A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
Status:
Completed
Trial end date:
2019-10-25
Target enrollment:
Participant gender:
Summary
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS
IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal
degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS
IIIA is characterized by devastating neurodegeneration with initially mild somatic
involvement. The aims of the present study is to assess the dose related safety,
tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, a chemically
modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).