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

ConditionSanfilippo Syndrome Type A

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age12-18

SponsorGC Biopharma Corp

About this trial

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 criteria

Qualifiers

Participants with documented MPS IIIA diagnosis

Participants aged ≥ 12 months and ≤ 18 years

Disqualifiers

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

Trial design

Treatments tested in this trial

GC1130A

Treatment groups

9 Participants

are divided into 3 treatment groups

Sponsors and collaborators

GC Biopharma Corp

Lead sponsor

Source ClinicalTrials.gov