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NCT Number: NCT06567769

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

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).

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Key information

Conditions

Sanfilippo Syndrome Type A Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis III Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

12 month–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

National Center for Child Health and Development, Setagaya City, Tokyo, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

GC1130A

Drug

ICV injection

Primary outcomes

  1. Incidences and characteristics of adverse events

    Time frame: up to 108 weeks

Secondary outcomes

  1. Maximum concentration in cerebrospinal fluid (CSF)

    Time frame: up to 104 weeks

    GC1130A PK parameters

  2. Area under the concentration-time curve in CSF

    Time frame: up to 104 weeks

    GC1130A PK parameters

  3. Maximum concentration in serum

    Time frame: up to 104 weeks

    GC1130A PK parameters

  4. Area under the concentration-time curve in serum

    Time frame: up to 104 weeks

    GC1130A PK parameters

  5. Change from baseline in CSF heparan sulfate concentration

    Time frame: up to 104 weeks

  6. Change from baseline in serum heparan sulfate concentration

    Time frame: up to 104 weeks

  7. Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF

    Time frame: up to 104 weeks

  8. Incidence of anti-drug and neutralizing antibodies of GC1130A in serum

    Time frame: up to 104 weeks

  9. Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)

    Time frame: up to 104 weeks

Study contacts

Contact information is provided by the study sponsor or research team.

GC Biopharma Corp.

CONTACT

[email protected]

+82312609300

Sponsors and collaborators

Lead sponsor

GC Biopharma Corp

Industry

Registry information

Official study 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)

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Aug 23, 2024
Registry last updated
Aug 28, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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