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New treatment option for Sanfilippo Syndrome Type A
Official title Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
ClinicalTrials.gov ID: NCT06567769
What this study is testing
What is GC1130A?
GC1130A is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for sanfilippo syndrome type a.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 18
You may be able to join if
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 12 months and ≤ 18 years
You likely can't join if
- Participants with significant non-MPS IIIA related central nervous system impairment
- 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...
- Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Minneapolis, Minnesota, United States
- Setagaya City, Tokyo, Japan
- Suwon, Gyeongi-do, South Korea
- Seoul, South Korea
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 months to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Minneapolis, Minnesota, United States; Setagaya City, Tokyo, Japan; Suwon, Gyeongi-do, South Korea; Seoul, South Korea. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.