NCT04554940ACTIVE_NOT_RECRUITINGanonymous

A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery

Sponsor

Source record

BioMarin Pharmaceutical

Phase

Source record

PHASE2

Modality

AI-normalized

protein therapy

Target

AI-normalized

vosoritide

Indication / condition

AI-normalized

Achondroplasia

Intervention

Source record

vosoritide

Related intelligence directories

Indication directory: Achondroplasia Modality directory: protein therapy Target directory: vosoritide

Source & freshness

Source record

NCT ID

NCT04554940

Original source

ClinicalTrials.gov

Source last updated

Mar 13, 2026

Ingested at

Jun 11, 2026

Internal sync

Jun 11, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record

View original source fields

NCT ID

NCT04554940

Title

A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery

Sponsor

BioMarin Pharmaceutical

Status

ACTIVE_NOT_RECRUITING

Phase

PHASE2

Condition raw

Achondroplasia

Condition normalized

Achondroplasia

Modality raw

protein therapy

Modality normalized

protein therapy

Target raw

vosoritide

Target normalized

vosoritide

Interventions

vosoritide

Public preview

Source record

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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TrialSignal

Clinical trial intelligence report

A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery

Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.

Generated

Jun 20, 2026

NCT ID

NCT04554940

Status

ACTIVE_NOT_RECRUITING

Phase

PHASE2

Sponsor

BioMarin Pharmaceutical

Executive brief

Investment-Ready Snapshot

Source & freshness

Provenance

SourceClinicalTrials.gov

Source updatedMar 13, 2026

Internal syncJun 11, 2026

Model versiontrialsignal-ai-v1

https://clinicaltrials.gov/study/NCT04554940

Indication

Achondroplasia

Modality

protein therapy

Target

vosoritide

Intervention

vosoritide

Source record