Phase 1 Study of GC1130A in Patients With Sanfilippo Synd... | NCT06567769

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

NCT06567769

Sponsor

GC Biopharma Corp

Status

Recruiting

Last Update Posted

Aug 28, 2025Actual

Enrollment

9Estimated

Phase

Phase 1

Conditions

Sanfilippo Syndrome Type A

Interventions

GC1130A

Countries

United States
Japan
South Korea

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

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)

Status Module

Record Verification Date

Aug 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 21, 2024Actual

Primary Completion Date

Jun 2027Estimated

Completion Date

Jun 2027Estimated

First Submitted Date

Aug 20, 2024

First Submission Date that Met QC Criteria

Aug 20, 2024

First Posted Date

Aug 23, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 21, 2025

Last Update Posted Date

Aug 28, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

Brief 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).

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

9Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Intervention Names
Cohort 1	Experimental	Drug: GC1130A
Cohort 2	Experimental	Drug: GC1130A
Cohort 3	Experimental	Drug: GC1130A

Interventions

Name	Type	Description	Arm Group Labels	Other Names
GC1130A	Drug	ICV injection	Cohort 1 Cohort 2 Cohort 3

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Incidences and characteristics of adverse events		up to 108 weeks

Secondary Outcomes

Measure	Description	Time Frame
Maximum concentration in cerebrospinal fluid (CSF)	GC1130A PK parameters	up to 104 weeks
Area under the concentration-time curve in CSF	GC1130A PK parameters

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

12 Months

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
GC Biopharma Corp.	Contact	+82312609300		mps3a_clinicaltrial@gccorp.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
UCSF Benioff Children's Hospital	Recruiting	Oakland	California	94609	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D009084	Mucopolysaccharidosis III

Ancestor Terms

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided