246. メチルマロン酸血症 Disease details / Clinical trials / Drug dev / DR info

臨床試験数 : 28 / 薬物数 : 25 - (DrugBank : 9) / 標的遺伝子数 : 18 - 標的パスウェイ数 : 26

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）
Ataluren
   PTC THERAPEUTICS, INC.
      2010   -   EUCTR2009-016654-41-IT   France;Italy;United Kingdom
   PTC Therapeutics
      2010   Phase 2   NCT01141075   Belgium;France;Germany;Italy;Switzerland;United Kingdom
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-BE   Belgium;France;Italy;Switzerland;United Kingdom
Ataluren 1000MG
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
Ataluren 125MG
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
Ataluren 250MG
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
BBP-671
   CoA Therapeutics, Inc., a BridgeBio company
      2021   Phase 1   NCT04836494   United States
Bucladesine
   Ito Tetsuya
      2022   Phase 1-2   JPRN-jRCT2041210065   -
Carbaglu
   Mendel Tuchman
      2012   Phase 2   NCT01599286   United States
   National Taiwan University Hospital
      2019   -   NCT04284917   Taiwan
Carglumic acid
   King Abdullah International Medical Research Center
      2015   Phase 3   NCT02426775   Saudi Arabia
   National Taiwan University Hospital
      2019   -   NCT04284917   Taiwan
   Recordati Rare Diseases
      2022   -   NCT05040178   United States
      2019   -   NCT04176523   France;Germany;Italy;Norway;Spain;Sweden;United Kingdom
   Target PharmaSolutions, Inc.
      2022   -   NCT05842837   United States
Compound 1A
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
Dimebutic acid
   HemoShear Therapeutics
      -   Phase 2   EUCTR2023-000785-33-Outside-EU/EEA   Australia;Saudi Arabia;United States
HLB-001
   LogicBio Therapeutics, Inc
      2022   -   NCT05506254   United States
      2021   Phase 1/Phase 2   NCT04581785   Saudi Arabia;United States
HST5040
   HemoShear Therapeutics
      2021   Phase 2   NCT04732429   Australia;Saudi Arabia;United States
      -   Phase 2   EUCTR2023-000785-33-Outside-EU/EEA   Australia;Saudi Arabia;United States
Modified mrna encoding human methylmalonyl-coenzyme A mutase
   ModernaTX, Inc.
      2023   Phase 1;Phase 2   EUCTR2020-004980-24-NL   Australia;Canada;France;Netherlands;Spain;Switzerland;United Kingdom;United States
      2023   Phase 1;Phase 2   EUCTR2020-004980-24-FR   Australia;Canada;France;Netherlands;Spain;United Kingdom;United States
      2023   Phase 1;Phase 2   EUCTR2020-004980-24-ES   Australia;Canada;France;Netherlands;Spain;United Kingdom;United States
MRNA-3704
   ModernaTX, Inc.
      2019   Phase 1/Phase 2   NCT03810690   United States
      2019   Phase 1;Phase 2   EUCTR2019-001061-32-GB   United Kingdom;United States
MRNA-3705
   ModernaTX, Inc.
      2023   Phase 1;Phase 2   EUCTR2020-004980-24-NL   Australia;Canada;France;Netherlands;Spain;Switzerland;United Kingdom;United States
      2023   Phase 1;Phase 2   EUCTR2020-004980-24-FR   Australia;Canada;France;Netherlands;Spain;United Kingdom;United States
      2023   Phase 1;Phase 2   EUCTR2020-004980-24-ES   Australia;Canada;France;Netherlands;Spain;United Kingdom;United States
      2022   Phase 1/Phase 2   NCT05295433   Canada;France;Netherlands;Spain;United Kingdom;United States
      2021   Phase 1/Phase 2   NCT04899310   Australia;Canada;France;Netherlands;Spain;United Kingdom;United States
N-carbamylglutamate
   Children's Hospital of Philadelphia
      2010   Phase 2   NCT01341379   United States
   Mendel Tuchman
      2012   Phase 2   NCT01597440   United States
PEG
   Zhengzhou Children's Hospital
      2022   Phase 4   ChiCTR2100054613   China
PTC-0161480
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
PTC-124
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
PTC-C124
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
PTC124
   PTC THERAPEUTICS, INC.
      2010   -   EUCTR2009-016654-41-IT   France;Italy;United Kingdom
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-BE   Belgium;France;Italy;Switzerland;United Kingdom
Rhgh
   Zhengzhou Children's Hospital
      2022   Phase 4   ChiCTR2100054613   China
RPS 2505
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom
      2010   Phase 2   EUCTR2009-016654-41-FR   Belgium;France;Italy;United Kingdom
SEL-302
   Selecta Biosciences, Inc.
      2022   Phase 1/Phase 2   NCT05778877   United States
Standard OF care treatment
   Mendel Tuchman
      2012   Phase 2   NCT01599286   United States