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A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

NCT03423186 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2021-11-19

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 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).

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 · Childrens's Hospital and Research Center Oakland

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
12 Months
Max Age
72 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-19
Primary Completion
2019-10-25
Completion
2019-10-25
FDA Drug
Yes

Countries

  • United States
  • Germany
  • 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 NCT03423186 on ClinicalTrials.gov