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DR info

Clinical trials : 15 / Drugs : 13 - (DrugBank : 6) / Drug target gene : 1 - Drug target pathways : 3

Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)

BBP-671
   CoA Therapeutics, Inc., a BridgeBio company
      2021   Phase 1   NCT04836494   United States
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
CX-016492
   ModernaTX, Inc
      2020   Phase 1;Phase 2   EUCTR2019-003529-36-GB   Canada;United Kingdom;United States
CX-017950
   ModernaTX, Inc
      2020   Phase 1;Phase 2   EUCTR2019-003529-36-GB   Canada;United Kingdom;United States
Dimebutic acid
   HemoShear Therapeutics
      -   Phase 2   EUCTR2023-000785-33-Outside-EU/EEA   Australia;Saudi Arabia;United States
Disodium citrate
   Nicola Longo
      2008   Phase 1   NCT00645879   United States
Glutamine
   Nicola Longo
      2008   Phase 1   NCT00645879   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
MRNA-3927
   ModernaTX, Inc
      2020   Phase 1;Phase 2   EUCTR2019-003529-36-GB   Canada;United Kingdom;United States
   ModernaTX, Inc.
      2021   Phase 1/Phase 2   NCT05130437   Canada;United Kingdom;United States
      2021   Phase 1/Phase 2   NCT04159103   Canada;Japan;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
Ornithine alpha ketoglutarate
   Nicola Longo
      2008   Phase 1   NCT00645879   United States
Standard OF care treatment
   Mendel Tuchman
      2012   Phase 2   NCT01599286   United States