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)

GC Biopharma Corp5 sites in 3 countries9 target enrollmentNovember 21, 2024

InterventionsGC1130A

DrugsGC1130A

Overview

Phase: Phase 1
Intervention: GC1130A
Conditions: Not specified
Sponsor: GC Biopharma Corp
Enrollment: 9
Locations: 5
Primary Endpoint: Incidences and characteristics of adverse events
Status: Recruiting
Last Updated: 8 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).

Registry

clinicaltrials.gov

Start Date

November 21, 2024

End Date

June 1, 2027

Last Updated

8 months ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

GC Biopharma Corp

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Participants with documented MPS IIIA diagnosis
•Participants aged ≥ 12 months and ≤ 18 years

Exclusion Criteria

•Participants with significant non-MPS IIIA related central nervous system impairment
•Participants with previous complication from intraventricular drug administration
•Participants with contraindications for MRI scans and for neurosurgery
•Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study
•Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy

Arms & Interventions

Cohort 1

Intervention: GC1130A

Cohort 2

Intervention: GC1130A

Cohort 3

Intervention: GC1130A

Outcomes

Primary Outcomes

Incidences and characteristics of adverse events

Time Frame: up to 108 weeks

Secondary Outcomes

Maximum concentration in cerebrospinal fluid (CSF)(up to 104 weeks)
Area under the concentration-time curve in CSF(up to 104 weeks)
Maximum concentration in serum(up to 104 weeks)
Area under the concentration-time curve in serum(up to 104 weeks)
Change from baseline in CSF heparan sulfate concentration(up to 104 weeks)
Change from baseline in serum heparan sulfate concentration(up to 104 weeks)
Incidence of anti-drug and neutralizing antibodies of GC1130A in serum(up to 104 weeks)
Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF(up to 104 weeks)
Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)(up to 104 weeks)