CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 6 enrolled
Drug / intervention
SOBI003drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03811028
NCT03811028Phase 2Completed
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An Open, Single-arm, Multicenter Extension Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Swedish Orphan Biovitrum·interventional·Posted Jan 22, 2019·Updated Feb 25, 2022

In Brief

A Phase 2 clinical trial evaluating SOBI003 for Sanfilippo Syndrome Type A (MPS IIIA). Completed, enrolled 6 participants across 3 sites in 2 countries.

Detailed 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).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSanfilippo Syndrome Type A (MPS IIIA)
CountriesTurkey (Türkiye), United States
Collaborators--

Timeline

Phase 2CompletedFinished
20192020202120222023202420252026
First PostedJan 22, 2019
Enrollment StartJan 19, 2019
Primary CompletionApr 30, 2021
Study CompletionMay 7, 2021
TodayJul 2, 2026
Enrollment to primary: 2.3 yearsPosted 7.4 years ago

Interventions

SOBI003drug

weekly i.v. infusion