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

Description

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).

Conditions

Sanfilippo Syndrome Type A

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Study Overview

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Study Details

Study overview

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).

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)

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

Condition
Sanfilippo Syndrome Type A
Intervention / Treatment

-

Contacts and Locations

Oakland

UCSF Benioff Children's Hospital, Oakland, California, United States, 94609

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

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

Ages Eligible for Study

24 Months to 72 Months

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

GC Biopharma Corp,

Study Record Dates

2027-06