deltatrials
Recruiting INTERVENTIONAL NCT07441876

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (ASPEN)

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Sponsor: BioMarin Pharmaceutical

Conditions Achondroplasia
Interventions BMN 333 Vosoritide Injection [Voxzogo]
Updated 2 times since 2026 Last updated: Apr 20, 2026 Started: Apr 1, 2026 Primary completion: Jun 1, 2029 Completion: Sep 1, 2029
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

A observational or N/A phase clinical study on Achondroplasia, this trial is actively recruiting participants. The trial is conducted by BioMarin Pharmaceutical and has accumulated 2 data snapshots since 2026. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.

Study Description(click to expand)

The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following: * Study duration: up to 61 weeks (from screening to Safety Follow-up visit) * Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily

The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following:

* Study duration: up to 61 weeks (from screening to Safety Follow-up visit) * Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily

Status Flow

~Mar 2026 – ~Apr 2026 · 53 days · monthly snapshotNot Yet RecruitingApr 23, 2026 – present · 4 months · daily APIRecruiting

Change History

2 versions recorded

  1. Apr 23, 2026 — Present [daily]

    Recruiting

    Status: Not Yet Recruiting → Recruiting · Phase: PHASE2/PHASE3 → None

  2. Mar 2026 — Apr 2026 [monthly]

    Not Yet Recruiting PHASE2/PHASE3

    First recorded

Eligibility Summary

This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.

Contact Information

Sponsor contact:
  • BioMarin Pharmaceutical
Data source: ClinicalTrials.gov

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations