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

Part of paid clinical trials in Oakland, California.

Sponsor
GC Biopharma Corp
Study ID
NCT06567769
Phase
PHASE1
Status
Recruiting
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Conditions

  • Sanfilippo Syndrome Type A

Eligibility Criteria

Sex
ALL
Age
12 Months - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • GC1130A — DRUG
    ICV injection

Study Details

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).

Key Dates

Start date
Nov 21, 2024
Status verified
Aug 2025
Primary completion
Jun 30, 2027
Completion
Jun 30, 2027

Study Design

Enrollment
9 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
  • Experimental: Cohort 2
  • Experimental: Cohort 3

Primary Outcome Measure

Incidences and characteristics of adverse events [ Time Frame: up to 108 weeks ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's HospitalOaklandCalifornia94609
Mariam Samara
[email protected]
510-428-3885
Paul Harmatz, MD (PRINCIPAL_INVESTIGATOR)
University of MinnesotaMinneapolisMinnesota55455
Brenda Diethelm-Okita
[email protected]
612-625-1594
Melissa Fetterley
[email protected]
612-672-5151
Chester Whitley, MD (PRINCIPAL_INVESTIGATOR)

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By research site
UCSF Benioff Children's Hospital· Oakland, CAUniversity of Minnesota· Minneapolis, MN