Clinical Research Directory
Browse clinical research sites, groups, and studies.
3 clinical studies listed.
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Tundra lists 3 Sanfilippo A clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT07855562
MPS IIIA Disease Monitoring Program
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Gender: All
Updated: 2026-10-02
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: 2026-09-22
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: 2026-07-17
2 states