231. Alpha-1-antitrypsin deficiency Clinical trials / Disease details
Clinical trials : 89 / Drugs : 89 - (DrugBank : 16) / Drug target genes : 35 - Drug target pathways : 47
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05146882 (ClinicalTrials.gov) | December 2021 | 9/11/2021 | An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) | A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease | Alpha 1-Antitrypsin Deficiency | Drug: Belcesiran | Dicerna Pharmaceuticals, Inc. | NULL | Enrolling by invitation | 18 Years | 75 Years | All | 54 | Phase 2 | New Zealand |
| 2 | NCT04764448 (ClinicalTrials.gov) | February 12, 2021 | 27/1/2021 | A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease | A Phase 2a, Randomized, Double-blind, Placebo Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: belcesiran;Other: Placebo | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | 18 Years | 70 Years | All | 54 | Phase 2 | Australia;New Zealand;Spain;United Kingdom |
| 3 | NCT04167345 (ClinicalTrials.gov) | January 13, 2020 | 15/11/2019 | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects | Alpha 1-Antitrypsin Deficiency | Drug: VX-814;Drug: Placebo | Vertex Pharmaceuticals Incorporated | NULL | Terminated | 18 Years | 80 Years | All | 48 | Phase 2 | United States;Canada;Germany;Ireland |
| 4 | NCT03946449 (ClinicalTrials.gov) | December 19, 2019 | 8/5/2019 | Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection | Arrowhead Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 75 Years | All | 16 | Phase 2 | Austria;Germany;United Kingdom |
| 5 | NCT04204252 (ClinicalTrials.gov) | November 25, 2019 | 17/12/2019 | Evaluate Efficacy and Safety of Kamada-AAT for Inhalation in Patients With AATD | A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation 80 mg Per Day in Adult Patients With Congenital Alpha-1 Antitrypsin Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: Alpha 1-Antitrypsin;Drug: Placebos | Kamada, Ltd. | Syneos Health | Recruiting | 18 Years | 65 Years | All | 220 | Phase 3 | Netherlands |
| 6 | NCT04174118 (ClinicalTrials.gov) | October 24, 2019 | 6/11/2019 | Study of DCR-A1AT in Healthy Adult Volunteers | A Phase 1 Single Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered Belcesiran in Healthy Adult Volunteers | Alpha 1-Antitrypsin Deficiency | Drug: belcesiran;Drug: Placebo | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | 18 Years | 55 Years | All | 36 | Phase 1 | New Zealand;Sweden |
| 7 | NCT03945292 (ClinicalTrials.gov) | August 7, 2019 | 8/5/2019 | Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT | A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA] | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection;Other: Placebo | Arrowhead Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 75 Years | All | 40 | Phase 2 | United States;Germany;Italy;Netherlands;Portugal;Spain;Sweden;Canada;Ireland |
| 8 | NCT03636347 (ClinicalTrials.gov) | October 29, 2018 | 20/6/2018 | A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. | A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 Antitrypsin Deficiency. | Alpha 1-Antitrypsin Deficiency;Emphysema;COPD | Drug: Placebo Oral Tablet;Drug: Alvelestat oral tablet - dose 1;Drug: Alvelestat oral tablet - dose 2 | Mereo BioPharma | Syneos Health | Active, not recruiting | 18 Years | 75 Years | All | 165 | Phase 2 | United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom |
| 9 | NCT03385395 (ClinicalTrials.gov) | July 2018 | 1/12/2017 | Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency | A Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intravenous Administration of OctaAlpha1 (Octapharma) With a Marketed Preparation Glassia® (Kamada Ltd.) in Subjects With Alpha-1-antitrypsin Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: OctaAlpha1;Drug: Glassia | Octapharma | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | NULL |
| 10 | NCT03362242 (ClinicalTrials.gov) | March 12, 2018 | 30/11/2017 | Study of ARO-AAT in Normal Adult Volunteers | A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection;Other: Sterile Normal Saline (0.9% NaCl) | Arrowhead Pharmaceuticals | NULL | Completed | 18 Years | 55 Years | All | 45 | Phase 1 | New Zealand |
| 11 | NCT01669421 (ClinicalTrials.gov) | July 2012 | 14/8/2012 | Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation. | Effect of a Higher Dose of Alpha-1 Antitrypsin Augmentation Therapy on Lung Inflammation in Subjects With Alpha-1 Antitrypsin Deficiency. | Alpha 1 Antitrypsin Deficiency | Drug: Alpha-1 Antitrypsin (human) | Michael Campos, MD | CSL Behring | Completed | 18 Years | 75 Years | All | 10 | Phase 2 | United States |
| 12 | EUCTR2007-004869-18-GB (EUCTR) | 03/07/2008 | 04/04/2008 | Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD | Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD | The medical condition is emphysema and will be investigated in subjects with alpha 1-antitrypsin deficiency. MedDRA version: 9.1;Level: LLT;Classification code 10001811;Term: Alpha-1 proteinase inhibitor deficiency MedDRA version: 9.1;Classification code 10014563;Term: Emphysema pulmonary MedDRA version: 9.1;Classification code 10010952;Term: COPD MedDRA version: 9.1;Classification code 10006458;Term: Bronchitis chronic | Trade Name: Prolastin Product Name: Prolastin INN or Proposed INN: alpha 1-proteinase inhibitor (human) Other descriptive name: ALPHA-1-ANTITRYPSIN | UHB NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 26 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): Therapeutic use (Phase 4): | United Kingdom | ||
| 13 | NCT00460096 (ClinicalTrials.gov) | March 2007 | 12/4/2007 | Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency | Randomized Double-Blind Comparison of an Alpha-1 Proteinase Inhibitor (Kamada API) With a Currently Marketed API Product in Individuals With Alpha-1 Antitrypsin Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: Kamada-API | Kamada, Ltd. | NULL | Completed | 18 Years | N/A | Both | 50 | Phase 2/Phase 3 | United States |
| 14 | NCT00396006 (ClinicalTrials.gov) | October 2006 | 3/11/2006 | Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor) | The Effect of Augmentation Therapy With ARALAST Fraction IV-1 (ARALAST) Alpha1-Proteinase Inhibitor (a1-PI) on the Level of a1-PI and Other Analytes in the Bronchoalveolar (BAL) Epithelial Lining Fluid (ELF) | Alpha 1-Antitrypsin Deficiency | Biological: Alpha1-Proteinase Inhibitor | Baxalta now part of Shire | NULL | Completed | 18 Years | N/A | All | 21 | Phase 4 | Australia;New Zealand |
| 15 | NCT00301366 (ClinicalTrials.gov) | June 2006 | 8/3/2006 | The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency | Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency | Alpha 1-Antitrypsin Deficiency | Drug: alpha-1 proteinase inhibitor (human) | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 38 | Phase 3 | United States;United Kingdom;Netherlands;Poland |
| 16 | NCT00295061 (ClinicalTrials.gov) | May 2006 | 20/2/2006 | Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults | Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency. | Alpha 1-Antitrypsin Deficiency | Drug: Alpha-1 MP;Drug: alpha-1 proteinase inhibitor (human) | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 24 | Phase 3 | United States |
| 17 | NCT00430768 (ClinicalTrials.gov) | February 2006 | 31/1/2007 | Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency | Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults | Alpha 1-Antitrypsin Deficiency | Biological: rAAV1-CB-hAAT | University of Massachusetts, Worcester | National Heart, Lung, and Blood Institute (NHLBI);Applied Genetic Technologies Corp;Alpha-1 Foundation;University of Florida;National Center for Research Resources (NCRR) | Completed | 18 Years | N/A | All | 9 | Phase 1 | United States |
| 18 | NCT00242385 (ClinicalTrials.gov) | December 2005 | 19/10/2005 | Pharmacokinetic Study of ARALAST (Human Alpha1- PI) | Single-Dose, Double-Blind, Crossover Study to Evaluate the Pharmacokinetic Comparability of ARALAST Fraction IV-1 Alpha1-Proteinase Inhibitor (ARALAST Fr. IV-1) and ARALAST | Alpha 1-Antitrypsin Deficiency | Biological: Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor;Biological: Dose of 60 mg/kg alpha1-proteinase inhibitor | Baxalta now part of Shire | Baxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia) | Completed | 18 Years | N/A | All | 25 | Phase 1 | Australia;New Zealand |
| 19 | NCT00263887 (ClinicalTrials.gov) | December 2003 | 12/9/2005 | Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE) | Multi-center, Randomized Trial With I.V. Prolastin® to Evaluate Frequency of Exacerbations and Progression of Emphysema by Means of Multi-slice CT Scans in Patients With Congenital Alpha-1-antitrypsin Deficiency. | Alpha 1-Antitrypsin Deficiency | Drug: Alpha1-Proteinase Inhibitor (Human);Drug: Albumin (Human) 20%, United States Pharmacopeia (USP) | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 77 | Phase 2 | Denmark;Sweden;United Kingdom |
| 20 | NCT00067756 (ClinicalTrials.gov) | November 2001 | 26/8/2003 | 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? | 4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals | Alpha 1-Antitrypsin Deficiency | Drug: 4-PBA | University of Florida | Alpha-1 Foundation;Brantly, Mark L., M.D. | Completed | 18 Years | 65 Years | Both | 12 | Phase 2 | United States |