231. Alpha-1-antitrypsin deficiency
[
38 clinical trials,
56 drugs（DrugBank:
17 drugs),
43 target genes / 49 target pathways ]
Searched query = "Alpha 1 antitrypsin deficiency", "AATD"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03946449 | November 15, 2019 | 11 November 2019 | Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AAT | A Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection | Arrowhead Pharmaceuticals | Recruiting | 18 Years | 75 Years | All | 12 | Phase 2 | Austria;Germany;United Kingdom | |
| 2 | NCT03945292 | August 7, 2019 | 22 October 2019 | Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AAT | 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) [SEQUOIA] | Alpha 1-Antitrypsin Deficiency | Drug: ARO-AAT Injection;Other: Placebo | Arrowhead Pharmaceuticals | Recruiting | 18 Years | 75 Years | All | 120 | Phase 2/Phase 3 | United States | |
| 3 | NCT03815396 | July 19, 2019 | 9 September 2019 | Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency | An Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency;AATD | Drug: INBRX-101/rhAAT-Fc | Inhibrx, Inc. | Recruiting | 18 Years | 70 Years | All | 30 | Phase 1 | United States | |
| 4 | NCT03679598 | April 8, 2019 | 24 June 2019 | Alvelestat (MPH996) for the Treatment of ALpha-1 ANTitrypsin Deficiency | A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan Disease | Alpha-1 Antitrypsin Deficiency (AATD);Pi*ZZ, Pi*SZ, or Pi*Null Phenotype;Emphysema or COPD | Drug: Alvelestat (MPH996);Other: Placebo | University of Alabama at Birmingham | National Institutes of Health (NIH);Mereo BioPharma | Recruiting | 25 Years | 80 Years | All | 66 | Phase 2 | United States |
| 5 | NCT03767829 | December 5, 2018 | 11 November 2019 | A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | Drug: ALN-AAT02;Drug: Placebo | Alnylam Pharmaceuticals | Not recruiting | 18 Years | 65 Years | All | 96 | Phase 1/Phase 2 | United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03636347 | October 29, 2018 | 28 October 2019 | 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 (PiZZ or Null) 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 | Recruiting | 18 Years | 75 Years | All | 165 | Phase 2 | United States;Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom |
| 7 | NCT03385395 | July 2018 | 11 June 2018 | 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 | Not recruiting | 18 Years | N/A | All | 0 | Phase 2 | ||
| 8 | EUCTR2015-004110-23-PL | 19/04/2018 | 30 April 2019 | A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency | An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLE | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Pharmaceutical Form: Powder and solvent for solution for infusion INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) CAS Number: 9041-92-3 Current Sponsor code: TAL6004 Other descriptive name: ALPHA-1-ANTITRYPSIN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- | Grifols Therapeutics LLC | Authorised | Female: yes Male: yes | 250 | Phase 3 | United States;Estonia;Canada;Poland;Denmark;Australia;New Zealand;Sweden | |||
| 9 | NCT03362242 | March 12, 2018 | 13 May 2019 | 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 | Not recruiting | 18 Years | 55 Years | All | 45 | Phase 1 | New Zealand | |
| 10 | NCT03172455 | July 25, 2017 | 26 November 2018 | Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) | Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) | Acute-graft-versus-host Disease;Steroid Refractory Acute Graft Versus Host Disease;Graft-versus-host-disease;Graft Vs Host Disease;Alpha 1-Antitrypsin Deficiency;Alpha-1 Proteinase Inhibitor;Alpha-1 Protease Inhibitor Deficiency;Acute Graft-Versus-Host Reaction Following Bone Marrow Transplant | Drug: Glassia | Impatients N.V. trading as myTomorrows | Kamada, Ltd. | Not recruiting | N/A | N/A | All | Phase 2/Phase 3 | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03114020 | March 22, 2017 | 4 November 2019 | Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency | Phase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of Emphysema | Emphysema;Alpha 1-Antitrypsin Deficiency | Drug: Hyaluronic Acid Inhalation Solution;Drug: Placebo Inhalation Solution | Gerard Turino | Recruiting | 18 Years | 80 Years | All | 40 | Phase 2 | United States;Canada | |
| 12 | NCT03008915 | January 2017 | 23 April 2019 | Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency | Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency | Alpha-1 Antitrypsin Deficiency;Emphysema;Chronic Obstructive Pulmonary Disease | Drug: Placebo;Drug: Aspirin | Columbia University | Alpha-1 Foundation;Stony Wold-Herbert Fund, Inc. | Not recruiting | 40 Years | N/A | All | 15 | Phase 2 | United States |
| 13 | NCT02796937 | July 2016 | 9 September 2019 | Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency | An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency | Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency | Biological: Alpha-1 MP | Grifols Therapeutics LLC | Recruiting | 20 Years | 72 Years | All | 250 | Phase 3 | United States;Australia;Canada;Denmark;Estonia;New Zealand;Poland;Sweden | |
| 14 | EUCTR2016-000917-59-IE | 29/06/2016 | 16 January 2017 | A clinical trial to assess the safety and effects of a novel medicine intended for the treatment of patients with liver disease due to Alpha-1 Antitrypsin Deficiency (AATD) | 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) | Alpha-1 Antitrypsin Deficiency related liver disease MedDRA version: 19.1 Level: PT Classification code 10001806 Term: Alpha-1 anti-trypsin deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: ARC-AAT Injection Product Code: ARC-AAT Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: API-AAT Current Sponsor code: API-AAT Other descriptive name: API-AAT, AD00370, ARC-AAT, API drug Substance Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 52- | Arrowhead Pharmaceuticals, Inc | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Canada;Ireland;Sweden | |||
| 15 | EUCTR2015-004110-23-DK | 29/03/2016 | 17 October 2016 | A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency | An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alphal-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLE | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 19.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Pharmaceutical Form: Powder and solvent for solution for infusion INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) CAS Number: 9041-92-3 Current Sponsor code: GTI1201-OLE Other descriptive name: ALPHA-1-ANTITRYPSIN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- | Grifols Therapeutics Inc. | Authorised | Female: yes Male: yes | 250 | Phase 3 | United States;Estonia;Spain;Russian Federation;France;Canada;Argentina;Brazil;Poland;Romania;Denmark;Australia;Germany;New Zealand;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | EUCTR2015-001297-18-GB | 29/06/2015 | 28 February 2019 | The First-in-Human Study of an Investigational Drug, ALN-AAT, in Healthy Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | A Phase 1/2, Randomized, Single-Blind, Placebo-Controlled, Single-Ascending, and Multiple-Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease MedDRA version: 19.1 Level: PT Classification code 10001806 Term: Alpha-1 anti-trypsin deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ALN-AAT Product Code: ALN-AAT Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-61444 Current Sponsor code: ALN-61444 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | Alnylam Pharmaceuticals, Inc. | Not Recruiting | Female: yes Male: yes | 66 | Phase 1;Phase 2 | United States;United Kingdom | |||
| 17 | NCT02363946 | February 2015 | 16 December 2017 | A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) | A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect of Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients With Alpha-1 Antitrypsin Deficiency (AATD) | Alpha-1 Antitrypsin Deficiency | Drug: ARC-AAT Injection;Other: Placebo | Arrowhead Pharmaceuticals | Not recruiting | 18 Years | 70 Years | All | 65 | Phase 1 | Australia;Germany;Netherlands;United Kingdom | |
| 18 | NCT02001688 | April 2014 | 27 June 2016 | Phase II, Safety and ELF Study of Kamada-API for Inhalation | Phase II, Double-blind, Placebo-controlled Study to Explore the ELF and Plasma Concentration as Well as Safety of Inhaled Alpha-1 Antitrypsin in Alpha-1 Antitrypsin Deficiency Subjects | Alpha-1 Antitrypsin Deficiency | Biological: Kamada-API for Inhalation, 80mg;Drug: Placebo;Biological: Kamada-API for Inhalation, 160mg | Kamada, Ltd. | Not recruiting | 18 Years | 65 Years | Both | 24 | Phase 2 | United States | |
| 19 | EUCTR2013-001870-38-SE | 09/01/2014 | 27 January 2014 | A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA | Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 16.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C Pharmaceutical Form: Powder for infusion INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) CAS Number: 9041-92-3 Current Sponsor code: TAL6004 Other descriptive name: ALPHA-1-ANTITRYPSIN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: not less then Concentration number: 47- Pharmaceutical form of the placebo: Solution for infusion Route of administration of the placebo: Intravenous use | Grifols Therapeutics Inc. | Authorised | Female: yes Male: yes | 339 | United States;Estonia;Slovakia;Finland;Spain;Ireland;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;New Zealand;Sweden | ||||
| 20 | NCT01983241 | November 2013 | 9 September 2019 | Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD) | A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency | Pulmonary Emphysema in Alpha-1 PI Deficiency | Biological: Alpha-1 MP;Other: 0.9% Sodium Chloride for Injection, USP | Grifols Therapeutics LLC | Recruiting | 18 Years | 70 Years | All | 339 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;Denmark;Estonia;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT01810458 | October 2013 | 13 May 2019 | Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD) | Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin Deficiency | Liver Fibrosis;Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD | Device: Abdominal ultrasound;Procedure: History and physical;Procedure: Intravenous catheter;Procedure: Blood draw;Other: Liver questionnaire;Procedure: Liver Biopsy;Drug: Midazolam;Drug: Fentanyl;Drug: Lidocaine;Drug: Acetaminophen;Drug: Lorazepam;Drug: Oxycodone/Acetaminophen;Drug: Ondansetron | University of Florida | Not recruiting | 18 Years | 70 Years | All | 109 | Phase 3 | United States | |
| 22 | NCT01669421 | July 2012 | 13 June 2016 | 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 | Not recruiting | 18 Years | 75 Years | Both | 10 | Phase 2 | United States |
| 23 | NCT01379469 | January 2012 | 15 July 2019 | Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency | A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency | Alpha-1-antitrypsin Deficiency;Liver Cirrhosis | Drug: Drug-Carbamazepine (Tegretol XR);Drug: Carbamazepine (Tegretol XR) Placebo | Washington University School of Medicine | Novartis;National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);University of Pittsburgh | Recruiting | 14 Years | 80 Years | All | 30 | Phase 2 | United States |
| 24 | NCT01280994 | January 2011 | 23 April 2019 | Hyperpolarized 129Xe MRI for Imaging Pulmonary Function | Hyperpolarized 129Xe MR Imaging of the Lung Function in Healthy Volunteers and Subjects With Pulmonary Disease | Interstitial Lung Disease;Cystic Fibrosis;Pulmonary Hypertension;NSIP;Alpha 1-Antitrypsin Deficiency | Drug: Xenon | Bastiaan Driehuys | Recruiting | 18 Years | 80 Years | All | 445 | Phase 1 | United States | |
| 25 | NCT01213043 | November 2010 | 19 October 2017 | Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency | A Randomized Double-blind Crossover Study to Assess the Safety and Pharmacokinetics of Two Different Doses of Weekly Intravenous Administration of Alpha1-Proteinase Inhibitor (Human) Prolastin®-C in Subjects With Alpha1-Antitrypsin Deficiency | Emphysema;Alpha 1-antitrypsin Deficiency (AATD) | Biological: Prolastin-C, 60 mg/kg;Biological: Prolastin-C, 120 mg/kg | Grifols Therapeutics Inc. | Not recruiting | 18 Years | 70 Years | All | 30 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT01054339 | June 2010 | 1 April 2019 | Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency | A Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha-1 Antitrypsin (rAAV1-CB-hAAT) in Patients With Alpha-1 Antitrypsin Deficiency | Alpha-1 Antitrypsin Deficiency | Drug: rAAV1-CB-hAAT | Applied Genetic Technologies Corp | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | 18 Years | 75 Years | All | 9 | Phase 2 | United States;Ireland |
| 27 | EUCTR2008-005326-36-GB | 02/11/2009 | 14 July 2014 | A clinical trial looking at the safety and effectiveness of a new drug for inhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients with Emphysema. | A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema | Alpha-1 Antitrypsin deficiency in patients with emphysema. MedDRA version: 17.0 Level: PT Classification code 10001806 Term: Alpha-1 anti-trypsin deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Aerosolized human Alpha-1 Antitrypsin Pharmaceutical Form: Nebuliser solution CAS Number: 8000047054 Other descriptive name: ALPHA-1-ANTITRYPSIN Concentration unit: g/l gram(s)/litre Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Nebuliser solution Route of administration of the placebo: Inhalation use | Kamada Limited | Authorised | Female: yes Male: yes | 200 | Phase 2/3 | Ireland;Denmark;Germany;Netherlands;United Kingdom;Sweden | |||
| 28 | EUCTR2007-004869-18-GB | 03/07/2008 | 10 July 2012 | 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 Level: LLT Classification code 10014563 Term: Emphysema pulmonary MedDRA version: 9.1 Level: LLT Classification code 10010952 Term: COPD MedDRA version: 9.1 Level: LLT Classification code 10006458 Term: Bronchitis chronic | Trade Name: Prolastin Product Name: Prolastin Pharmaceutical Form: Powder and solvent for solution for infusion INN or Proposed INN: alpha 1-proteinase inhibitor (human) CAS Number: 8000047054 Other descriptive name: ALPHA-1-ANTITRYPSIN Concentration unit: mg/l milligram(s)/litre Concentration type: equal | UHB NHS Foundation Trust | Authorised | Female: yes Male: yes | United Kingdom | |||||
| 29 | NCT01851642 | September 2007 | 26 August 2019 | Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs | The Role of Conformational Diseases on Macrophage Function | Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD;Cystic Fibrosis (CF) | Procedure: History and physical exam.;Procedure: Blood draw.;Procedure: Pulmonary function testing.;Drug: Albuterol inhaler. | University of Florida | Recruiting | 18 Years | N/A | All | 220 | Phase 1/Phase 2 | United States | |
| 30 | NCT00460096 | March 2007 | 19 February 2015 | 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. | Not recruiting | 18 Years | N/A | Both | 50 | Phase 2/Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT00396006 | October 2006 | 16 December 2017 | 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 | Not recruiting | 18 Years | N/A | All | 21 | Phase 4 | Australia;New Zealand | |
| 32 | NCT00301366 | June 2006 | 19 October 2017 | 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. | Not recruiting | 18 Years | N/A | All | 38 | Phase 3 | United States;United Kingdom;Netherlands;Poland | |
| 33 | NCT00295061 | May 2006 | 19 October 2017 | 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. | Not recruiting | 18 Years | N/A | All | 24 | Phase 3 | United States | |
| 34 | NCT00430768 | February 2006 | 19 October 2017 | 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) | Not recruiting | 18 Years | N/A | All | 9 | Phase 1 | United States |
| 35 | NCT00242385 | December 2005 | 16 December 2017 | 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 Corporation | Not recruiting | 18 Years | N/A | All | 25 | Phase 1 | Australia;New Zealand |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2004-001688-23-ES | 27/10/2004 | 19 March 2012 | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | A multicentre, double-blind, placebo-controlled randomized trial of the efficacy, safety and tolerability of 12 months of once daily treatment with 1 mg of RO3300074 in patients with symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | symptomatic emphysema secondary to alpha-1-antitrypsin deficiency | Product Name: Not applicable Product Code: RO3300074 Pharmaceutical Form: Capsule, soft INN or Proposed INN: None assigned Other descriptive name: Retinoic Acid Receptor gamma Agonist Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.5- Pharmaceutical form of the placebo: Capsule, soft Route of administration of the placebo: Oral use | F. Hoffmann-La Roche Ltd | Authorised | Female: yes Male: yes | 300 | United Kingdom;Denmark;Spain | ||||
| 37 | NCT00263887 | December 2003 | 19 October 2017 | 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. | Not recruiting | 18 Years | N/A | All | 77 | Phase 2 | Denmark;Sweden;United Kingdom | |
| 38 | NCT00067756 | November 2001 | 19 February 2015 | 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. | Not recruiting | 18 Years | 65 Years | Both | 12 | Phase 2 | United States |