BMN 333 Long-Acting CNP
(333-301 [ASPEN])
Trial Status Recruiting
Condition
Achondroplasia
Technology
- Peptide
Title
A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (ASPEN)
Study Description
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.
Primary Outcome Measure*
- Predicted annualized growth velocity (AGV; Phase 2) and AGV (Phase 3)
Select Inclusion Criteria*
- Aged ≥ 2 to < 11 years (Phase 2) or ≥ 2 to < 18 years (Phase 3)
- Achondroplasia (confirmed by documented genetic testing) and open epiphyses
- Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
- Ambulatory and able to stand without assistance
Secondary Outcome Measures*
- Change from baseline in standing height, height Z-score, and upper to lower body segment ratio
- Incidence of adverse events (AEs), serious AEs, and events of interest
Select Exclusion Criteria*
- Any short stature condition other than achondroplasia
- History of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension
- Bone fractures of the long bones or spine within 6 months prior to screening
- Used vosoritide, any other approved product (except growth hormone [GH], as detailed below), investigational product, or investigational medical device for the treatment of achondroplasia or short stature at any time
- Been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start
* Additional measures/criteria may apply.
For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07441876
Pre-IND
Phase 1
Phase 2
Phase 3
Phase 4