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

NCT03811028 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2022-02-25

Study results available
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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).

Conditions

  • Sanfilippo Syndrome Type A (MPS IIIA)

Interventions

DRUG

SOBI003

weekly i.v. infusion

Sponsors & Collaborators

  • Swedish Orphan Biovitrum

    lead INDUSTRY

Principal Investigators

  • Paul Harmatz, MD · Children´s Hospital and Research Center, Oakland

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Months
Max Age
78 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-19
Primary Completion
2021-04-30
Completion
2021-05-07
FDA Drug
Yes

Countries

  • United States
  • Turkey (Türkiye)

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03811028 on ClinicalTrials.gov