RecruitingPhase 1NCT02254863
Rare condition
Sanfilippo syndrome type A
6
studies recruiting now
as of 7 Sept 2026
48
studies registered in total
as of 7 Sept 2026
6
countries with a recruiting site
as of 7 Sept 2026
23 Aug 2024
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2 / Phase 3NCT02716246
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
RecruitingNCT05825131
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
RecruitingNCT03333200
Longitudinal Study of Neurodegenerative Disorders
RecruitingPhase 1NCT06567769
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 6 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Patient organisations
We do not yet list a dedicated organisation for this condition. The directories below are the best route.
About Sanfilippo syndrome type A
RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the gene involved (SGSH).
Treatments being studied
2 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 2N-Sulfoglucosamine Sulfohydrolase Recombinant
Phase 1/2Sobi-003
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).