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

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

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06567769

Enrollment

Registered

2024-08-23

Start date

2024-11-21

Completion date

2027-06-01

Last updated

2026-09-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sanfilippo Syndrome Type A

Keywords

Mucopolysaccharidosis Type III Subtype A, MPS IIIA

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).

Interventions

DRUGGC1130A

ICV injection

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

12 Months to 18 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Incidences and characteristics of adverse events	up to 108 weeks

Secondary

Measure	Time frame	Description
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	—

Countries

South Korea, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 9, 2026