Status:
COMPLETED
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
Lead Sponsor:
Swedish Orphan Biovitrum
Conditions:
Sanfilippo Syndrome Type A (MPS IIIA)
Eligibility:
All Genders
12-72 years
Phase:
PHASE1
PHASE2
Brief 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 th...
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 patie...
Eligibility Criteria
Inclusion
- Informed consent obtained from the patient's legally authorized representative(s)
- 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
- Chronological age of ≥12 and ≤72 months (i.e., 1 to 6 years) at the time of the first SOBI003 infusion and a developmental age ≥12 months at screening as assessed by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
- Medically stable patient who is expected to be able to comply with study procedures
Exclusion
- At least one S298P mutation in the SGSH gene
- Contraindications for anesthetic procedures, surgical procedure (venous access port) MRI scans and/or lumbar punctures
- History of poorly controlled seizures
- Patients is currently receiving psychotropic or other medications which in the investigator's opinion, would be likely to substantially confound test results
- Significant non-MPS IIIA-related central nervous system (CNS) impairment or behavioral disturbances, which in the investigator's opinion, would confound the scientific integrity or interpretation of study assessments
- Prior administration of stem cell or gene therapy, or ERT for MPS IIIA
- Concurrent or prior (within 30 days of enrolment into this study) participation in a study involving invasive procedures
Key Trial Info
Start Date :
June 19 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 25 2019
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT03423186
Start Date
June 19 2018
End Date
October 25 2019
Last Update
November 19 2021
Active Locations (4)
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1
Childrens's Hospital and Research Center
Oakland, California, United States, 94609
2
University of North Carolina Hospitals
Chapel Hill, North Carolina, United States, 27599
3
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
4
Gazi University Hospital
Ankara, Turkey (Türkiye)