Sanfilippo Syndrome
10
3
3
2
Key Insights
Highlights
Success Rate
40% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
30.0%
3 terminated out of 10 trials
40.0%
-46.5% vs benchmark
10%
1 trials in Phase 3/4
250%
5 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (10)
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Study of Cannabidiol in Sanfilippo Syndrome
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
The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
HSCT for High Risk Inherited Inborn Errors