BioMarin Pharmaceutical has initiated a Phase III study, to evaluate N-acetylgalactosamine 6-sulfatase (GALNS) as a treatment for Morquio Syndrome type A, a lysosomal storage disorder.
The disease is characterised by excessive storage of keratan sulphate, which can lead to systemic skeletal dysplasia, short stature, and joint abnormalities.
The randomised, double-blind and placebo-controlled Phase III trial will evaluate the safety and efficacy of GALNS in patients with the syndrome.
Patients will receive doses of 2mg/kg per week and 2mg/kg every other week for a treatment period of 24 weeks.
The primary endpoint will be the six-minute walk test, while the secondary endpoints will be the three-minute stair climb test and the concentration of keratin sulphate in urine.
The study will be conducted at approximately 40 centres worldwide and will enrol 160 patients.
