Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Phase 1

Not yet recruiting

• Conditions: Sanfilippo Syndrome Type A

• Registration Number: NCT06567769
• Lead Sponsor: GC Biopharma Corp

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-8-20
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Not yet recruiting
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

Inclusion Criteria:<br><br> - Participants with documented MPS IIIA diagnosis<br><br> - Participants aged = 24 months and = 72 months<br><br>Exclusion Criteria:<br><br> - Participants with significant non-MPS IIIA related central nervous system impairment<br><br> - Participants with previous complication from intraventricular drug administration<br><br> - Participants with contraindications for MRI scans and for neurosurgery<br><br> - Participants that received treatment with any investigational drug or a device<br> intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the<br> study<br><br> - Participants that received a hematopoietic stem cell or bone marrow transplant or<br> received gene therapy

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Incidences and characteristics of adverse events

Secondary Outcome Measures

Name	Time	Method
Maximum concentration in cerebrospinal fluid (CSF);Area under the concentration-time curve in CSF;Maximum concentration in serum;Area under the concentration-time curve in serum;Change from baseline in CSF heparan sulfate concentration;Change from baseline in serum heparan sulfate concentration;Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF;Incidence of anti-drug and neutralizing antibodies of GC1130A in serum;Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)