Sanfilippo Syndrome
11
4
4
2
Key Insights
Highlights
Success Rate
40% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
27.3%
3 terminated out of 11 trials
40.0%
-46.6% vs benchmark
9%
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: 86.6%
Based on 2 completed trials
Clinical Trials (11)
MPS IIIA Disease Monitoring Program
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Study of Cannabidiol in Sanfilippo Syndrome
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
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