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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: Recruiting Phase 1 study of Sanfilippo Syndrome Type A, sponsored by GC Biopharma.
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
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 |
What NCT06567769 shows while recruiting
NCT06567769 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
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 lists 5 locations in 5 states (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.
Learn More About Clinical Trials
Similar trials for Sanfilippo Syndrome Type A
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06567769's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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