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

a study on Mucopolysaccharidosis Sanfilippo Syndrome

Summary

Eligibility
for people ages 24 months to 72 months (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around
Principal Investigator
by Paul Harmatz, MD
Headshot of Paul Harmatz
Paul Harmatz

Description

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 pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA).

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)

Keywords

Sanfilippo Syndrome Type A, Mucopolysaccharidosis Type III Subtype A, MPS IIIA, Mucopolysaccharidosis III, Syndrome

Eligibility

You can join if…

Open to people ages 24 months to 72 months

  • Participants with documented MPS IIIA diagnosis
  • Participants aged ≥ 24 months and ≤ 72 months

You CAN'T join 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

Locations

  • UCSF Benioff Children's Hospital accepting new patients
    Oakland California 94609 United States
  • Ajou University Medical Center accepting new patients
    Suwon-si Gyeongi-do Korea, Republic of
  • Samsung Medical Center accepting new patients
    Seoul-si Korea, Republic of

Lead Scientist at UCSF

  • Paul Harmatz, MD
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
GC Biopharma Corp
ID
NCT06567769
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 9 study participants
Last Updated
Contact us about this trial