Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, United Kingdom
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: Through Week 260
Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software
Time frame: Through Week 260
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.