30. Distal myopathy Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 13 / Drugs : 11 - (DrugBank : 2) / Drug target gene : 1 - Drug target pathway : 1
Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
Aceneuramic acid
Suzuki Ryoichi
2021 Phase 3 JPRN-jRCT2021200030 -
ULTRAGENYX PHARMACEUTICAL INC.
2017 Phase 3 EUCTR2016-000360-42-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom
Aceneuramic acid extended-release
Ultragenyx Pharmaceutical Inc
2016 Phase 2 NCT02731690 Bulgaria;Canada;United States
2015 Phase 3 NCT02377921 Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
Aceneuramic acid extended-release tablets
Ultragenyx Pharmaceutical Inc
2016 Phase 3 NCT02736188 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.
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
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
Suzuki Ryoichi
2021 Phase 3 JPRN-jRCT2021200030 -
Tohoku University
2010 Phase 1 NCT01236898 Japan
Sialic acid
ULTRAGENYX PHARMACEUTICAL INC.
2015 Phase 3 EUCTR2014-005432-33-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
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
Suzuki Ryoichi
2021 Phase 3 JPRN-jRCT2021200030 -
ULTRAGENYX PHARMACEUTICAL INC.
2017 Phase 3 EUCTR2016-000360-42-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom
Aceneuramic acid extended-release
Ultragenyx Pharmaceutical Inc
2016 Phase 2 NCT02731690 Bulgaria;Canada;United States
2015 Phase 3 NCT02377921 Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
Aceneuramic acid extended-release tablets
Ultragenyx Pharmaceutical Inc
2016 Phase 3 NCT02736188 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.
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
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
Suzuki Ryoichi
2021 Phase 3 JPRN-jRCT2021200030 -
Tohoku University
2010 Phase 1 NCT01236898 Japan
Sialic acid
ULTRAGENYX PHARMACEUTICAL INC.
2015 Phase 3 EUCTR2014-005432-33-IT Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States
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