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

GC Biopharma

Status and phase: Enrolling; Phase 1

Conditions: Sanfilippo Syndrome Type A

Treatments: Drug: GC1130A

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT06567769

GC1130_MPS3A_P0101

Details and patient eligibility

About

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 pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA).

Enrollment: 9 estimated patients

Sex: All

Ages: 24 to 72 months old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants with documented MPS IIIA diagnosis
• Participants aged ≥ 24 months and ≤ 72 months

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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9 participants in 3 patient groups

Cohort 1

Experimental group

Treatment:

Drug: GC1130A

Cohort 2

Experimental group

Treatment:

Drug: GC1130A

Cohort 3

Experimental group

Treatment:

Drug: GC1130A