CompletedPhase 1/2

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

United States, Turkey (Türkiye)6 participantsStarted 2019-01-19

Plain-language 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).

Who can participate

Age range18 Months – 78 Months

SexALL

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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

What they're measuring

Safety as Measured by Adverse Events Frequencies (by Type and Severity)

Timeframe: From infusion week 25 up to week 104

Trial details

NCT IDNCT03811028

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-04-30

Results submitted2021-12-07

View on ClinicalTrials.gov More Sanfilippo Syndrome Type A (MPS IIIA) trials