229. 肺胞蛋白症（自己免疫性又は先天性） Disease details / Clinical trials / Drug dev / DR info

臨床試験数 : 46 / 薬物数 : 23 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 15

薬物、開発者（Primary Sponsor）、臨床試験情報（症例登録開始年（Enrollment year）、フェーズ、ID、実施国）
Baseline WLL
   Fondazione IRCCS Policlinico San Matteo di Pavia
      2009   Phase 2   NCT00901511   Italy
Blood collection
   Assistance Publique - Hôpitaux de Paris
      2021   -   NCT04811274   France
Brain MRI
   Assistance Publique - Hôpitaux de Paris
      2019   Phase 1/Phase 2   NCT03887169   France
Bronchoalveolar lavage fluid collection
   Assistance Publique - Hôpitaux de Paris
      2021   -   NCT04811274   France
Centricyte 1000
   Black Tie Medical, Inc.
      2020   Early Phase 1   NCT04326036   United States
GM-CSF
   Dai Huaping
      2016   Phase 2   NCT03316651   China
   Peking Union Medical College Hospital
      2014   Phase 2   NCT02243228   China
   The Cleveland Clinic
      2001   Phase 2   NCT00030056   United States
IV deployment OF csvf IN sterile normal saline IV solution
   Black Tie Medical, Inc.
      2020   Early Phase 1   NCT04326036   United States
Leukine
   Children's Hospital Medical Center, Cincinnati
      2012   Phase 2   NCT01511068   United States
   OSPEDALE POLICLINICO S. MATTEO
      2008   -   EUCTR2008-007086-23-IT   Italy
Methionine/homocysteine dosage
   Assistance Publique - Hôpitaux de Paris
      2019   Phase 1/Phase 2   NCT03887169   France
Molgramostim
   Savara Inc.
      2021   Phase 3   NCT04544293   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   NCT03482752   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;Turkey;United Kingdom
      2016   Phase 2   NCT02702180   Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
      2015   Phase 1   NCT02468908   United Kingdom
Molgramostim 300 MCG nebuliser solution
   Savara ApS
      2018   Phase 2;Phase 3   EUCTR2015-003878-33-PT   Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-003878-33-NL   Belgium;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-003878-33-IT   Denmark;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Portugal;Russian Federation;Spain;Switzerland;United Kingdom
      2016   Phase 2;Phase 3   EUCTR2015-003878-33-GB   Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-003878-33-ES   Denmark;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Portugal;Russian Federation;Spain;Switzerland;United Kingdom
      2016   Phase 2;Phase 3   EUCTR2015-003878-33-DE   Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-003878-33-DK   Belgium;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
   Serendex Pharmaceuticals A/S
      2016   Phase 2;Phase 3   EUCTR2015-003878-33-GR   Belgium;Denmark;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Portugal;Russian Federation;Spain;Switzerland;United Kingdom
Molgramostim 300 µG nebulizer solution
   SAVARA APS
      2020   Phase 3   EUCTR2020-001263-85-IT   Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Turkey;United Kingdom;United States
   Savara ApS
      2022   Phase 3   EUCTR2020-001263-85-PT   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2020-001263-85-GR   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2020-001263-85-ES   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Spain;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2020-001263-85-RO   Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2020-001263-85-NL   Belgium;Canada;France;Germany;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2020-001263-85-IE   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2020-001263-85-DE   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2020-001263-85-BE   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Türkiye;United Kingdom;United States
      2020   Phase 3   EUCTR2020-001263-85-PL   Australia;Belgium;Canada;France;Germany;Greece;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Türkiye;United Kingdom;United States
Molgramostim nebuliser solution 300 MCG
   Savara ApS
      2019   Phase 3   EUCTR2017-004078-32-IT   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
      2018   Phase 3   EUCTR2017-004078-32-NL   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
      2018   Phase 3   EUCTR2017-004078-32-GR   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
      2018   Phase 3   EUCTR2017-004078-32-GB   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
      2018   Phase 3   EUCTR2017-004078-32-FR   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
      2018   Phase 3   EUCTR2017-004078-32-DK   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
      2018   Phase 3   EUCTR2017-004078-32-DE   Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;United Kingdom
Molgramostim nebulizer solution
   Savara Inc.
      2024   -   NCT06546098   -
Pari eflow nebulizer system
   Savara Inc.
      2016   Phase 2   NCT02702180   Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;United States
Pioglitazone
   Children's Hospital Medical Center, Cincinnati
      2017   Phase 1   NCT03231033   United States
Rescue WLL
   Fondazione IRCCS Policlinico San Matteo di Pavia
      2009   Phase 2   NCT00901511   Italy
RHGM-CSF
   Shanghai Pulmonary Hospital, Shanghai, China
      2012   Phase 2   NCT01983657   China
Rituximab
   East Carolina University
      2007   Phase 2   NCT00552461   United States
   National Institute of Allergy and Infectious Diseases (NIAID)
      2014   Phase 1   NCT01842386   United States
Sargramostim
   Children's Hospital Medical Center, Cincinnati
      2017   Phase 1   NCT03006146   United States
   Fondazione IRCCS Policlinico San Matteo di Pavia
      2009   Phase 2   NCT00901511   Italy
   Niigata University Medical & Dental Hospital
      2016   Phase 1   NCT02840708   Japan
      2016   Phase 2   NCT02835742   Japan
   OSPEDALE POLICLINICO S. MATTEO
      2008   -   EUCTR2008-007086-23-IT   Italy
   The Cleveland Clinic
      2001   Phase 2   NCT00030056   United States
Sterile normal saline FOR intravenous USE
   Black Tie Medical, Inc.
      2020   Early Phase 1   NCT04326036   United States
Thoracic CT scan
   Assistance Publique - Hôpitaux de Paris
      2019   Phase 1/Phase 2   NCT03887169   France
Vitamin B12, B9, B6, C supplementation
   Assistance Publique - Hôpitaux de Paris
      2019   Phase 1/Phase 2   NCT03887169   France