sentinel participants received vosoritide and 64 were randomized equally to vosoritide or placebo. The sentinel and randomized groups serve different purposes. NCT03583697 ↗
More findings and the sources behind them
Selected sponsor-submitted ClinicalTrials.gov records retrieved September 19, 2026, with design, population, endpoint and result-availability checks. This does not complete a registry search or replace primary-publication appraisal.
The growth endpoint requires its analysis population
The primary efficacy outcome was change in height Z-score at week 52. The registry separately reports the randomized comparison and an analysis adding the sentinel group; their modeled estimates should not be merged. NCT03583697 ↗
Safety observations are not all treatment-caused events
The posted table reports adverse events in every group and one death in the randomized vosoritide group. The participant-flow account labels that sudden-infant-death event unrelated, and the safety table reports no treatment-related serious events. These small samples cannot establish a precise rare-event risk. NCT03583697 ↗
The extension is open-label follow-up
NCT03989947 follows 73 participants who completed the earlier study, using a single-group open-label design. Its March 13, 2026 record is active, not recruiting, without posted summary results; it is not an additional independent placebo-controlled trial. NCT03989947 ↗
What remains to be checked
- Reconcile the young-child publication and regulatory extension with sentinel and randomized populations.
- Review longer-term developmental, functional and safety follow-up separately from growth endpoints.
Source dates and review coverage
PDF citations use file page numbers. Named webpage sections and registry fields identify the portions read. These are selected readings, not complete source coverage.
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia ↗
Source dated 2024-06-13; retrieved 2026-09-19.
- protocolSection.eligibilityModule and armsInterventionsModule
- resultsSection.participantFlowModule
- resultsSection.outcomeMeasuresModule
SHA-256:
67e90e6eeb1b0d458b42edcb6e30ca37b0c76a6d08db5a1ce96226678acda430An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia ↗
Source dated 2026-03-13; retrieved 2026-09-19.
- protocolSection.identificationModule, designModule and armsInterventionsModule
- protocolSection.eligibilityModule and armsInterventionsModule
- protocolSection.statusModule; presence or absence of resultsSection in retrieved record
SHA-256:
9f16f6b15187f3aaf6ef47241d4be33ab90022159089572c18758a667866b299