Alpha 1-Antitrypsin Deficiency Clinical Trials

101 clinical trials for Alpha 1-Antitrypsin Deficiency across international registries, including 19 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD) — RECRUITING · PHASE3
• A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD) — RECRUITING · PHASE1
• A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) — RECRUITING · PHASE1
• Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency — RECRUITING
• German Registry for Children and Adolescents with Alpha‑1 Antitrypsin Deficiency (AATD). — RECRUITING
• A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) — RECRUITING · PHASE1
• Gene Therapy for Alpha 1- Antitrypsin Deficiency — RECRUITING · PHASE1
• PiMZ Longitudinal Cohort (PiMZ Logic) — RECRUITING
• Alpha-1 Foundation DNA and Tissue Bank — RECRUITING
• The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study — RECRUITING
• Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs — RECRUITING
• Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction — RECRUITING
• A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency — RECRUITING · PHASE1
• A PHASE 1B/2A OPEN-LABEL SINGLE ASCENDING DOSES (SAD) AND MULTIPLE ASCENDING DOSES (MAD) RESEARCH STUDY TO EVALUATE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS IN PARTICIPANTS WITH AATD PI*ZZ ON WVE-006 (RESTORAATION-2) — RECRUITING · PHASE1
• Alpha-1 Research Registry — RECRUITING
• A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF FAZIRSIRAN IN THE TREATMENT OF ALPHA-1 ANTITRYPSIN DEFICIENCY¿ASSOCIATED LIVER DISEASE WITH METAVIR STAGE F2 TO F4 FIBROSIS — RECRUITING · PHASE3
• Czech AATD Registry — RECRUITING
• A PLACEBO-CONTROLLED, MULTI-DOSE, PHASE 2/3 STUDY TO DETERMINE THE SAFETY, TOLERABILITY AND EFFECT ON LIVER HISTOLOGIC PARAMETERS IN RESPONSE TO ARO-AAT IN PATIENTS WITH ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD) — RECRUITING · PHASE2
• AN OPEN-LABEL, MULTI-DOSE, PHASE 2 STUDY TO DETERMINE THE SAFETY, TOLERABILITY AND EFFECT ON CIRCULATING AND INTRAHEPATIC ALPHA-1 ANTITRYPSIN LEVELS OF ARC-AAT AS EVIDENCED BY CHANGES IN LIVER BIOPSY IN PATIENTS WITH ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD) — RECRUITING · PHASE2
• Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation" — TERMINATED · PHASE3
• A Phase 1 Study of AIR-001 in Adults with AATD. — NOT_YET_RECRUITING · PHASE1
• An Open-label, Multicenter, Randomized, Non-Inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Weekly Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Patients with Alpha1-Antitrypsin Deficiency Compared to Corresponding Standard 60 mg/kg/week and 120 mg/kg/week Doses of Intravenous Alpha1-Proteinase Inhibitor (5%) — TERMINATED · PHASE3
• Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease. — NOT_YET_RECRUITING
• Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) — SUSPENDED
• A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants with AATD Pi*ZZ on WVE-006 (RestorAATion-2) — TERMINATED · PHASE2
• Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants — ACTIVE_NOT_RECRUITING · PHASE1
• Alpha-1 Antitrypsin Deficiency Adult Liver Study — ACTIVE_NOT_RECRUITING
• A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD — ACTIVE_NOT_RECRUITING · PHASE1
• Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema — ACTIVE_NOT_RECRUITING · PHASE2
• A Study of Belcesiran in Patients With AATLD — TERMINATED · PHASE2
• A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) — COMPLETED · PHASE1
• Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease — ACTIVE_NOT_RECRUITING
• A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) — TERMINATED · PHASE1
• Study of ARO-AAT in Normal Adult Volunteers — COMPLETED · PHASE1
• Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels — WITHDRAWN · PHASE2
• Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency — COMPLETED
• Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) — COMPLETED · PHASE2
• Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) — COMPLETED · PHASE2
• Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) — ACTIVE_NOT_RECRUITING · PHASE1
• A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype — TERMINATED · PHASE2
• Patient-related outcome measures (PROM) for use in patients with alpha-1-antitrypsin deficiency (AATD) — OTHER
• Baby Detect : Genomic Newborn Screening — COMPLETED
• Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema — COMPLETED · PHASE2
• Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency — COMPLETED
• Respreeza® Self-administration and Learning Program (AmAREtTI Study) — COMPLETED
• NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease — WITHDRAWN · PHASE1
• Study of DCR-A1AT in Healthy Adult Volunteers — COMPLETED · PHASE1
• Long-Term Follow-up Study of ADVM-043 — COMPLETED
• An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) — WITHDRAWN · PHASE2
• Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency — COMPLETED · PHASE2

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