246. Methylmalonic acidemia Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 23 / Drugs : 31 - (DrugBank : 8) / Drug target genes : 17 - Drug target pathways : 23
Drugs and their primary sponsors and trial info
291844
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
Ataluren
PTC Therapeutics
2010 Phase 2 NCT01141075 Belgium;France;Germany;Italy;Switzerland;United Kingdom;
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 Phase 2 EUCTR2009-016654-41-BE Belgium;France;Italy;Switzerland;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
BBP-671
CoA Therapeutics, Inc., a BridgeBio company
2021 Phase 1 NCT04836494 United States;
Bucladesine
Ito Tetsuya
2021 Phase 1-2 JPRN-jRCT2041210065 Japan;
Carbaglu
Mendel Tuchman
2012 Phase 2 NCT01599286 United States;
Carglumic Acid (Carbaglu®)
National Taiwan University Hospital
2019 - NCT04284917 Taiwan;
Compound 1a
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
HLB-001
LogicBio Therapeutics, Inc.
2021 Phase 1/Phase 2 NCT04581785 Saudi Arabia;United States;
HST5040
HemoShear Therapeutics
2021 Phase 2 NCT04732429 United States;
MRNA-3704
ModernaTX, Inc.
2019 Phase 1;Phase 2 EUCTR2019-001061-32-GB United Kingdom;United States;
2019 Phase 1/Phase 2 NCT03810690 United States;
MRNA-3705
ModernaTX, Inc.
2021 Phase 1/Phase 2 NCT04899310 Canada;United Kingdom;
PTC-0161480
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
PTC-124
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
PTC-C124
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR 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-BE Belgium;France;Italy;Switzerland;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
RPS 2505
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
Ataluren
PTC Therapeutics
2010 Phase 2 NCT01141075 Belgium;France;Germany;Italy;Switzerland;United Kingdom;
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 Phase 2 EUCTR2009-016654-41-BE Belgium;France;Italy;Switzerland;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
BBP-671
CoA Therapeutics, Inc., a BridgeBio company
2021 Phase 1 NCT04836494 United States;
Bucladesine
Ito Tetsuya
2021 Phase 1-2 JPRN-jRCT2041210065 Japan;
Carbaglu
Mendel Tuchman
2012 Phase 2 NCT01599286 United States;
Carglumic Acid (Carbaglu®)
National Taiwan University Hospital
2019 - NCT04284917 Taiwan;
Compound 1a
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
HLB-001
LogicBio Therapeutics, Inc.
2021 Phase 1/Phase 2 NCT04581785 Saudi Arabia;United States;
HST5040
HemoShear Therapeutics
2021 Phase 2 NCT04732429 United States;
MRNA-3704
ModernaTX, Inc.
2019 Phase 1;Phase 2 EUCTR2019-001061-32-GB United Kingdom;United States;
2019 Phase 1/Phase 2 NCT03810690 United States;
MRNA-3705
ModernaTX, Inc.
2021 Phase 1/Phase 2 NCT04899310 Canada;United Kingdom;
PTC-0161480
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
PTC-124
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
PTC-C124
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR 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-BE Belgium;France;Italy;Switzerland;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;
RPS 2505
PTC Therapeutics, Inc.
2010 Phase 2 EUCTR2009-016654-41-GB Belgium;France;Italy;United Kingdom;
2010 - EUCTR2009-016654-41-FR France;Italy;United Kingdom;