The randomized, double-blind, placebo-controlled Phase III trial will evaluate the safety and efficacy of GALNS in patients with MPS IVA.
In the study, patients will receive doses of 2mg/kg/week and 2mg/kg/every other week for a treatment period of 24 weeks.
BioMarin said that the primary endpoint will be the six-minute walk test, and the secondary endpoints will be the three-minute stair climb test and urine keratan sulfate concentration.
The study will be conducted at approximately 40 centres worldwide and will enroll 160 patients.