340. 線毛機能不全症候群（カルタゲナー症候群を含む。） Disease details / Clinical trials / Drug dev / DR info /

臨床試験数 : 23 / 薬物数 : 29 - (DrugBank : 8) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 18

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）
4.2% nacl
   PARION SCIENCES, INC.
      -   Phase 2   EUCTR2015-004917-26-IT   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Poland;Sweden;United Kingdom;United States
4.2% nacl/inhalation solution
   Parion Sciences, Incorporated
      2017   Phase 2   EUCTR2015-004917-26-NL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-GB   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Poland;Sweden;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DK   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DE   Australia;Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      -   Phase 2   EUCTR2015-004917-26-PL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
Albuterol
   University of North Carolina, Chapel Hill
      2021   Early Phase 1   NCT04901715   United States
Azithromycin monohydrate
   Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
      2014   Phase 3   EUCTR2013-004664-58-NL   Denmark;Germany;Netherlands;Switzerland;United Kingdom
      2014   Phase 3   EUCTR2013-004664-58-GB   Denmark;Germany;Netherlands;Switzerland;United Kingdom
      2014   Phase 3   EUCTR2013-004664-58-DK   Denmark;Germany;Netherlands;Switzerland;United Kingdom
      2014   Phase 3   EUCTR2013-004664-58-DE   Denmark;Germany;Netherlands;Switzerland;United Kingdom
Azithromycine CF 250 MG
   Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
      2014   Phase 3   EUCTR2013-004664-58-NL   Denmark;Germany;Netherlands;Switzerland;United Kingdom
      2014   Phase 3   EUCTR2013-004664-58-GB   Denmark;Germany;Netherlands;Switzerland;United Kingdom
Azitromycine CF 250 MG
   Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
      2014   Phase 3   EUCTR2013-004664-58-DK   Denmark;Germany;Netherlands;Switzerland;United Kingdom
      2014   Phase 3   EUCTR2013-004664-58-DE   Denmark;Germany;Netherlands;Switzerland;United Kingdom
BEET-IT juice
   The University of Texas Health Science Center, Houston
      2019   -   NCT04489472   United States
Exhaled nitric oxide
   The Hospital for Sick Children
      2010   -   NCT01155115   Canada
Glycine
   Instituto Nacional de Enfermedades Respiratorias
      2025   -   NCT06959251   Mexico
Hypertonic saline
   Parion Sciences
      2016   Phase 2   NCT02871778   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
Ivacaftor
   Parion Sciences
      2016   Phase 2   NCT02871778   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
   Parion Sciences, Incorporated
      2017   Phase 2   EUCTR2015-004917-26-NL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DK   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DE   Australia;Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      -   Phase 2   EUCTR2015-004917-26-PL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
Kalydeco
   Parion Sciences, Incorporated
      2017   Phase 2   EUCTR2015-004917-26-NL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DK   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DE   Australia;Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      -   Phase 2   EUCTR2015-004917-26-PL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
Magnesium+thiamine
   Instituto Nacional de Enfermedades Respiratorias
      2025   -   NCT06959251   Mexico
Nasal nitric oxide testing
   Connecticut Children's Medical Center
      2023   -   NCT05889013   United States
Pulmonary function testing
   The Hospital for Sick Children
      2010   -   NCT01155115   Canada
QD-27
   University of Leeds
      2021   -   NCT06028607   United Kingdom
RCT1100
   ReCode Therapeutics
      2024   Phase 1   NCT06633757   Denmark;Germany;United States
      2024   Phase 1   NCT06600425   United Kingdom
      2023   Phase 1   NCT05737485   Australia;New Zealand;United Kingdom;United States
Salbutamol
   Rambam Health Care Campus
      2023   -   NCT05932316   Israel
Sano genetics testing KIT
   ReCode Therapeutics
      2023   -   NCT06172374   United States
Soya lecithin
   Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
      2014   Phase 3   EUCTR2013-004664-58-DK   Denmark;Germany;Netherlands;Switzerland;United Kingdom
Spirometry
   Rambam Health Care Campus
      2023   -   NCT05932316   Israel
Sputum collection
   The Hospital for Sick Children
      2010   -   NCT01155115   Canada
TEM
   Peking Union Medical College Hospital
      2024   -   ChiCTR2400082471   China
VX-371
   Parion Sciences
      2016   Phase 2   NCT02871778   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
VX-371 % 0,17% nacl
   PARION SCIENCES, INC.
      -   Phase 2   EUCTR2015-004917-26-IT   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Poland;Sweden;United Kingdom;United States
VX-371 / 0.17% nacl
   Parion Sciences, Incorporated
      2017   Phase 2   EUCTR2015-004917-26-NL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-GB   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Poland;Sweden;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DK   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DE   Australia;Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      -   Phase 2   EUCTR2015-004917-26-PL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
VX-371 / 4,2% nacl
   PARION SCIENCES, INC.
      -   Phase 2   EUCTR2015-004917-26-IT   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Poland;Sweden;United Kingdom;United States
VX-371 / 4.2% nacl
   Parion Sciences, Incorporated
      2017   Phase 2   EUCTR2015-004917-26-NL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-GB   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Poland;Sweden;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DK   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DE   Australia;Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      -   Phase 2   EUCTR2015-004917-26-PL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
VX-770
   Parion Sciences, Incorporated
      2017   Phase 2   EUCTR2015-004917-26-NL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DK   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      2016   Phase 2   EUCTR2015-004917-26-DE   Australia;Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States
      -   Phase 2   EUCTR2015-004917-26-PL   Canada;Denmark;Germany;Italy;Netherlands;Poland;United Kingdom;United States