Summary:
Safety and Efficacy Study for Children with Early Stage MPSIIIA Disease (Sanfilippo A Syndrome).
WHAT IS THE PURPOSE OF THIS STUDY?
This study is being conducted to evaluate the safety and efficacy of intrathecal delivery of rhHNS on cognitive function in children, and includes random assignment of participants to treatment or non-treatment comparator groups.
Study Intervention: Recombinant human heparan N-sulfatase [rhHNS]
Possibility of Active Treatment: There is a 67% chance that a study participant will receive active study treatment.
Phase:2b
Enrollment Goal: 18
Start Date: Feb 2014
End Date: March 2016
Study Identifiers: ClinicalTrials.gov NCT02060526
PARTICIPATION DURATION/ REQUIREMENTS
Study participation will last between 49 to 56 weeks, including 1 to 4 weeks for screening, a 48-week treatment or observation phase, and an additional 4 weeks of safety follow up if not electing to enroll in an extension study.
ENTRY CRITERIA
- Confirmed diagnoses of MPS IIIA (Sanfilippo Syndrome Type A)
- Is between 12 and 48 months of age
- The patient has a DQ score ≥60
- Patients with at least one S298P mutation in SGSH associated with attenuated disease OR there is documentation of the S298P mutation in a sibling affected by MPS IIIA (provided parental consent is obtained to use this information) will not be eligible
- No presence of significant non-MPS IIIA related central nervous system impairment or behavioral disturbances that would interfere with study interpretation or results
- No safety contra-indications to the implantation of an IDDD or lumbar puncture