30. Distal myopathy Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 15 / Drugs : 17 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathway : 1
Drugs and their primary sponsors and trial info
Aceneuramic Acid Extended-Release
Ultragenyx Pharmaceutical Inc
2016 Phase 2 NCT02731690 Bulgaria;Canada;United States;
Aceneuramic Acid Extended-Release Tablets
Ultragenyx Pharmaceutical Inc
2016 Phase 3 NCT02736188 Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Aceneuramic acid
Suzuki Ryoichi
2021 Phase 3 JPRN-jRCT2021200030 Japan;
Aceneuramic acid extended-release (Ace-ER)
Ultragenyx Pharmaceutical Inc
2015 Phase 3 NCT02377921 Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Acido Sialico - Sialic acid (INN: aceneuramic acid)
ULTRAGENYX PHARMACEUTICAL INC.
2015 Phase 3 EUCTR2014-005432-33-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Common Name: Sialic Acid
ULTRAGENYX PHARMACEUTICAL INC.
2017 Phase 3 EUCTR2016-000360-42-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000360-42-GB Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Deflazacort
Ludwig-Maximilians - University of Munich
2003 Phase 2/Phase 3 NCT00527228 Germany;
INN: aceneuramic acid
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000360-42-GB Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
N-acetylneuraminic acid
Ultragenyx Pharmaceutical Inc.
2017 Phase 2 EUCTR2015-004553-41-BG Bulgaria;Canada;United States;
NPC-09
Nobelpharma
2021 Phase 3 NCT04671472 Japan;
Tohoku University
2010 Phase 1 NCT01236898 Japan;
SA
Tohoku University Hospital, Department of Neurology
2017 Phase 2,3 JPRN-UMIN000026354 Japan;
2016 Phase 2,3 JPRN-UMIN000020683 Japan;
Sialic acid (INN: aceneuramic acid)
Ultragenyx Pharmaceutical Inc.
2017 Phase 2 EUCTR2015-004553-41-BG Bulgaria;Canada;United States;
2016 Phase 3 EUCTR2014-005432-33-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Sialic acid, N-acetylneuraminic acid
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
UX001
ULTRAGENYX PHARMACEUTICAL INC.
2017 Phase 3 EUCTR2016-000360-42-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;
2015 Phase 3 EUCTR2014-005432-33-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2017 Phase 2 EUCTR2015-004553-41-BG Bulgaria;Canada;United States;
2016 Phase 3 EUCTR2016-000360-42-GB Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2016 Phase 3 EUCTR2014-005432-33-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Ultragenyx Pharmaceutical Inc
2016 Phase 2 NCT02731690 Bulgaria;Canada;United States;
Aceneuramic Acid Extended-Release Tablets
Ultragenyx Pharmaceutical Inc
2016 Phase 3 NCT02736188 Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Aceneuramic acid
Suzuki Ryoichi
2021 Phase 3 JPRN-jRCT2021200030 Japan;
Aceneuramic acid extended-release (Ace-ER)
Ultragenyx Pharmaceutical Inc
2015 Phase 3 NCT02377921 Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Acido Sialico - Sialic acid (INN: aceneuramic acid)
ULTRAGENYX PHARMACEUTICAL INC.
2015 Phase 3 EUCTR2014-005432-33-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Common Name: Sialic Acid
ULTRAGENYX PHARMACEUTICAL INC.
2017 Phase 3 EUCTR2016-000360-42-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000360-42-GB Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Deflazacort
Ludwig-Maximilians - University of Munich
2003 Phase 2/Phase 3 NCT00527228 Germany;
INN: aceneuramic acid
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000360-42-GB Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
N-acetylneuraminic acid
Ultragenyx Pharmaceutical Inc.
2017 Phase 2 EUCTR2015-004553-41-BG Bulgaria;Canada;United States;
NPC-09
Nobelpharma
2021 Phase 3 NCT04671472 Japan;
Tohoku University
2010 Phase 1 NCT01236898 Japan;
SA
Tohoku University Hospital, Department of Neurology
2017 Phase 2,3 JPRN-UMIN000026354 Japan;
2016 Phase 2,3 JPRN-UMIN000020683 Japan;
Sialic acid (INN: aceneuramic acid)
Ultragenyx Pharmaceutical Inc.
2017 Phase 2 EUCTR2015-004553-41-BG Bulgaria;Canada;United States;
2016 Phase 3 EUCTR2014-005432-33-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Sialic acid, N-acetylneuraminic acid
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
UX001
ULTRAGENYX PHARMACEUTICAL INC.
2017 Phase 3 EUCTR2016-000360-42-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;
2015 Phase 3 EUCTR2014-005432-33-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Ultragenyx Pharmaceutical Inc.
2017 Phase 3 EUCTR2016-000360-42-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2017 Phase 2 EUCTR2015-004553-41-BG Bulgaria;Canada;United States;
2016 Phase 3 EUCTR2016-000360-42-GB Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
2016 Phase 3 EUCTR2014-005432-33-BG Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;