A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Completed · Phase 1/Phase 2

Conditions studied: Sanfilippo Syndrome Type A (MPS IIIA)

In brief

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).

Key facts

Study ID

NCT03811028

Run by

Swedish Orphan Biovitrum

People needed

6

Starts

2019-01-19

Expected to finish

2021-05-07

Last updated by the study team

2022-02-25

Who can join

Age: 2 and older, up to 7. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Completion of study SOBI003-001
• Informed consent obtained from the patient´s legally authorized representative

You may not qualify if…

• If, in the opinion of the investigator, there are patient specific safety concerns that contraindicates further treatment with SOBI003

Where it is running

• Children´s Hospital and research center — Oakland, California, United States
• University of North Carolina hospitals — Chapel Hill, North Carolina, United States
• Gazi University Hospital — Ankara, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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