RECRUITING

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

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Conditions

Sanfilippo Syndrome Type AMucopolysaccharidosis Type III Subtype AMPS IIIA

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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 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)

Quick Facts

Study Start:2024-11-21
Study Completion:2027-06
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06567769

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.

Ages Eligible for Study:12 Months to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Participants with documented MPS IIIA diagnosis
  2. * Participants aged ≥ 12 months and ≤ 18 years
  1. * Participants with significant non-MPS IIIA related central nervous system impairment
  2. * Participants with previous complication from intraventricular drug administration
  3. * Participants with contraindications for MRI scans and for neurosurgery
  4. * 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
  5. * Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy

Contacts and Locations

Study Contact

GC Biopharma Corp.
CONTACT
+82312609300
mps3a_clinicaltrial@gccorp.com

Study Locations (Sites)

UCSF Benioff Children's Hospital
Oakland, California, 94609
United States
University of Minnesota
Minneapolis, Minnesota, 55455
United States

Collaborators and Investigators

Sponsor: GC Biopharma Corp

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-11-21
Study Completion Date2027-06

Study Record Updates

Study Start Date2024-11-21
Study Completion Date2027-06

Terms related to this study

Keywords Provided by Researchers

  • Mucopolysaccharidosis Type III Subtype A
  • MPS IIIA

Additional Relevant MeSH Terms

  • Sanfilippo Syndrome Type A