231. α1−アンチトリプシン欠乏症 Disease details / Clinical trials / Drug dev / DR info

4-PBA
   University of Florida
      2001   Phase 2   NCT00067756   United States
Abdominal ultrasound
   University of Florida
      2013   -   NCT01810458   United States
ADS-001
   ARROWHEAD PHARMACEUTICALS
      2019   Phase 2   EUCTR2018-003385-14-IT   Austria;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States
   Arrowhead Pharmaceuticals, Inc.
      2021   Phase 2   EUCTR2018-003385-14-DE   Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2018-003385-14-AT   Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2018-003385-14-SE   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
      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
      2019   Phase 2;Phase 3   EUCTR2018-003385-14-IE   Austria;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
      -   Phase 2   EUCTR2019-000068-86-DE   Austria;Germany;United Kingdom
ADVM-043
   Adverum Biotechnologies, Inc.
      2019   -   NCT03804021   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-NL   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
      2010   -   EUCTR2008-005326-36-IE   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-DE   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
      2009   Phase 2;Phase 3   EUCTR2008-005326-36-GB   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
Albuterol
   University of Florida
      2007   -   NCT01851642   United States
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
Alpha 1-antitrypsin
   Kamada, Ltd.
      2019   Phase 3   NCT04204252   Belgium;Finland;Ireland;Netherlands;Sweden;United Kingdom
Alpha 1-proteinase inhibitor
   UHB NHS Foundation Trust
      2008   Phase 2   EUCTR2007-004869-18-GB   United Kingdom
Alpha-1 antitrypsin (AAT)
   Kamada Ltd.
      2021   Phase 3   EUCTR2019-000602-30-IE   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
      2020   Phase 3   EUCTR2019-000602-30-SE   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
      2020   Phase 3   EUCTR2019-000602-30-FI   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
      2020   Phase 3   EUCTR2019-000602-30-BE   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
Alpha-1 MP
   Grifols Therapeutics Inc.
      2006   Phase 3   NCT00295061   United States
   Grifols Therapeutics LLC
      2016   Phase 3   NCT02796937   Australia;Canada;Denmark;Estonia;Finland;France;New Zealand;Poland;Russian Federation;Sweden;United States
Alpha-1-antitrypsin
   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-SE   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States
      2014   Phase 3   EUCTR2013-001870-38-PL   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Slovakia;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
      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
      2023   Phase 3   EUCTR2015-004110-23-FR   Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Moldova, Republic of;New Zealand;Poland;Russian Federation;Serbia;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2015-004110-23-FI   Australia;Canada;Denmark;Estonia;Finland;France;Moldova, Republic of;New Zealand;Poland;Russian Federation;Sweden;United States
      2018   Phase 3   EUCTR2015-004110-23-PL   Australia;Canada;Denmark;Estonia;Finland;New Zealand;Poland;Russian Federation;Sweden;United States
      2016   Phase 3   EUCTR2015-004110-23-EE   Australia;Canada;Denmark;Estonia;Finland;France;New Zealand;Poland;Russian Federation;Sweden;United States
      2016   Phase 3   EUCTR2015-004110-23-DK   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;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
   Kamada Limited
      2011   -   EUCTR2008-005326-36-SE   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
      2010   Phase 2;Phase 3   EUCTR2008-005326-36-NL   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
      2010   -   EUCTR2008-005326-36-IE   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-DE   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
      2009   Phase 2;Phase 3   EUCTR2008-005326-36-GB   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
   UHB NHS Foundation Trust
      2008   Phase 2   EUCTR2007-004869-18-GB   United Kingdom
Alpha1-proteinase inhibitor
   Baxalta now part of Shire
      2006   Phase 4   NCT00396006   Australia;New Zealand
   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-SE   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States
      2014   Phase 3   EUCTR2013-001870-38-PL   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Slovakia;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
      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
      2023   Phase 3   EUCTR2015-004110-23-FR   Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Moldova, Republic of;New Zealand;Poland;Russian Federation;Serbia;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2015-004110-23-FI   Australia;Canada;Denmark;Estonia;Finland;France;Moldova, Republic of;New Zealand;Poland;Russian Federation;Sweden;United States
      2018   Phase 3   EUCTR2015-004110-23-PL   Australia;Canada;Denmark;Estonia;Finland;New Zealand;Poland;Russian Federation;Sweden;United States
      2016   Phase 3   EUCTR2015-004110-23-EE   Australia;Canada;Denmark;Estonia;Finland;France;New Zealand;Poland;Russian Federation;Sweden;United States
      2016   Phase 3   EUCTR2015-004110-23-DK   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;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
   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
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
   Arrowhead Pharmaceuticals
      2019   Phase 2   NCT03946449   Austria;Germany;United Kingdom
ARO-AAT injection
   ARROWHEAD PHARMACEUTICALS
      2019   Phase 2   EUCTR2018-003385-14-IT   Austria;Germany;Ireland;Italy;Netherlands;Poland;Portugal;Spain;Sweden;United Kingdom;United States
   Arrowhead Pharmaceuticals
      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
      2020   Phase 2   EUCTR2018-003385-14-SE   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
      2019   Phase 2   EUCTR2019-000068-86-GB   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
      2019   Phase 2;Phase 3   EUCTR2018-003385-14-IE   Austria;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
Aspirin
   Columbia University
      2017   Phase 2   NCT03008915   United States
BEAM-302
   Beam Therapeutics Inc.
      2024   Phase 1/Phase 2   NCT06389877   Australia;Netherlands;New Zealand;United Kingdom
Belcesiran
   Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
      2021   Phase 2   NCT05146882   New Zealand
      2021   Phase 2   NCT04764448   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Netherlands;New Zealand;Portugal;Spain;Sweden;United Kingdom;United States
      2019   Phase 1   NCT04174118   New Zealand;Sweden
Biological NTLA-3001
   Intellia Therapeutics
      2024   Phase 1/Phase 2   NCT06622668   New Zealand
Blood draw
   University of Florida
      2013   -   NCT01810458   United States
      2007   -   NCT01851642   United States
BMN 349
   BioMarin Pharmaceutical
      2025   Phase 1   NCT06738017   United States
Carbamazepine
   Washington University School of Medicine
      2012   Phase 2   NCT01379469   United States
Dose OF 60 MG/KG alpha1-proteinase inhibitor
   Baxalta now part of Shire
      2005   Phase 1   NCT00242385   Australia;New Zealand
Dose OF 60 MG/KG fraction IV-1 alpha1-proteinase inhibitor
   Baxalta now part of Shire
      2005   Phase 1   NCT00242385   Australia;New Zealand
Fazirsiran
   Arrowhead Pharmaceuticals, Inc.
      -   Phase 2   EUCTR2019-000068-86-DE   Austria;Germany;United Kingdom
Fazirsiran (TAK-999, also referred TO AS ARO-AAT) injection
   Arrowhead Pharmaceuticals, Inc.
      2021   Phase 2   EUCTR2018-003385-14-DE   Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States
Fazirsiran (TAK-999, ARO-AAT) injection
   Arrowhead Pharmaceuticals, Inc.
      2019   Phase 2   EUCTR2019-000068-86-AT   Austria;Germany;United Kingdom
Fazisiran injection
   Arrowhead Pharmaceuticals
      2019   Phase 2   NCT03945292   Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United States
Fentanyl
   University of Florida
      2013   -   NCT01810458   United States
Genetic sequencing
   Medical University of South Carolina
      2015   -   NCT02810327   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
      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
Inhaler
   University of Florida
      2007   -   NCT01851642   United States
Intravenous catheter
   University of Florida
      2013   -   NCT01810458   United States
Kamada -AAT FOR inhalation or inhaled AAT (AAT)
   Kamada Ltd.
      2021   Phase 3   EUCTR2019-000602-30-IE   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
      2020   Phase 3   EUCTR2019-000602-30-SE   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
      2020   Phase 3   EUCTR2019-000602-30-FI   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
      2020   Phase 3   EUCTR2019-000602-30-BE   Australia;Belgium;Finland;Ireland;Netherlands;New Zealand;Sweden;United Kingdom
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-NL   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
      2010   -   EUCTR2008-005326-36-IE   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-DE   Denmark;Germany;Ireland;Netherlands;Sweden;United Kingdom
Kamada-AAT FOR inhalation, 160MG
   Kamada, Ltd.
      2014   Phase 2   NCT02001688   United States
Kamada-AAT FOR inhalation, 80MG
   Kamada, Ltd.
      2014   Phase 2   NCT02001688   United States
Kamada-API
   Kamada, Ltd.
      2007   Phase 2/Phase 3   NCT00460096   United States
KRRO-110
   Korro Bio, Inc.
      2025   Phase 1/Phase 2   NCT06677307   Australia;New Zealand
Lidocaine
   University of Florida
      2013   -   NCT01810458   United States
Liver biopsy
   University of Florida
      2013   -   NCT01810458   United States
Liver questionnaire
   University of Florida
      2013   -   NCT01810458   United States
Lorazepam
   University of Florida
      2013   -   NCT01810458   United States
Midazolam
   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
   University of Alabama at Birmingham
      2019   Phase 2   NCT03679598   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   Phase 2   EUCTR2004-001688-23-ES   Denmark;Spain;United Kingdom
Octaalpha1
   Octapharma
      2018   Phase 2   NCT03385395   -
Ondansetron
   University of Florida
      2013   -   NCT01810458   United States
Osrhaat 1 MG/KG IV
   Healthgen Biotechnology Corp.
      2022   Phase 1   NCT05315921   United States
Osrhaat 10 MG/KG IV
   Healthgen Biotechnology Corp.
      2022   Phase 1   NCT05315921   United States
Osrhaat 20 MG/KG IV
   Healthgen Biotechnology Corp.
      2022   Phase 1   NCT05315921   United States
Osrhaat 3 MG/KG IV
   Healthgen Biotechnology Corp.
      2022   Phase 1   NCT05315921   United States
Osrhaat 40 MG/KG IV
   Healthgen Biotechnology Corp.
      2022   Phase 1   NCT05315921   United States
Osrhaat 60 MG/KG IV
   Healthgen Biotechnology Corp.
      2022   Phase 1   NCT05315921   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-SE   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Slovakia;Spain;Sweden;United States
      2014   Phase 3   EUCTR2013-001870-38-PL   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;Ireland;New Zealand;Poland;Romania;Slovakia;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
      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
      2023   Phase 3   EUCTR2015-004110-23-FR   Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Moldova, Republic of;New Zealand;Poland;Russian Federation;Serbia;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2015-004110-23-FI   Australia;Canada;Denmark;Estonia;Finland;France;Moldova, Republic of;New Zealand;Poland;Russian Federation;Sweden;United States
      2018   Phase 3   EUCTR2015-004110-23-PL   Australia;Canada;Denmark;Estonia;Finland;New Zealand;Poland;Russian Federation;Sweden;United States
      2016   Phase 3   EUCTR2015-004110-23-EE   Australia;Canada;Denmark;Estonia;Finland;France;New Zealand;Poland;Russian Federation;Sweden;United States
      2016   Phase 3   EUCTR2015-004110-23-DK   Argentina;Australia;Brazil;Canada;Denmark;Estonia;Finland;France;Germany;New Zealand;Poland;Romania;Russian Federation;Spain;Sweden;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, 120 MG/KG
   Grifols Therapeutics Inc.
      2010   Phase 2   NCT01213043   United States
Prolastin-C, 60 MG/KG
   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
RAAV2-CB-haat gene vector
   University of Massachusetts, Worcester
      2004   Early Phase 1   NCT00377416   United States
Recombinant adeno-associated viral vector expressing THE human alpha-1-antitrypsin gene
   Kamada Ltd.
      2019   Phase 3   EUCTR2019-000602-30-NL   Belgium;Finland;Ireland;Netherlands;Sweden;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   Phase 2   EUCTR2004-001688-23-ES   Denmark;Spain;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   Phase 2   EUCTR2004-001688-23-ES   Denmark;Spain;United Kingdom
SAR447537
   Sanofi
      2024   Phase 2   NCT05897424   Australia;Denmark;Ireland;New Zealand;Poland;Spain;Sweden;United Kingdom;United States
      2023   Phase 2   NCT05856331   Australia;Denmark;Ireland;New Zealand;Poland;Spain;Sweden;United Kingdom;United States
TAK-999, ARO-AAT
   Arrowhead Pharmaceuticals, Inc.
      2021   Phase 2   EUCTR2018-003385-14-DE   Austria;Canada;Germany;Ireland;Italy;Netherlands;Portugal;Spain;Sweden;United Kingdom;United States
      -   Phase 2   EUCTR2019-000068-86-DE   Austria;Germany;United Kingdom
VX-634
   Vertex Pharmaceuticals Incorporated
      2022   Phase 1   NCT05579431   United States
VX-668
   Vertex Pharmaceuticals Incorporated
      2023   Phase 1   NCT05727800   United States
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
      2023   Phase 2   NCT05643495   Germany;Ireland;United Kingdom;United States
      2023   Phase 2   EUCTR2022-002746-40-IE   France;Germany;Ireland
      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
      2023   Phase 2   EUCTR2022-002746-40-IE   France;Germany;Ireland
      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
Withdrawal from alpha1 antitrypsin replacement therapy
   Columbia University
      2017   Phase 2   NCT03008915   United States
WVE-006
   Wave Life Sciences Ltd.
      2024   Phase 1/Phase 2   NCT06405633   Australia;Canada;Finland;New Zealand;United Kingdom
      2023   Phase 1   NCT06186492   United Kingdom
Zemaira
   Sanofi
      2023   Phase 2   NCT05856331   Australia;Denmark;Ireland;New Zealand;Poland;Spain;Sweden;United Kingdom;United States