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Table 3 A list of last decade clinical trials testing AAT safety and efficacy in conditions different from severe deficiency AAT according to clinicaltrials.gov, last access 15 july 2018

From: Alpha-1 antitrypsin deficiency as a common treatable mechanism in chronic respiratory disorders and for conditions different from pulmonary emphysema? A commentary on the new European Respiratory Society statement

Study Title Condition Phase Start Date Status
A proof-of-concept pilot trial of AAT for pre-emption of steroid refractory acute GVDH GVDH 2 2018 Recruiting
Multicenter trial of the effect of AAT on Islet Transplant engraftment and durability after Renal Transplant Diabetes Mellitus, Type 1 2 2017 Recruiting
Anti-inflammatory therapy to improve outcomes in patients with Chronic Pancreatitis undergoing Total Pancreatectomy Islet Autotransplantation Chronic Pancreatitis; Diabetes Mellitus, Type 1 4 2016 Recruiting
AAT enhances islet autograft survival Chronic Pancreatitis 1–2 2016 Recruiting
A phase 2/3 clinical study to evaluate the safety and efficacy of AAT as an add-on biopharmacotherapy to conventional steroid treatment in subjects with acute GVDH with lower gastrointestinal involvement GVDH 2–3 2016 Terminated
A proof-of-concept study evaluating the safety and efficacy of human AAT treatment in first Lung Transplantation Transplantation, Lung Rejection 2 2015 Active, not recruiting
Improving single donor success rate in clinical islet using AAT Diabetes Mellitus, Type 1 1–2 2015 Active, not recruiting
A multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of a human plasma-derived AAT in subjects with new-onset Type 1 Diabetes Mellitus Diabetes Mellitus, Type 1 2 2014 Terminated
Single dose administration of AAT for the amelioration of organ injury and post-operative bleeding in patients undergoing cardiac surgery with cariopulmonary bypass Post Cardiac Surgery Systemic Inflammatory Response 1–2 2014 Unknown
Pilot study of AAT: a novel treatment to mitigate Neuromyelitis Optica attacks Neuromyelitis Optica 1 2014 Unknown
Long-term treatment safety, tolerability and efficacy of AAT in Type 1 Diabetes Diabetes Mellitus, Type 1 2 2013 Completed
AAT to quench the acute inflammatory response in ST-segment elevation Acute Myocardial Infarction Acute Myocardial Infarction 1–2 2013 Completed
Phase 2 study to evaluate the efficacy and safety of human AAT in the treatment of new onset Type 1 Diabetes Diabetes Mellitus, Type 1 2 2013 Completed
Effects of Prolastin aerosol therapy on bacterial density in the airways of patients with CF Cystic Fibrosis 2 2013 Terminated
AAT in treating patients with acute GVDH GVDH 1–2 2012 Completed
Safety and tolerability of inhaled AAT once-a-day in patients with Cystic Fibrosis Cystic Fibrosis 2 2012 Completed
Safety and efficacy of AAT in HIV Disease HIV Disease 2–3 2012 Terminated
A pilot study of AAT in steroid refractory acute GVDH GVDH 2 2012 Active, not recruiting
The effects of AAT on the progression of Type-1 Diabetes in subjects with detectable C-peptide Diabetes Mellitus, Type 1 1 2011 Completed
Proof-of-concept study of the safety, tolerability and efficacy of intravenous AAT in Type 1 Diabetes Diabetes Mellitus, Type 1 1–2 2011 Completed
Safety and efficacy of inhaled AAT in preventing Bronchiolitis Obliterable Syndrome in Lung Transplant recipients Transplantation, Lung Rejection 2 2011 Unknown
Safety and tolerability study of AAT inhalation solution in patients with Cystic Fibrosis Cystic Fibrosis 1 2011 Completed
Effects of intravenous AAT on preserving beta-cell function in new-onset Type 1 Diabetes Mellitus Diabetes Mellitus, Type 1 2 2010 Withdrawn
  1. AAT alpha-1 antitrypsin, CF cystic fibrosis GVDH graft-versus-host disease, HIV human immunodeficiency virus