Phase 1 Study of GC1130A in Pediatric 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) - GC1130_MPS3A_P0101

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2031240514

Enrollment

Registered

2024-11-28

Start date

2024-12-26

Completion date

Unknown

Last updated

2025-07-18

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

Conditions

Sanfilippo syndrome Type A (MPS IIIA)

Interventions

GC1130A is administered intracerebroventricularly via intracerebroventricular access device at dose levels 1, 2, and 3 every 2 weeks. Dose higher than dose level 3 can be used after review of safety d

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Participants with documented MPS IIIA diagnosis 2. Participants aged => 24 months and =< 72 months

Exclusion criteria

Exclusion criteria: 1. Participants with significant non-MPS IIIA related central nervous system impairment 2. Participants with previous complication from intraventricular drug administration 3. Participants with contraindications for MRI scans and for neurosurgery 4. 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 5. 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	—

Countries

Japan, South Korea, United States

Contacts

Public ContactRyuichi Nagao

Medpace Japan K.K.

RSJapan1@medpace.com+81-3-4563-7000

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Feb 4, 2026