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Recruiting NCT06567769

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

Phase I Interventional Sanfilippo Syndrome Type A

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: GC1130A.
Who it may be relevant to
Registry conditions: Sanfilippo Syndrome Type A. Basic parameters: 12 months — 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Japan, South Korea
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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)

Overview

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

  • Drug GC1130A
    ICV injection

Primary outcome measures

  • Incidences and characteristics of adverse events [Time frame: up to 108 weeks]
Secondary outcome measures (9)
  • Maximum concentration in cerebrospinal fluid (CSF) [Time frame: up to 104 weeks]
  • Area under the concentration-time curve in CSF [Time frame: up to 104 weeks]
  • Maximum concentration in serum [Time frame: up to 104 weeks]
  • Area under the concentration-time curve in serum [Time frame: up to 104 weeks]
  • Change from baseline in CSF heparan sulfate concentration [Time frame: up to 104 weeks]
  • Change from baseline in serum heparan sulfate concentration [Time frame: up to 104 weeks]
  • Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF [Time frame: up to 104 weeks]
  • Incidence of anti-drug and neutralizing antibodies of GC1130A in serum [Time frame: 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) [Time frame: up to 104 weeks]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 2 centers
  • UCSF Benioff Children's Hospital — Oakland
  • University of Minnesota — Minneapolis
South Korea · 2 centers
  • Ajou University Medical Center — Suwon
  • Samsung Medical Center — Seoul
Japan · 1 center
  • National Center for Child Health and Development — Setagaya City

Identifiers

NCT: NCT06567769 · GC1130_MPS3A_P0101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗