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 2

Withdrawn

• Conditions: mucopolysaccharidosis type IIIA; Sanfilippo syndrome; 10083624

• Registration Number: NL-OMON45765
• Lead Sponsor: Swedish Orphan International

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 1

Inclusion Criteria

1. Informed consent obtained from the patient*s legally authorized representative(s)
2. Patients with MPS IIIA, 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
3. Chronological age of >=12 and <=72 months (i.e., 1 to 6 years) at the time of the first SOBI003 infusion and has a developmental age >=12 months at screening as assessed by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
4. Medically stable patient who is expected to be able to comply with study procedures;* as determined by the reference ranges applied by the Greenwood Genetic Center

Exclusion Criteria

1. At least one S298P mutation in the SGSH gene
2. Contraindications for anesthetic procedures, surgical procedure (venous access port) MRI
scans and/or lumbar punctures
3. History of poorly controlled seizures
4. Patients is currently receiving psychotropic or other medications which in the investigator's opinion, would be likely to substantially confound test results
5. Significant non-MPS IIIA-related CNS impairment or behavioral disturbances, which in the investigator's opinion, would confound the scientific integrity or interpretation of study assessments
6. Prior administration of stem cell or gene therapy, or ERT for MPS IIIA
7. Concurrent or prior (within 30 days of enrolment into this study) participation in a study
involving invasive procedures

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary endpoint: Treatment Emergent Adverse Events (TEAEs) and Serious Adverse<br /><br>Events (SAEs).</p><br>