303. アッシャー症候群 Disease details / Clinical trials / Drug dev / DR info /

臨床試験数 : 15 / 薬物数 : 19 - (DrugBank : 5) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1

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）
AAVB-081
   AAVantgarde Bio Srl
      2024   Phase 1/Phase 2   NCT06591793   Italy;United Kingdom
BF844
   EyeXCel Pty. Ltd.
      2024   Phase 1   NCT06592131   Australia
Blood draw FOR THE laboratory assessment
   Sanofi
      2013   Phase 2   NCT02065011   France;United States
Dual AAV8.MYO7A
   FONDAZIONE TELETHON
      2023   Phase 1;Phase 2   EUCTR2022-001092-14-IT   Italy;Spain
Intravitreal injection OF ultevursen
   Laboratoires Thea
      2024   Phase 2   NCT06627179   United Kingdom;United States
Lentiviral vector containing MYO7A gene
   sanofi-aventis recherche & développement
      2013   Phase 1;Phase 2   EUCTR2012-002574-31-FR   France;United States
Lentiviral vector containing THE human MY07A gene
   sanofi-aventis recherche & développement
      2013   Phase 1;Phase 2   EUCTR2012-002574-31-FR   France;United States
NO intervention, will NOT receive ANY active study intervention
   Laboratoires Thea
      2024   Phase 2   NCT06627179   United Kingdom;United States
NPI-001
   Nacuity Pharmaceuticals, Inc.
      2020   Phase 1/Phase 2   NCT04355689   Australia
NT-501
   Neurotech Pharmaceuticals
      2012   Phase 2   NCT01530659   United States
OPCT-001
   BlueRock Therapeutics
      2025   Phase 1/Phase 2   NCT06789445   United States
Prednisone
   FONDAZIONE TELETHON
      2023   Phase 1;Phase 2   EUCTR2022-001092-14-IT   Italy;Spain
QR-421A
   ProQR Therapeutics
      2021   Phase 2/Phase 3   NCT05176717   United Kingdom;United States
      2019   Phase 1/Phase 2   NCT03780257   Belgium;Canada;France;United States
RNA antisense oligonucleotide FOR intravitreal injection
   ProQR Therapeutics
      2021   Phase 2   NCT05085964   Canada;France;United States
SAR421869
   Sanofi
      2012   Phase 1/Phase 2   NCT01505062   France;United States
   sanofi-aventis recherche & développement
      2015   Phase 2   EUCTR2013-000597-29-FR   France;United States
      2013   Phase 1;Phase 2   EUCTR2012-002574-31-FR   France;United States
Sham
   Neurotech Pharmaceuticals
      2012   Phase 2   NCT01530659   United States
SHAM-procedure
   Laboratoires Thea
      2021   Phase 2/Phase 3   NCT05158296   Germany;Netherlands;United Kingdom;United States
   ProQR Therapeutics
      2021   Phase 2/Phase 3   NCT05176717   United Kingdom;United States
      2019   Phase 1/Phase 2   NCT03780257   Belgium;Canada;France;United States
Ultevursen
   Laboratoires Thea
      2021   Phase 2/Phase 3   NCT05158296   Germany;Netherlands;United Kingdom;United States
Ushstat
   sanofi-aventis recherche & développement
      2015   Phase 2   EUCTR2013-000597-29-FR   France;United States
      2013   Phase 1;Phase 2   EUCTR2012-002574-31-FR   France;United States