Alpha 1-Antitrypsin Deficiency
46
7
10
24
Key Insights
Highlights
Success Rate
89% trial completion (above average)
Published Results
11 trials with published results (24%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
6.5%
3 terminated out of 46 trials
88.9%
+2.3% vs benchmark
9%
4 trials in Phase 3/4
46%
11 of 24 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 24 completed trials
Clinical Trials (46)
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Gene Therapy for Alpha 1- Antitrypsin Deficiency
PiMZ Longitudinal Cohort (PiMZ Logic)
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Study of ARO-AAT in Normal Adult Volunteers
Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"
Baby Detect : Genomic Newborn Screening
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
A Study of Belcesiran in Patients With AATLD
Study of DCR-A1AT in Healthy Adult Volunteers
An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease
A Phase 1, First-in-human Study of VX-634