Indication
Sanfilippo syndrome
3 clinical trials
3 products
2 drugs
Clinical trial
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIAStatus: Active (not recruiting), Estimated PCD: 2024-06-01
Product
ABO-102Product
Adjuvant ImmunosuppressionClinical trial
A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA DiseaseStatus: Terminated, Estimated PCD: 2022-03-10
Clinical trial
Randomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo SyndromeStatus: Not yet recruiting, Estimated PCD: 2026-01-01
Drug
cannabidiolDrug
Varlilumab