Alpha-1 Antitrypsin Deficiency

Top drugs studied for this disease

All Approvals Safety alerts Label changes Trial results Research Guidance Withdrawals

CLINICAL_TRIALS withdrawal · 2025-12-18
Clinical Trial Update: Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels [Phase 2]
Phase 2 study ARC-AAT for Alpha-1 Antitrypsin Deficiency was withdrawn; the trial evaluated safety, tolerability, and effects on circulating…
CLINICAL_TRIALS withdrawal · 2025-01-17
Clinical Trial Update: NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease [Phase 1, Phase 2]
A Phase 1/2 trial of NTLA-3001 for Alpha-1 Antitrypsin Deficiency–associated lung disease has been withdrawn.
CLINICAL_TRIALS withdrawal · 2024-09-19
Clinical Trial Update: An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) [Phase 2]
The Phase 2 extension study of belcesiran for Alpha-1 Antitrypsin Deficiency-associated liver disease has been withdrawn. Sponsor: Dicerna P…
CLINICAL_TRIALS withdrawal · 2023-05-06
Clinical Trial Update: A Study of RYMPHYSIA for Alpha1-Proteinase Inhibitor (A1PI) Therapy in Adults With A1PI Deficiency and Chronic Obstructive Pulmonary Disease (COPD)-Emphysema [Phase 4]
Phase 4 trial of RYMPHYSIA for A1PI deficiency in adults with COPD-emphysema has been withdrawn.
CLINICAL_TRIALS withdrawal · 2020-09-14
Clinical Trial Update: ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study [Phase 4]
A Phase 4 clinical trial for ARALAST NP in COPD with Alpha1-antitrypsin deficiency has been withdrawn.
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CLINICAL_TRIALS withdrawal · 2018-04-10
Clinical Trial Update: Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency [Phase 2]
A Phase 2 trial comparing OctaAlpha1 to Glassia for Alpha-1 antitrypsin deficiency has been withdrawn.
CLINICAL_TRIALS withdrawal · 2017-05-02
Clinical Trial Update: Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency [NA]
A ClinicalTrials.gov study on Lung Volume Reduction Coils for emphysema in Alpha-1 antitrypsin deficiency has been withdrawn.