DesignRandomized, double-blind, placebo-controlled in children 2-11 years
Enrollment63
Treatment Arms
Primary Endpoints
[{"name":"Annualized Height Velocity at 52 weeks","type":"continuous","results":[{"unit":"cm/year","value":5.39,"arm_name":"TransCon CNP 100µg/kg/week"}]}]
Efficacy Results
Achieved primary efficacy objective. Superiority at 100 µg/kg/week vs placebo. Mean AHV 5.39 cm/year (n=40) at pivotal dose. All 57 children completed and entered OLE.
Assessment
Phase 2 complete. Supported Phase 3 ApproaCH trial which also succeeded. NDA filed March 2025.