Key Insights

Highlights

Success Rate

70% trial completion

Clinical Risk Assessment

Based on trial outcomes

High Risk

Score: 74/100

Termination Rate

23.3%

7 terminated out of 30 trials

Success Rate

69.6%

-16.9% vs benchmark

Late-Stage Pipeline

3%

1 trials in Phase 3/4

Results Transparency

50%

8 of 16 completed with results

Key Signals

8 with results70% success

Data Visualizations

Phase Distribution

16Total
Not Applicable (3)
P 1 (7)
P 2 (5)
P 4 (1)

Trial Status

Completed16
Terminated7
Withdrawn2
Active Not Recruiting2
Recruiting2
Not Yet Recruiting1

Trial Success Rate

69.6%

Benchmark: 86.5%

Based on 16 completed trials

Clinical Trials (30)

Showing 20 of 20 trials
NCT02014415Active Not RecruitingPrimary

Alpha-1 Antitrypsin Deficiency Adult Liver Study

NCT06677307Phase 1TerminatedPrimary

A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110

NCT06405633Phase 1Active Not RecruitingPrimary

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD

NCT07326592Phase 4Not Yet RecruitingPrimary

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)

NCT02363946Phase 1TerminatedPrimary

A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)

NCT02900183Phase 2WithdrawnPrimary

Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels

NCT00500123RecruitingPrimary

The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study

NCT01851642RecruitingPrimary

Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs

NCT05643495Phase 2TerminatedPrimary

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

NCT04262284CompletedPrimary

Respreeza® Self-administration and Learning Program (AmAREtTI Study)

NCT06622668Phase 1Withdrawn

NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

NCT03804021CompletedPrimary

Long-Term Follow-up Study of ADVM-043

NCT02922751Completed

FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

NCT03823443Terminated

COPD Exacerbation Blood and Urine Biomarkers Study

NCT05727800Phase 1CompletedPrimary

A Phase 1, First-in-human Study of VX-668

NCT01810458Completed

Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)

NCT04443192Phase 1TerminatedPrimary

A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects

NCT03815396Phase 1CompletedPrimary

Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency

NCT03008915Phase 2CompletedPrimary

Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency

NCT02001688Phase 2CompletedPrimary

Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"

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