Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
NCT ID: NCT06567769
Last Updated: 2025-08-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
9 participants
INTERVENTIONAL
2024-11-21
2027-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Cohort 1
GC1130A
ICV injection
Cohort 2
GC1130A
ICV injection
Cohort 3
GC1130A
ICV injection
Interventions
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GC1130A
ICV injection
Eligibility Criteria
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Inclusion Criteria
* Participants aged ≥ 12 months and ≤ 18 years
Exclusion Criteria
* 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
12 Months
18 Years
ALL
No
Sponsors
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GC Biopharma Corp
INDUSTRY
Responsible Party
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Locations
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UCSF Benioff Children's Hospital
Oakland, California, United States
University of Minnesota
Minneapolis, Minnesota, United States
National Center for Child Health and Development
Setagaya City, Tokyo, Japan
Ajou University Medical Center
Suwon, Gyeongi-do, South Korea
Samsung Medical Center
Seoul, , South Korea
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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GC1130_MPS3A_P0101
Identifier Type: -
Identifier Source: org_study_id
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