Alpha-1 Antitrypsin Deficiency
30
2
4
16
Key Insights
Highlights
Success Rate
70% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 74/100
23.3%
7 terminated out of 30 trials
69.6%
-16.9% vs benchmark
3%
1 trials in Phase 3/4
50%
8 of 16 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 16 completed trials
Clinical Trials (30)
Alpha-1 Antitrypsin Deficiency Adult Liver Study
A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
Respreeza® Self-administration and Learning Program (AmAREtTI Study)
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
Long-Term Follow-up Study of ADVM-043
FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
COPD Exacerbation Blood and Urine Biomarkers Study
A Phase 1, First-in-human Study of VX-668
Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"