Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07441876 | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | PHASE2 | RECRUITING | 160 | — | — | Apr 1, 2026 | Sep 1, 2029 | May 29, 2026 | 10 | United States, Australia +4 |
| Arm | Type | Description |
|---|---|---|
| Phase 2: Low Dose | EXPERIMENTAL | Participants will be randomized to receive different dose levels of BMN 333 |
| Phase 2: Medium Dose | EXPERIMENTAL | Participants will be randomized to receive different dose levels of BMN 333 |
| Phase 2: High Dose | EXPERIMENTAL | Participants will be randomized to receive different dose levels of BMN 333 |
| Phase 2: Vosoritide | ACTIVE_COMPARATOR | weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide |
| Phase 3: BMN 333 at selected dose after Phase 2 | EXPERIMENTAL | - |
| Phase 3: Vosoritide | ACTIVE_COMPARATOR | weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide |
| Name | Type | Description |
|---|---|---|
| BMN 333 | DRUG | Administration: Weekly subcutaneous injection |
| Vosoritide Injection [Voxzogo] | DRUG | Administration: Daily subcutaneous injection |
Inclusion Criteria: 1. Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent 2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses 3. Are Tanner Stage I (Phase 2) or any Tanner stage (...