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/NCT03423186
NCT03423186Phase 2Completed
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An Open, Non-controlled, Parallel, Ascending Multiple-dose, Multicenter Study to Assess Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of SOBI003 in Pediatric MPS IIIA Patients

Swedish Orphan Biovitrum·interventional·Posted Feb 6, 2018·Updated Nov 19, 2021

In Brief

A Phase 2 clinical trial evaluating SOBI003 for Sanfilippo Syndrome Type A (MPS IIIA). Completed, enrolled 6 participants across 4 sites in 3 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 aims of the present study is to assess the dose related safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, 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)
CountriesGermany, Turkey (Türkiye), United States
Collaborators--

Timeline

Phase 2CompletedFinished
201820192020202120222023202420252026
First PostedFeb 6, 2018
Enrollment StartJun 19, 2018
Primary CompletionOct 25, 2019
TodayJul 2, 2026
Enrollment to primary: 1.3 yearsPosted 8.4 years ago

Interventions

SOBI003drug

Weekly i.v.infusion