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

Conditions

• Achondroplasia

Eligibility Criteria

Sex

ALL

Age

2 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• BMN 333 — DRUG
  Administration: Weekly subcutaneous injection
• Vosoritide Injection [Voxzogo] — DRUG
  Administration: Daily subcutaneous injection

Study Details

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.

Key Dates

Start date

Apr 30, 2026

Status verified

Apr 2026

Primary completion

Jun 30, 2029

Completion

Sep 30, 2029

Study Design

Enrollment

160 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Phase 2: Low Dose
  Participants will be randomized to receive different dose levels of BMN 333
• Experimental: Phase 2: Medium Dose
  Participants will be randomized to receive different dose levels of BMN 333
• Experimental: Phase 2: High Dose
  Participants will be randomized to receive different dose levels of BMN 333
• Active Comparator: Phase 2: Vosoritide
  weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide
• Experimental: Phase 3: BMN 333 at selected dose after Phase 2
• Active Comparator: Phase 3: Vosoritide
  weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide

Primary Outcome Measure

Phase 2: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data] [ Time Frame: 52 weeks ]

Central Contacts

• Trial Specialist
  1-800-983-4587
  [email protected]
• Study Manager
  1-800-983-4587
  [email protected]

Locations (5)

Find similar trials in Oakland, CA

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