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Enzyme Replacement Therapy

GC1130A for Sanfilippo Syndrome

Phase 1
Recruiting
Research Sponsored by GC Biopharma Corp
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
* Participants with documented MPS IIIA diagnosis
* Participants aged ≥ 24 months and ≤ 72 months
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 104 weeks
Awards & highlights
No Placebo-Only Group

Summary

This trial aims to test the safety and effectiveness of a new treatment called recombinant human heparan N-sulfatase in children with Sanfilippo Syndrome Type A. The treatment will be

Who is the study for?
This trial is for young children aged 2 to 6 years with a confirmed diagnosis of Sanfilippo Syndrome Type A (MPS IIIA), which is a rare genetic condition affecting brain function.
What is being tested?
The study tests GC1130A, an experimental medication intended to replace a missing enzyme in patients. It's given directly into the brain fluid through a special device to see if it's safe and effective.
What are the potential side effects?
Possible side effects may include reactions at the injection site, headache, fever, vomiting, or changes in behavior due to direct administration into the brain but specific side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with MPS IIIA.
Select...
I am between 2 and 6 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 104 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 104 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Area under the concentration-time curve in CSF
Area under the concentration-time curve in serum
Maximum concentration in cerebrospinal fluid (CSF)
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: Cohort 3Experimental Treatment1 Intervention
Group II: Cohort 2Experimental Treatment1 Intervention
Group III: Cohort 1Experimental Treatment1 Intervention

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Who is running the clinical trial?

Novel Pharma Inc.UNKNOWN
GC Biopharma CorpLead Sponsor
8 Previous Clinical Trials
629 Total Patients Enrolled
~6 spots leftby Jun 2027
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