276. 軟骨無形成症 Disease details / Clinical trials / Drug dev / DR info

BMN 111
   BioMarin Pharmaceutical
      2019   Phase 2   NCT03989947   Australia;Japan;United Kingdom;United States
      2018   Phase 2   NCT03583697   Australia;Japan;United Kingdom;United States
      2017   Phase 3   NCT03424018   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   NCT03197766   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2016   Phase 2   NCT02724228   Australia;France;United Kingdom;United States
      2014   Phase 2   NCT02055157   Australia;France;United Kingdom;United States
      2012   Phase 1   NCT01590446   United States
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States
   BioMarin Pharmaceutical Inc.(ICCC:EPS Corporation)
      2019   Phase 2   JPRN-jRCT2080224833   Europe;Japan;North America;Oceania
   BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)
      2018   Phase 3   JPRN-jRCT2080224106   Europe;Japan;North America;Oceania
BMN 111 administration VIA injector PEN
   BioMarin Pharmaceutical
      2023   Phase 1   NCT05813314   United States
BMN 111 administration VIA vial and syringe
   BioMarin Pharmaceutical
      2023   Phase 1   NCT05813314   United States
C-type natriuretic peptide conjugated TO A multi-ARM polyethylene glycol carrier molecule through A cleavable linker
   Ascendis Pharma Growth Disorders A/S
      2023   Phase 2   EUCTR2022-002954-25-DK   Australia;Canada;Denmark;Ireland;New Zealand;Spain;United Kingdom;United States
C-type natriuretic peptide conjugated TO multi-ARM polyethylene glycol carrier through A cleavable linker
   Ascendis Pharma Growth Disorders A/S
      2023   Phase 2   EUCTR2022-002954-25-DK   Australia;Canada;Denmark;Ireland;New Zealand;Spain;United Kingdom;United States
      2021   Phase 2   EUCTR2019-002754-22-PT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002754-22-DK   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002754-22-DE   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002754-22-AT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2019   Phase 2   EUCTR2019-002754-22-IE   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      -   Phase 2   EUCTR2019-002754-22-GB   Australia;Austria;Canada;Denmark;Germany;Ireland;Italy;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
Combination OF navepegritide and lonapegsomatropin administered AS TWO separate S.C. injections
   Ascendis Pharma Growth Disorders A/S
      2024   Phase 2   NCT06433557   Denmark;Ireland;United Kingdom
Infigratinib
   QED Therapeutics, Inc.
      2022   Phase 2   EUCTR2021-001855-15-FR   Australia;Canada;France;Spain;United Kingdom;United States
      2022   Phase 2   EUCTR2021-001855-15-ES   Australia;Canada;France;Spain;United Kingdom;United States
      2021   Phase 2   NCT05145010   Australia;Canada;France;Singapore;Spain;United Kingdom;United States
Infigratinib 0.016 MG/KG
   QED Therapeutics, Inc.
      2020   Phase 2   NCT04265651   Australia;Canada;France;Spain;United Kingdom;United States
Infigratinib 0.032 MG/KG
   QED Therapeutics, Inc.
      2020   Phase 2   NCT04265651   Australia;Canada;France;Spain;United Kingdom;United States
Infigratinib 0.064 MG/KG
   QED Therapeutics, Inc.
      2020   Phase 2   NCT04265651   Australia;Canada;France;Spain;United Kingdom;United States
Infigratinib 0.128 MG/KG
   QED Therapeutics, Inc.
      2020   Phase 2   NCT04265651   Australia;Canada;France;Spain;United Kingdom;United States
Infigratinib 0.25 MG/KG
   QED Therapeutics, Inc.
      2020   Phase 2   NCT04265651   Australia;Canada;France;Spain;United Kingdom;United States
Infigratinib 0.25 MG/KG/DAY
   QED Therapeutics, Inc.
      2023   Phase 3   NCT06164951   Argentina;Australia;Canada;France;Germany;Italy;Norway;Singapore;Spain;United Kingdom;United States
Infigratinib phosphate
   QED Therapeutics, Inc.
      2020   Phase 2   EUCTR2019-002954-21-GB   Australia;Canada;France;Spain;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002954-21-FR   Australia;Canada;France;Spain;United Kingdom;United States
KK8398
   Kyowa Kirin Co., Ltd.
      2024   Phase 3   NCT06926491   Japan
Modified recombinant human C-type
   BioMarin Pharmaceutical Inc.
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States
Modified recombinant human C-type natriuretic peptide
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States
   BioMarin Pharmaceutical Inc.(ICCC:EPS Corporation)
      2019   Phase 2   JPRN-jRCT2080224833   Europe;Japan;North America;Oceania
   BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)
      2018   Phase 3   JPRN-jRCT2080224106   Europe;Japan;North America;Oceania
Modified rhcnp
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States
Natriuretic peptide
   Ascendis Pharma Growth Disorders A/S
      2023   Phase 2   EUCTR2022-002954-25-IE   Australia;Canada;Denmark;Ireland;New Zealand;Spain;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States
Navepegritide
   Ascendis Pharma A/S
      2024   Phase 2   NCT06732895   Denmark;France
      2024   Phase 2   NCT06079398   Australia;Austria;Denmark;Finland;France;Germany;Ireland;Italy;New Zealand;Norway;Portugal;Sweden;United Kingdom;United States
Normal saline
   BioMarin Pharmaceutical
      2012   Phase 1   NCT01590446   United States
PF-07256472
   PFIZER INC
      2021   Phase 2   EUCTR2020-001189-13-IT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States
   Pfizer Inc.
      2022   Phase 2   EUCTR2021-003149-39-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      2021   Phase 2   EUCTR2021-003149-39-ES   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      2021   Phase 2   EUCTR2020-001189-13-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States
      -   Phase 2   EUCTR2021-003149-39-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      -   Phase 2   EUCTR2021-003149-39-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
   Pfizer Inc., 235 East 42nd Street, New York, NY 10017
      2020   Phase 2   EUCTR2020-001189-13-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      -   Phase 2   EUCTR2020-001189-13-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
Recifercept
   Kawai Norisuke
      2022   Phase 2   JPRN-jRCT2061220040   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
   PFIZER INC
      2021   Phase 2   EUCTR2020-001189-13-IT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States
   Pfizer
      2022   -   NCT05659719   United States
      2021   Phase 2   NCT05116046   Australia;Belgium;Denmark;Italy;Portugal;Spain;United States
      2020   Phase 2   NCT04638153   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
   Pfizer Inc.
      2022   Phase 2   EUCTR2021-003149-39-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      2021   Phase 2   EUCTR2021-003149-39-ES   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      2021   Phase 2   EUCTR2020-001189-13-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States
      -   Phase 2   EUCTR2021-003149-39-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      -   Phase 2   EUCTR2021-003149-39-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
   Pfizer Inc., 235 East 42nd Street, New York, NY 10017
      2020   Phase 2   EUCTR2020-001189-13-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      -   Phase 2   EUCTR2020-001189-13-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
Recombinant human growth hormone
   GeneScience Pharmaceuticals Co., Ltd.
      2022   Phase 4   NCT05353192   China
SAR442501
   Sanofi
      2023   Phase 2   NCT06067425   Australia;China;Italy;Korea, Republic of;Spain
Somatropin
   Novo Nordisk A/S
      2012   -   NCT01435629   Japan
      1997   -   NCT01516229   Japan
TA-46
   Pfizer Inc.
      2022   Phase 2   EUCTR2021-003149-39-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      2021   Phase 2   EUCTR2021-003149-39-ES   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      2021   Phase 2   EUCTR2020-001189-13-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States
      -   Phase 2   EUCTR2021-003149-39-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      -   Phase 2   EUCTR2021-003149-39-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
   Pfizer Inc., 235 East 42nd Street, New York, NY 10017
      2020   Phase 2   EUCTR2020-001189-13-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
      -   Phase 2   EUCTR2020-001189-13-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States
TBD
   BioMarin Pharmaceutical Inc.
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States
Transcon CNP
   Ascendis Pharma A/S
      2022   Phase 2   NCT05246033   China
      2020   Phase 2   NCT04085523   Australia;Austria;Denmark;Germany;Ireland;New Zealand;Portugal;United States
   Ascendis Pharma Growth Disorders A/S
      2023   Phase 2/Phase 3   NCT05929807   Australia;Austria;Denmark;Germany;Ireland;New Zealand;Portugal;United States
      2023   Phase 2/Phase 3   NCT05598320   Australia;Canada;Denmark;Ireland;New Zealand;Spain;United States
      2023   Phase 2   EUCTR2022-002954-25-IE   Australia;Canada;Denmark;Ireland;New Zealand;Spain;United Kingdom;United States
Transcon CNP 3.9 MG CNP-38/vial
   Ascendis Pharma Growth Disorders A/S
      2023   Phase 2   EUCTR2022-002954-25-DK   Australia;Canada;Denmark;Ireland;New Zealand;Spain;United Kingdom;United States
      2021   Phase 2   EUCTR2019-002754-22-PT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002754-22-DK   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002754-22-DE   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-002754-22-AT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      2019   Phase 2   EUCTR2019-002754-22-IE   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
      -   Phase 2   EUCTR2019-002754-22-GB   Australia;Austria;Canada;Denmark;Germany;Ireland;Italy;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States
TYRA-300 0.125 MG/KG
   Tyra Biosciences, Inc
      2025   Phase 2   NCT06842355   United States
TYRA-300 0.25 MG/KG
   Tyra Biosciences, Inc
      2025   Phase 2   NCT06842355   United States
TYRA-300 0.375 MG/KG
   Tyra Biosciences, Inc
      2025   Phase 2   NCT06842355   United States
TYRA-300 0.50 MG/KG
   Tyra Biosciences, Inc
      2025   Phase 2   NCT06842355   United States
Vorsoritide
   BioMarin Pharmaceutical Inc.
      2017   Phase 1   EUCTR2017-000701-21-DE   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States
Vosoritide
   BioMarin Pharmaceutical
      2020   Phase 2   NCT04554940   Australia;United Kingdom
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States
   BioMarin Pharmaceutical Inc.(ICCC:EPS Corporation)
      2019   Phase 2   JPRN-jRCT2080224833   Europe;Japan;North America;Oceania
   BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)
      2018   Phase 3   JPRN-jRCT2080224106   Europe;Japan;North America;Oceania
Voxzogo
   BioMarin Pharmaceutical Inc.
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States