Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
6
1
1
2
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
0.0%
0 terminated out of 6 trials
100.0%
+13.5% vs benchmark
33%
2 trials in Phase 3/4
0%
0 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 (6)
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)