SOBI003 / SOBI - LARVOL DELTA

Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study. (PubMed, Mol Genet Metab) - P1/2 | "SOBI003 was well tolerated when administered as weekly intravenous infusions at doses of up to 20 mg/kg for up to 104 weeks. ADA development was common and likely affected both PK and PD parameters. SOBI003 crossed the BBB and showed pharmacological activity on HS in CSF."

Clinical • Journal • CNS Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients (clinicaltrials.gov) - P1/2; N=6; Completed; Sponsor: Swedish Orphan Biovitrum; Active, not recruiting ➔ Completed; Trial completion date: Dec 2021 ➔ May 2021; Trial primary completion date: Dec 2021 ➔ Apr 2021

Clinical • Trial completion • Trial completion date • Trial primary completion date • Lysosomal Storage Diseases • Pediatrics

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients (clinicaltrials.gov) - P1/2; N=6; Completed; Sponsor: Swedish Orphan Biovitrum; Active, not recruiting ➔ Completed; Trial completion date: Feb 2020 ➔ Oct 2019; Trial primary completion date: Feb 2020 ➔ Oct 2019

Clinical • Trial completion • Trial completion date • Trial primary completion date

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients (clinicaltrials.gov) - P1/2; N=9; Active, not recruiting; Sponsor: Swedish Orphan Biovitrum; Recruiting ➔ Active, not recruiting

Enrollment closed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients (clinicaltrials.gov) - P1/2; N=9; Active, not recruiting; Sponsor: Swedish Orphan Biovitrum; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients (clinicaltrials.gov) - P1/2; N=9; Recruiting; Sponsor: Swedish Orphan Biovitrum; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients (clinicaltrials.gov) - P1/2; N=9; Not yet recruiting; Sponsor: Swedish Orphan Biovitrum

Clinical • New P1/2 trial