NCT03423186

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

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 12 Months – 72 Months
• Healthy volunteers: Not accepted

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).

Detailed description

This is an open-label, non-controlled, parallel, sequential ascending multiple-dose, multicenter study to assess the dose related safety, tolerability, PK and PD of SOBI003 in pediatric MPS IIIA patients. Patients between 1 and 6 years of age who have not received previous treatment for MPS IIIA with an ERT, gene- or stem cell therapy will be eligible to participate in the study. The study is planned to consist of 3 dose cohorts, each comprising 3 patients. Treatment initiations will be staggered within each cohort in order to be able to observe, interpret and treat possible adverse reactions. SOBI003 is administered as weekly i.v. infusions over a period of 24 weeks. Upon completion of the 24-week treatment period with satisfactory tolerability, the patient is offered to receive continued SOBI003 treatment by participation in an extension study.

Conditions

• Sanfilippo Syndrome Type A (MPS IIIA)

Interventions

Type	Name	Description
DRUG	SOBI003	Weekly i.v.infusion

Timeline

• Start date: 2018-06-19
• Primary completion: 2019-10-25
• Completion: 2019-10-25
• First posted: 2018-02-06
• Last updated: 2021-11-19
• Results posted: 2021-04-22

Locations

4 sites across 3 countries: United States, Germany, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study