There are currently 3 active clinical trials seeking participants for Sanfilippo Syndrome research studies. The states with the highest number of trials for Sanfilippo Syndrome participants are Minnesota, South Australia, California and North Carolina.
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Recruiting
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Gender:
ALL
Ages:
All
Trial Updated:
07/16/2025
Locations: Nationwide Children's Hospital, Columbus, Ohio +4 locations
Conditions: MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III
Recruiting
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
Gender:
ALL
Ages:
Between 18 years and 99 years
Trial Updated:
08/18/2025
Locations: Lysosomal & Rare Disorders Research & Treatment Center, Inc., Fairfax, Virginia
Conditions: Sanfilippo Syndrome, MPS3
The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
Recruiting
The natural history study of patients with Sanfilippo disease(s) (MPS3)
Gender:
ALL
Ages:
Between 5 years and 99 years
Trial Updated:
03/07/2024
Locations: LDRTC, Fairfax, Virginia
Conditions: Sanfilippo Syndrome, MPS3