Apply to trial NCT06567769

A few quick questions so the study team can decide if you might be a fit.

RecruitingPhase 1Drug trial

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).

How this works

  1. Answer a few questions

    About 5 to 10 minutes. Skip-friendly where possible.

  2. We connect you with the study team

    When a site picks up your application, we introduce you to their team directly — with you on the thread.

  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

We save your progress under this email and copy you when we connect you with the study team.

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