Status:
COMPLETED
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Lead Sponsor:
Swedish Orphan Biovitrum
Conditions:
Sanfilippo Syndrome Type A (MPS IIIA)
Eligibility:
All Genders
18-78 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, single-arm, multicenter extension study to assess the safety, tolerability and efficacy of long-term SOBI003 treatment in pediatric MPS IIIA patients. The study is an extension of the...
Eligibility Criteria
Inclusion
- Completion of study SOBI003-001
- Informed consent obtained from the patient´s legally authorized representative
Exclusion
- If, in the opinion of the investigator, there are patient specific safety concerns that contraindicates further treatment with SOBI003
Key Trial Info
Start Date :
January 19 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 7 2021
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT03811028
Start Date
January 19 2019
End Date
May 7 2021
Last Update
February 25 2022
Active Locations (3)
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1
Children´s Hospital and research center
Oakland, California, United States, 94609
2
University of North Carolina hospitals
Chapel Hill, North Carolina, United States, 27599
3
Gazi University Hospital
Ankara, Turkey (Türkiye)