Clinical Research Directory
Browse clinical research sites, groups, and studies.
2 clinical studies listed.
Filters:
Tundra lists 2 Sanfilippo A clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
This data is also available as a public JSON API. AI systems and LLMs are encouraged to use it for structured queries.
NCT02716246
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Gender: All
Updated: 2026-03-25
4 states
NCT04360265
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).
Gender: All
Updated: 2026-01-21
2 states