StudyFinder
A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients with Sanfilippo Syndrome Type A (MPS IIIA).
Status: Recruiting
The purpose of the study is to see if GC1130A, delivered directly to the central ventricle of the brain is safe and tolerable as a means of treating the neurologic disease in MPS 3A.
Sex: Male or Female
Age Group: Up to 18 years old
Inclusion Criteria:
• documented MPS IIIA diagnosis
• ≥ 24 months and ≤ 72 months of age
Exclusion Criteria:
• significant non-MPS IIIA related central nervous system impairment
• previous complication from intraventricular drug administration
• contraindications for MRI scans and for neurosurgery
• received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days
• received a hematopoietic stem cell or bone marrow transplant or received gene therapy
Conditions:
Rare Diseases, Rare Diseases
Keywords:
MPS IIIA, Sanfilippo Syndrome
Study Contact: Melissa Fetterley - melissa.fetterley@fairview.org
Principal Investigator: Chester Whitley, MD, PhD
Phase: PHASE1
IRB Number: STUDY00022733
See this study on ClinicalTrials.gov