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

NCT06567769 — Phase 1, Active not recruiting, 9 participants, GC Biopharma Corp

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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)

Registry	NCT06567769
Status	Active not recruiting
Phase	Phase 1
Study type	Interventional; non randomized, sequential, masking: none, treatment
Enrollment	9 (Estimated)
Sponsor	GC Biopharma Corp (Industry)
Conditions	Sanfilippo Syndrome Type A
Interventions	GC1130A (Drug)
Dates	start 2024-11-21, primary completion 2027-06-01 (estimated), completion 2027-06-01 (estimated), first posted 2024-08-23, last updated 2026-09-08
Locations	4 sites in United States, South Korea

-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).

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+Arms and interventions

Arm	Type	Interventions
Cohort 1	Experimental	Drug: GC1130A
Cohort 2	Experimental	Drug: GC1130A
Cohort 3	Experimental	Drug: GC1130A

GC1130A (Drug): ICV injection

+Outcome measures

Primary outcomes

Incidences and characteristics of adverse events [up to 108 weeks]

Secondary outcomes

Maximum concentration in cerebrospinal fluid (CSF) [up to 104 weeks]: GC1130A PK parameters
Area under the concentration-time curve in CSF [up to 104 weeks]: GC1130A PK parameters
Maximum concentration in serum [up to 104 weeks]: GC1130A PK parameters
Area under the concentration-time curve in serum [up to 104 weeks]: GC1130A PK parameters
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 CSF [up to 104 weeks]
Incidence of anti-drug and neutralizing antibodies of GC1130A in serum [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]

+Eligibility

Sex: All, ages: 12 Months to 18 Years

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

+Locations (4)

Facility	City	Country
UCSF Benioff Children's Hospital	Oakland, California	United States
University of Minnesota	Minneapolis, Minnesota	United States
Ajou University Medical Center	Suwon, Gyeongi-do	South Korea
Samsung Medical Center	Seoul	South Korea

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