231. α1−アンチトリプシン欠乏症 Disease details / Clinical trials / Drug dev / DR info
臨床試験数 : 93 / 薬物数 : 77 - (DrugBank : 8) / 標的遺伝子数 : 10 - 標的パスウェイ数 : 36
薬物ごとの開発者（Primary Sponsor）、臨床試験情報（抜粋）です。
(4S)-4-[4-cyano-2-(methylsulfonyl)phenyl]-3,6-dimethyl-2-oxo-1-[3-(trifluoromethyl)phenyl]-1,2,3,4-tetrahydropyrimidine-5-carbonitrile
pH Pharma Inc.
2020 Phase 2 EUCTR2019-002501-22-GB United Kingdom;
4-PBA
University of Florida
2001 Phase 2 NCT00067756 United States;
ADS-001
Arrowhead Pharmaceuticals, Inc.
2021 Phase 2;Phase 3 EUCTR2018-003385-14-AT Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;Switzerland;United Kingdom;United States;
2021 Phase 2 EUCTR2018-003385-14-DE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2;Phase 3 EUCTR2018-003385-14-GB Canada;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003385-14-SE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-IE Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-ES Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United States;
2019 Phase 2 EUCTR2019-000068-86-GB Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2019-000068-86-AT Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2018-003385-14-PT Austria;Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003385-14-NL Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
- Phase 2 EUCTR2019-000068-86-DE Austria;Germany;United Kingdom;
ALN-61444
Alnylam Pharmaceuticals, Inc.
2015 Phase 1;Phase 2 EUCTR2015-001297-18-GB United Kingdom;United States;
ALN-77412
Alnylam Pharmaceuticals, Inc.
2018 Phase 1;Phase 2 EUCTR2018-001362-41-GB Germany;United Kingdom;United States;
ALN-AAT
Alnylam Pharmaceuticals, Inc.
2015 Phase 1;Phase 2 EUCTR2015-001297-18-GB United Kingdom;United States;
ALN-AAT02
Alnylam Pharmaceuticals
2018 Phase 1/Phase 2 NCT03767829 United Kingdom;
Alnylam Pharmaceuticals, Inc.
2018 Phase 1;Phase 2 EUCTR2018-001362-41-GB Germany;United Kingdom;United States;
API-AAT, AD00370, ARC-AAT, API drug Substance
Arrowhead Pharmaceuticals Inc
2016 Phase 2 EUCTR2016-000917-59-SE Canada;Ireland;Sweden;
Arrowhead Pharmaceuticals, Inc
2016 Phase 2 EUCTR2016-000917-59-IE Canada;Ireland;Sweden;
ARC-AAT Injection
Arrowhead Pharmaceuticals
2016 Phase 2 NCT02900183 Canada;Ireland;Italy;Sweden;
2015 Phase 1 NCT02363946 Australia;Germany;Netherlands;United Kingdom;
Arrowhead Pharmaceuticals Inc
2016 Phase 2 EUCTR2016-000917-59-SE Canada;Ireland;Sweden;
Arrowhead Pharmaceuticals, Inc
2016 Phase 2 EUCTR2016-000917-59-IE Canada;Ireland;Sweden;
ARO-AAT Injection
ARROWHEAD PHARMACEUTICALS
2019 Phase 2 EUCTR2018-003385-14-IT Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
Arrowhead Pharmaceuticals
2019 Phase 2 NCT03946449 Austria;Germany;United Kingdom;
2019 Phase 2 NCT03945292 Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United States;
2018 Phase 1 NCT03362242 New Zealand;
Arrowhead Pharmaceuticals, Inc.
2021 Phase 2;Phase 3 EUCTR2018-003385-14-AT Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;Switzerland;United Kingdom;United States;
2021 Phase 2 EUCTR2018-003385-14-DE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2;Phase 3 EUCTR2018-003385-14-GB Canada;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003385-14-SE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-IE Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-ES Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United States;
2019 Phase 2 EUCTR2019-000068-86-GB Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2019-000068-86-AT Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2018-003385-14-PT Austria;Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003385-14-NL Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
- Phase 2 EUCTR2019-000068-86-DE Austria;Germany;United Kingdom;
Abdominal ultrasound
University of Florida
2013 - NCT01810458 United States;
Aerosolized human Alpha-1 Antitrypsin
Kamada Limited
2011 - EUCTR2008-005326-36-SE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 Phase 2;Phase 3 EUCTR2008-005326-36-DK Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-NL Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-IE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-DE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2009 Phase 2;Phase 3 EUCTR2008-005326-36-GB Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
Albumin (Human) , United States Pharmacopeia (USP)
Grifols Therapeutics Inc.
2003 Phase 2 NCT00263887 Denmark;Sweden;United Kingdom;
Albuterol inhaler.
University of Florida
2007 - NCT01851642 United States;
Alpha 1-proteinase inhibitor (human)
UHB NHS Foundation Trust
2008 Phase 2 EUCTR2007-004869-18-GB United Kingdom;
Alpha-1 Antitrypsin (AAT)
Kamada Ltd.
2020 Phase 3 EUCTR2019-000602-30-SE Argentina;Belgium;Finland;Ireland;Italy;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-GB Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-FI Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2019 Phase 3 EUCTR2019-000602-30-NL Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
Alpha-1 Antitrypsin (human)
Michael Campos, MD
2012 Phase 2 NCT01669421 United States;
Alpha-1 MP
Grifols Therapeutics Inc.
2006 Phase 3 NCT00295061 United States;
Grifols Therapeutics LLC
2016 Phase 3 NCT02796937 Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden;United States;
Alpha-1 proteinase inhibitor (human)
Grifols Therapeutics Inc.
2006 Phase 3 NCT00301366 Netherlands;Poland;United Kingdom;United States;
2006 Phase 3 NCT00295061 United States;
Alpha1-Proteinase Inhibitor (Human)
Grifols Therapeutics Inc.
2015 Phase 3 EUCTR2013-001870-38-DE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-IE Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
2014 - EUCTR2013-001870-38-PL Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States;
2013 Phase 3 EUCTR2013-001870-38-ES Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
2003 Phase 2 NCT00263887 Denmark;Sweden;United Kingdom;
Grifols Therapeutics LLC
2018 Phase 3 EUCTR2015-004110-23-PL Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden;United States;
2016 Phase 3 EUCTR2015-004110-23-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-SE Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2014 Phase 3 EUCTR2013-001870-38-FI Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-EE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
Alvelestat (MPH966)
University of Alabama at Birmingham
2019 Phase 2 NCT03679598 United States;
Alvelestat oral tablet - dose 1
Mereo BioPharma
2018 Phase 2 NCT03636347 Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Alvelestat oral tablet - dose 2
Mereo BioPharma
2018 Phase 2 NCT03636347 Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Alvelestat tosylate
Mereo BioPharma 4 Ltd
2019 Phase 2 EUCTR2018-001309-95-PL Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-001309-95-BE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-SE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-GB Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-DK Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Aspirin
Columbia University
2017 Phase 2 NCT03008915 United States;
Belcesiran
Dicerna Pharmaceuticals, Inc.
2021 Phase 2 NCT05146882 New Zealand;
2021 Phase 2 NCT04764448 Australia;New Zealand;Spain;United Kingdom;
2019 Phase 1 NCT04174118 New Zealand;Sweden;
Blood draw
University of Florida
2013 - NCT01810458 United States;
Blood draw.
University of Florida
2007 - NCT01851642 United States;
Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor
Baxalta now part of Shire
2005 Phase 1 NCT00242385 Australia;New Zealand;
Dose of 60 mg/kg alpha1-proteinase inhibitor
Baxalta now part of Shire
2005 Phase 1 NCT00242385 Australia;New Zealand;
Drug-Carbamazepine (Tegretol XR)
Washington University School of Medicine
2012 Phase 2 NCT01379469 United States;
Fentanyl
University of Florida
2013 - NCT01810458 United States;
Glassia
Impatients N.V. trading as myTomorrows
2017 - NCT03172455 -
Octapharma
2018 Phase 2 NCT03385395 -
History and physical
University of Florida
2013 - NCT01810458 United States;
History and physical exam.
University of Florida
2007 - NCT01851642 United States;
Hyaluronic Acid Inhalation Solution
Gerard Turino
2017 Phase 2 NCT03114020 Canada;United States;
INBRX-101/rhAAT-Fc
Inhibrx, Inc.
2019 Phase 1 NCT03815396 New Zealand;United Kingdom;United States;
Intravenous catheter
University of Florida
2013 - NCT01810458 United States;
Kamada -AAT for Inhalation or inhaled AAT (AAT)
Kamada Ltd.
2020 Phase 3 EUCTR2019-000602-30-SE Argentina;Belgium;Finland;Ireland;Italy;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-GB Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-FI Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2019 Phase 3 EUCTR2019-000602-30-NL Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
Kamada-AAT for Inhalation,
Kamada, Ltd.
2014 Phase 2 NCT02001688 United States;
Kamada-AAT for inhalation
Kamada Limited
2011 - EUCTR2008-005326-36-SE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 Phase 2;Phase 3 EUCTR2008-005326-36-DK Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-NL Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-IE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-DE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
Kamada-API
Kamada, Ltd.
2007 Phase 2/Phase 3 NCT00460096 United States;
Lidocaine
University of Florida
2013 - NCT01810458 United States;
Liver Biopsy
University of Florida
2013 - NCT01810458 United States;
Lorazepam
University of Florida
2013 - NCT01810458 United States;
MPH966
Mereo BioPharma 4 Ltd
2019 Phase 2 EUCTR2018-001309-95-PL Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-001309-95-BE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-SE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-GB Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-DK Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Midazolam
University of Florida
2013 - NCT01810458 United States;
None assigned
F. Hoffmann-La Roche Ltd
2008 Phase 2 EUCTR2004-001688-23-GB Denmark;Spain;United Kingdom;
2006 - EUCTR2004-001688-23-DK Denmark;Spain;United Kingdom;
2004 - EUCTR2004-001688-23-ES Denmark;Spain;United Kingdom;
OctaAlpha1
Octapharma
2018 Phase 2 NCT03385395 -
Ondansetron
University of Florida
2013 - NCT01810458 United States;
Oxycodone/Acetaminophen
University of Florida
2013 - NCT01810458 United States;
PHP-303
pH Pharma Inc.
2020 Phase 2 EUCTR2019-002501-22-GB United Kingdom;
Prolastin
UHB NHS Foundation Trust
2008 Phase 2 EUCTR2007-004869-18-GB United Kingdom;
Prolastin-C
Grifols Therapeutics Inc.
2015 Phase 3 EUCTR2013-001870-38-DE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-IE Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
2014 - EUCTR2013-001870-38-PL Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States;
2013 Phase 3 EUCTR2013-001870-38-ES Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
Grifols Therapeutics LLC
2018 Phase 3 EUCTR2015-004110-23-PL Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden;United States;
2016 Phase 3 EUCTR2015-004110-23-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-SE Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2014 Phase 3 EUCTR2013-001870-38-FI Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-EE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
Prolastin-C,
Grifols Therapeutics Inc.
2010 Phase 2 NCT01213043 United States;
Pulmonary function testing.
University of Florida
2007 - NCT01851642 United States;
RAAV1-CB-hAAT
Applied Genetic Technologies Corp
2010 Phase 2 NCT01054339 Ireland;United States;
Applied Genetic Technologies Corporation
2010 Phase 2 EUCTR2009-014286-20-IE Ireland;
University of Massachusetts, Worcester
2006 Phase 1 NCT00430768 United States;
RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING THE HUMAN ALPHA-1-ANTITRYPSIN GENE
Kamada Ltd.
2020 Phase 3 EUCTR2019-000602-30-SE Argentina;Belgium;Finland;Ireland;Italy;Netherlands;Sweden;United Kingdom;
2019 Phase 3 EUCTR2019-000602-30-NL Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
RO3300074
F. Hoffmann-La Roche Ltd
2008 Phase 2 EUCTR2004-001688-23-GB Denmark;Spain;United Kingdom;
2006 - EUCTR2004-001688-23-DK Denmark;Spain;United Kingdom;
2004 - EUCTR2004-001688-23-ES Denmark;Spain;United Kingdom;
Retinoic Acid Receptor gamma Agonist
F. Hoffmann-La Roche Ltd
2008 Phase 2 EUCTR2004-001688-23-GB Denmark;Spain;United Kingdom;
2006 - EUCTR2004-001688-23-DK Denmark;Spain;United Kingdom;
2004 - EUCTR2004-001688-23-ES Denmark;Spain;United Kingdom;
VX-814
Vertex Pharmaceuticals Incorporated
2020 Phase 2 NCT04167345 Canada;Germany;Ireland;United States;
2020 Phase 2 EUCTR2019-003650-92-SE Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-003650-92-IE Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-003650-92-GB Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-003650-92-DE Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
VX-864
Vertex Pharmaceuticals Incorporated
2020 Phase 2 EUCTR2019-004881-16-SE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-IE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-GB Canada;Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-DE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
VX864
Vertex Pharmaceuticals Incorporated
2020 Phase 2 EUCTR2019-004881-16-SE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-IE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-GB Canada;Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-DE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
pH Pharma Inc.
2020 Phase 2 EUCTR2019-002501-22-GB United Kingdom;
4-PBA
University of Florida
2001 Phase 2 NCT00067756 United States;
ADS-001
Arrowhead Pharmaceuticals, Inc.
2021 Phase 2;Phase 3 EUCTR2018-003385-14-AT Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;Switzerland;United Kingdom;United States;
2021 Phase 2 EUCTR2018-003385-14-DE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2;Phase 3 EUCTR2018-003385-14-GB Canada;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003385-14-SE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-IE Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-ES Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United States;
2019 Phase 2 EUCTR2019-000068-86-GB Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2019-000068-86-AT Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2018-003385-14-PT Austria;Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003385-14-NL Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
- Phase 2 EUCTR2019-000068-86-DE Austria;Germany;United Kingdom;
ALN-61444
Alnylam Pharmaceuticals, Inc.
2015 Phase 1;Phase 2 EUCTR2015-001297-18-GB United Kingdom;United States;
ALN-77412
Alnylam Pharmaceuticals, Inc.
2018 Phase 1;Phase 2 EUCTR2018-001362-41-GB Germany;United Kingdom;United States;
ALN-AAT
Alnylam Pharmaceuticals, Inc.
2015 Phase 1;Phase 2 EUCTR2015-001297-18-GB United Kingdom;United States;
ALN-AAT02
Alnylam Pharmaceuticals
2018 Phase 1/Phase 2 NCT03767829 United Kingdom;
Alnylam Pharmaceuticals, Inc.
2018 Phase 1;Phase 2 EUCTR2018-001362-41-GB Germany;United Kingdom;United States;
API-AAT, AD00370, ARC-AAT, API drug Substance
Arrowhead Pharmaceuticals Inc
2016 Phase 2 EUCTR2016-000917-59-SE Canada;Ireland;Sweden;
Arrowhead Pharmaceuticals, Inc
2016 Phase 2 EUCTR2016-000917-59-IE Canada;Ireland;Sweden;
ARC-AAT Injection
Arrowhead Pharmaceuticals
2016 Phase 2 NCT02900183 Canada;Ireland;Italy;Sweden;
2015 Phase 1 NCT02363946 Australia;Germany;Netherlands;United Kingdom;
Arrowhead Pharmaceuticals Inc
2016 Phase 2 EUCTR2016-000917-59-SE Canada;Ireland;Sweden;
Arrowhead Pharmaceuticals, Inc
2016 Phase 2 EUCTR2016-000917-59-IE Canada;Ireland;Sweden;
ARO-AAT Injection
ARROWHEAD PHARMACEUTICALS
2019 Phase 2 EUCTR2018-003385-14-IT Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
Arrowhead Pharmaceuticals
2019 Phase 2 NCT03946449 Austria;Germany;United Kingdom;
2019 Phase 2 NCT03945292 Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United States;
2018 Phase 1 NCT03362242 New Zealand;
Arrowhead Pharmaceuticals, Inc.
2021 Phase 2;Phase 3 EUCTR2018-003385-14-AT Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;Switzerland;United Kingdom;United States;
2021 Phase 2 EUCTR2018-003385-14-DE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2;Phase 3 EUCTR2018-003385-14-GB Canada;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003385-14-SE Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-IE Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-003385-14-ES Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United States;
2019 Phase 2 EUCTR2019-000068-86-GB Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2019-000068-86-AT Austria;Germany;United Kingdom;
2019 Phase 2 EUCTR2018-003385-14-PT Austria;Canada;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003385-14-NL Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States;
- Phase 2 EUCTR2019-000068-86-DE Austria;Germany;United Kingdom;
Abdominal ultrasound
University of Florida
2013 - NCT01810458 United States;
Aerosolized human Alpha-1 Antitrypsin
Kamada Limited
2011 - EUCTR2008-005326-36-SE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 Phase 2;Phase 3 EUCTR2008-005326-36-DK Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-NL Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-IE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-DE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2009 Phase 2;Phase 3 EUCTR2008-005326-36-GB Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
Albumin (Human) , United States Pharmacopeia (USP)
Grifols Therapeutics Inc.
2003 Phase 2 NCT00263887 Denmark;Sweden;United Kingdom;
Albuterol inhaler.
University of Florida
2007 - NCT01851642 United States;
Alpha 1-proteinase inhibitor (human)
UHB NHS Foundation Trust
2008 Phase 2 EUCTR2007-004869-18-GB United Kingdom;
Alpha-1 Antitrypsin (AAT)
Kamada Ltd.
2020 Phase 3 EUCTR2019-000602-30-SE Argentina;Belgium;Finland;Ireland;Italy;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-GB Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-FI Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2019 Phase 3 EUCTR2019-000602-30-NL Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
Alpha-1 Antitrypsin (human)
Michael Campos, MD
2012 Phase 2 NCT01669421 United States;
Alpha-1 MP
Grifols Therapeutics Inc.
2006 Phase 3 NCT00295061 United States;
Grifols Therapeutics LLC
2016 Phase 3 NCT02796937 Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden;United States;
Alpha-1 proteinase inhibitor (human)
Grifols Therapeutics Inc.
2006 Phase 3 NCT00301366 Netherlands;Poland;United Kingdom;United States;
2006 Phase 3 NCT00295061 United States;
Alpha1-Proteinase Inhibitor (Human)
Grifols Therapeutics Inc.
2015 Phase 3 EUCTR2013-001870-38-DE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-IE Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
2014 - EUCTR2013-001870-38-PL Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States;
2013 Phase 3 EUCTR2013-001870-38-ES Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
2003 Phase 2 NCT00263887 Denmark;Sweden;United Kingdom;
Grifols Therapeutics LLC
2018 Phase 3 EUCTR2015-004110-23-PL Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden;United States;
2016 Phase 3 EUCTR2015-004110-23-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-SE Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2014 Phase 3 EUCTR2013-001870-38-FI Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-EE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
Alvelestat (MPH966)
University of Alabama at Birmingham
2019 Phase 2 NCT03679598 United States;
Alvelestat oral tablet - dose 1
Mereo BioPharma
2018 Phase 2 NCT03636347 Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Alvelestat oral tablet - dose 2
Mereo BioPharma
2018 Phase 2 NCT03636347 Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Alvelestat tosylate
Mereo BioPharma 4 Ltd
2019 Phase 2 EUCTR2018-001309-95-PL Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-001309-95-BE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-SE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-GB Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-DK Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Aspirin
Columbia University
2017 Phase 2 NCT03008915 United States;
Belcesiran
Dicerna Pharmaceuticals, Inc.
2021 Phase 2 NCT05146882 New Zealand;
2021 Phase 2 NCT04764448 Australia;New Zealand;Spain;United Kingdom;
2019 Phase 1 NCT04174118 New Zealand;Sweden;
Blood draw
University of Florida
2013 - NCT01810458 United States;
Blood draw.
University of Florida
2007 - NCT01851642 United States;
Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor
Baxalta now part of Shire
2005 Phase 1 NCT00242385 Australia;New Zealand;
Dose of 60 mg/kg alpha1-proteinase inhibitor
Baxalta now part of Shire
2005 Phase 1 NCT00242385 Australia;New Zealand;
Drug-Carbamazepine (Tegretol XR)
Washington University School of Medicine
2012 Phase 2 NCT01379469 United States;
Fentanyl
University of Florida
2013 - NCT01810458 United States;
Glassia
Impatients N.V. trading as myTomorrows
2017 - NCT03172455 -
Octapharma
2018 Phase 2 NCT03385395 -
History and physical
University of Florida
2013 - NCT01810458 United States;
History and physical exam.
University of Florida
2007 - NCT01851642 United States;
Hyaluronic Acid Inhalation Solution
Gerard Turino
2017 Phase 2 NCT03114020 Canada;United States;
INBRX-101/rhAAT-Fc
Inhibrx, Inc.
2019 Phase 1 NCT03815396 New Zealand;United Kingdom;United States;
Intravenous catheter
University of Florida
2013 - NCT01810458 United States;
Kamada -AAT for Inhalation or inhaled AAT (AAT)
Kamada Ltd.
2020 Phase 3 EUCTR2019-000602-30-SE Argentina;Belgium;Finland;Ireland;Italy;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-GB Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2020 Phase 3 EUCTR2019-000602-30-FI Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
2019 Phase 3 EUCTR2019-000602-30-NL Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
Kamada-AAT for Inhalation,
Kamada, Ltd.
2014 Phase 2 NCT02001688 United States;
Kamada-AAT for inhalation
Kamada Limited
2011 - EUCTR2008-005326-36-SE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 Phase 2;Phase 3 EUCTR2008-005326-36-DK Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-NL Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-IE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
2010 - EUCTR2008-005326-36-DE Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom;
Kamada-API
Kamada, Ltd.
2007 Phase 2/Phase 3 NCT00460096 United States;
Lidocaine
University of Florida
2013 - NCT01810458 United States;
Liver Biopsy
University of Florida
2013 - NCT01810458 United States;
Lorazepam
University of Florida
2013 - NCT01810458 United States;
MPH966
Mereo BioPharma 4 Ltd
2019 Phase 2 EUCTR2018-001309-95-PL Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2019 Phase 2 EUCTR2018-001309-95-BE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-SE Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-GB Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
2018 Phase 2 EUCTR2018-001309-95-DK Belgium;Canada;Denmark;Poland;Spain;Sweden;United Kingdom;United States;
Midazolam
University of Florida
2013 - NCT01810458 United States;
None assigned
F. Hoffmann-La Roche Ltd
2008 Phase 2 EUCTR2004-001688-23-GB Denmark;Spain;United Kingdom;
2006 - EUCTR2004-001688-23-DK Denmark;Spain;United Kingdom;
2004 - EUCTR2004-001688-23-ES Denmark;Spain;United Kingdom;
OctaAlpha1
Octapharma
2018 Phase 2 NCT03385395 -
Ondansetron
University of Florida
2013 - NCT01810458 United States;
Oxycodone/Acetaminophen
University of Florida
2013 - NCT01810458 United States;
PHP-303
pH Pharma Inc.
2020 Phase 2 EUCTR2019-002501-22-GB United Kingdom;
Prolastin
UHB NHS Foundation Trust
2008 Phase 2 EUCTR2007-004869-18-GB United Kingdom;
Prolastin-C
Grifols Therapeutics Inc.
2015 Phase 3 EUCTR2013-001870-38-DE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-IE Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
2014 - EUCTR2013-001870-38-PL Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States;
2013 Phase 3 EUCTR2013-001870-38-ES Argentina;Australia;Brazil;Canada;Czech Republic;Denmark;Estonia;Finland;France;Germany;Hungary;Ireland;Latvia;Lithuania;Netherlands;New Zealand;Norway;Poland;Serbia;Slovakia;Spain;Sweden;United Kingdom;United States;
Grifols Therapeutics LLC
2018 Phase 3 EUCTR2015-004110-23-PL Australia;Canada;Denmark;Estonia;New Zealand;Poland;Russian Federation;Sweden;United States;
2016 Phase 3 EUCTR2015-004110-23-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;United States;
2014 Phase 3 EUCTR2013-001870-38-SE Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2014 Phase 3 EUCTR2013-001870-38-FI Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-EE Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
2013 Phase 3 EUCTR2013-001870-38-DK Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;Moldova, Republic of;New Zealand;Poland;Romania;Russian Federation;Serbia;Spain;Sweden;United Kingdom;United States;
Prolastin-C,
Grifols Therapeutics Inc.
2010 Phase 2 NCT01213043 United States;
Pulmonary function testing.
University of Florida
2007 - NCT01851642 United States;
RAAV1-CB-hAAT
Applied Genetic Technologies Corp
2010 Phase 2 NCT01054339 Ireland;United States;
Applied Genetic Technologies Corporation
2010 Phase 2 EUCTR2009-014286-20-IE Ireland;
University of Massachusetts, Worcester
2006 Phase 1 NCT00430768 United States;
RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING THE HUMAN ALPHA-1-ANTITRYPSIN GENE
Kamada Ltd.
2020 Phase 3 EUCTR2019-000602-30-SE Argentina;Belgium;Finland;Ireland;Italy;Netherlands;Sweden;United Kingdom;
2019 Phase 3 EUCTR2019-000602-30-NL Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom;
RO3300074
F. Hoffmann-La Roche Ltd
2008 Phase 2 EUCTR2004-001688-23-GB Denmark;Spain;United Kingdom;
2006 - EUCTR2004-001688-23-DK Denmark;Spain;United Kingdom;
2004 - EUCTR2004-001688-23-ES Denmark;Spain;United Kingdom;
Retinoic Acid Receptor gamma Agonist
F. Hoffmann-La Roche Ltd
2008 Phase 2 EUCTR2004-001688-23-GB Denmark;Spain;United Kingdom;
2006 - EUCTR2004-001688-23-DK Denmark;Spain;United Kingdom;
2004 - EUCTR2004-001688-23-ES Denmark;Spain;United Kingdom;
VX-814
Vertex Pharmaceuticals Incorporated
2020 Phase 2 NCT04167345 Canada;Germany;Ireland;United States;
2020 Phase 2 EUCTR2019-003650-92-SE Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-003650-92-IE Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-003650-92-GB Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-003650-92-DE Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
VX-864
Vertex Pharmaceuticals Incorporated
2020 Phase 2 EUCTR2019-004881-16-SE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-IE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-GB Canada;Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-DE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
VX864
Vertex Pharmaceuticals Incorporated
2020 Phase 2 EUCTR2019-004881-16-SE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-IE Canada;Germany;Ireland;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-GB Canada;Germany;Ireland;Netherlands;Sweden;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004881-16-DE Canada;Germany;Ireland;Sweden;United Kingdom;United States;