Overview
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Status:
Completed
Completed
Trial end date:
2021-05-07
2021-05-07
Target enrollment:
0
0
Participant gender:
All
All
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 aim of the present study is to assess the safety, tolerability and efficacy of long-term SOBI003 treatment. SOBI003 is a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).Phase:
Phase 1/Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Swedish Orphan Biovitrum
Criteria
Inclusion Criteria:- Completion of study SOBI003-001
- Informed consent obtained from the patient´s legally authorized representative
Exclusion Criteria:
- If, in the opinion of the investigator, there are patient specific safety concerns
that contraindicates further treatment with SOBI003