An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients

Phase 1

• Conditions: Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome; MedDRA version: 20.1Level: LLTClassification code 10028094Term: Mucopolysaccharidosis IHSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-002806-10-DE
• Lead Sponsor: Swedish Orphan Biovitrum AB (publ)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-10-01
• Last Update Posted: 23 March 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

MPS IIIA patients, as confirmed by both a documented deficiency in sulfamidase enzyme activity in concordance with a diagnosis of MPS IIIA and normal enzyme activity level of at least one other sulfatase measured in leukocytes, that are =12 to =72 months of age and that have a developmental age =12 months.

Are the trial subjects under 18? yes
Number of subjects for this age range: 9
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients with at least one S298P mutation in the SGSH gene are excluded, as well as patients that have received prior stem cell or gene therapy, or enzyme replacement therapy for MPS IIIA.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified