Amyloidosis
132
29
34
60
Key Insights
Highlights
Success Rate
87% trial completion (above average)
Published Results
20 trials with published results (15%)
Research Maturity
60 completed trials (45% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
6.8%
9 terminated out of 132 trials
87.0%
+0.5% vs benchmark
8%
10 trials in Phase 3/4
33%
20 of 60 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 60 completed trials
Clinical Trials (132)
NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis
A Phase 3 Study of NTLA-2001 in ATTRv-PN
A Study of Daratumumab-Based Therapies in Participants With Amyloid Light Chain (AL) Amyloidosis
Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
Impact of a Cardiac Rehabilitation Program on Patients With Cardiac Amyloidosis
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
A Study to Evaluate the Efficacy and Safety of Belantamab Mafodotin in Combination With Cyclophosphamide, Bortezomib, and Dexamethasone in Adult Participants With Newly Diagnosed Amyloid Light Chain (AL) Amyloidosis
Diagnostic Utility of F-18 Florbetapir PET/MR in Peripheral Nerve Amyloidosis
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
BE.Amycon Biobank & Data Registry UZ Leuven
24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
Phase 1/2a Study of Belantamab Mafodotin in Relapsed or Refractory AL Amyloidosis
Study of Olfactory Disorders in Patients With Cardiac Amyloidosis
A Study to Evaluate the Safety and Efficacy of SCTC21C in Combination With Bortezomib, Cyclophosphamide, and Dexamethasone in Patients With Newly Diagnosed Systemic Light-Chain Amyloidosis (NDSLCA)
Expanded Access to Venetoclax
HIBA-Institutional Registry of Amyloidosis
Institutional Registry of Rare Diseases
Minimal Residual Disease Response-adapted Deferral of Transplant in Dysproteinemia (MILESTONE)