NCT06567769

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).

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
13mo left

Started Nov 2024

Typical duration for phase_1

Geographic Reach
3 countries

5 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress58%
Nov 2024Jun 2027

First Submitted

Initial submission to the registry

August 20, 2024

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 23, 2024

Completed
3 months until next milestone

Study Start

First participant enrolled

November 21, 2024

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2027

Last Updated

August 28, 2025

Status Verified

August 1, 2025

Enrollment Period

2.5 years

First QC Date

August 20, 2024

Last Update Submit

August 21, 2025

Conditions

Sanfilippo Syndrome Type A

Keywords

Mucopolysaccharidosis Type III Subtype AMPS IIIA

Outcome Measures

Primary Outcomes (1)

  • Incidences and characteristics of adverse events

    up to 108 weeks

Secondary Outcomes (9)

  • Maximum concentration in cerebrospinal fluid (CSF)

    up to 104 weeks

  • Area under the concentration-time curve in CSF

    up to 104 weeks

  • Maximum concentration in serum

    up to 104 weeks

  • Area under the concentration-time curve in serum

    up to 104 weeks

  • Change from baseline in CSF heparan sulfate concentration

    up to 104 weeks

  • +4 more secondary outcomes

Study Arms (3)

Cohort 1

EXPERIMENTAL
Drug: GC1130A

Cohort 2

EXPERIMENTAL
Drug: GC1130A

Cohort 3

EXPERIMENTAL
Drug: GC1130A

Interventions

GC1130ADRUG

ICV injection

Cohort 1Cohort 2Cohort 3

Eligibility Criteria

Age12 Months - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Participants with documented MPS IIIA diagnosis
  • Participants aged ≥ 12 months and ≤ 18 years

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

National Center for Child Health and Development

Setagaya City, Tokyo, Japan

RECRUITING

Ajou University Medical Center

Suwon, Gyeongi-do, South Korea

RECRUITING

Samsung Medical Center

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Mucopolysaccharidosis III

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

GC Biopharma Corp.

CONTACT

+82312609300mps3a_clinicaltrial@gccorp.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 20, 2024

First Posted

August 23, 2024

Study Start

November 21, 2024

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

June 1, 2027

Last Updated

August 28, 2025

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will not share

Locations

US(2)KR(2)JP(1)