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NCT06567769 · ClinicalTrials.gov registry record · Phase 1
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
A Phase 1 study of Sanfilippo Syndrome Type A, sponsored by GC Biopharma.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
- 5
- Study locations
NCT06567769 is a Phase 1 study of Sanfilippo Syndrome Type A that is actively recruiting participants, run by GC Biopharma. The registered enrollment target is 9 participants. The trial reports 5 study locations across 5 states.
The verdict
NCT06567769, a Phase 1 study of Sanfilippo Syndrome Type A, is actively recruiting participants, sponsored by GC Biopharma.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
- 5
- Study locations
Study 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 patients with Sanfilippo Syndrome Type A (MPS IIIA).
Conditions Studied
Interventions
- DRUG GC1130A
Study Locations (5)
California
- UCSF Benioff Children's Hospital - Oakland
Minnesota
- University of Minnesota - Minneapolis
Tokyo
- National Center for Child Health and Development - Setagaya City
Gyeongi-do
- Ajou University Medical Center - Suwon
Other
- Samsung Medical Center - Seoul
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2024-11-21 |
| Est. Completion | 2027-06 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06567769
The ClinicalTrials.gov registry entry for NCT06567769 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is GC Biopharma, which has 3 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Sanfilippo Syndrome Type A appearing as the primary indexed condition, and to 1 intervention - of which GC1130A is the first listed.
NCT06567769 reports 5 study locations spanning 5 distinct geographic areas - top geographies include California, Minnesota, Tokyo.
Frequently Asked Questions
What is clinical trial NCT06567769 about?
NCT06567769 is a clinical study titled "Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)". 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).
What is the current status of trial NCT06567769?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2024-11-21. Estimated completion is 2027-06.
What conditions does trial NCT06567769 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome Type A.
What interventions are being tested in trial NCT06567769?
The interventions under investigation include: GC1130A (DRUG).
Who is sponsoring clinical trial NCT06567769?
This trial is sponsored by GC Biopharma, which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06567769 being conducted?
This trial has 5 study locations across California, Minnesota, Tokyo, Gyeongi-do. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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