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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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

  • GC1130A

    Drug

    ICV injection

Treatment groups

9 Participants
are divided into 3 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 3Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidences and characteristics of adverse events

Time frame
up to 108 weeks

Secondary outcomes

1

Maximum concentration in cerebrospinal fluid (CSF)

GC1130A PK parameters

Time frame
up to 104 weeks
2

Area under the concentration-time curve in CSF

GC1130A PK parameters

Time frame
up to 104 weeks
3

Maximum concentration in serum

GC1130A PK parameters

Time frame
up to 104 weeks
4

Area under the concentration-time curve in serum

GC1130A PK parameters

Time frame
up to 104 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

GC Biopharma Corp

Lead sponsor

Source ClinicalTrials.gov
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