RecruitingPhase 1NCT06567769

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)

Sponsor

GC Biopharma Corp

Enrollment

9 participants

Start Date

Nov 21, 2024

Study Type

INTERVENTIONAL

Conditions

Sanfilippo Syndrome Type A

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).

Eligibility

Min Age: 12 MonthsMax Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called GC1130A for people with sanfilippo syndrome type a. The study is currently recruiting participants at 5 locations. People eligible for this study include aged 12 Months to 18 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGGC1130A

ICV injection

Locations(5)

UCSF Benioff Children's Hospital

Oakland, California, United States

University of Minnesota

Minneapolis, Minnesota, United States

National Center for Child Health and Development

Setagaya City, Tokyo, Japan

Ajou University Medical Center

Suwon, Gyeongi-do, South Korea

Samsung Medical Center

Seoul, South Korea

View Full Details on ClinicalTrials.gov

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NCT06567769