234. Peroxisomal disease (except Adrenoleukodystrophy) Disease details / Clinical trials / Drug dev / DR info

Clinical trials : 39 /  Drugs : 35 - (DrugBank : 12) / Drug target genes : 13 - Drug target pathways : 45

Drugs and their primary sponsors and trial info
ALN-65585   
   Alnylam Pharmaceuticals, Inc.
      2018   Phase 2   EUCTR2016-003134-24-NL   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003134-24-GB   France;Germany;Israel;Netherlands;United Kingdom;
      2017   Phase 2   EUCTR2016-003134-24-FR   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003134-24-DE   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
      2017   Phase 1;Phase 2   EUCTR2015-004407-23-NL   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004407-23-GB   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
      -   Phase 1;Phase 2   EUCTR2015-004407-23-DE   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
ALN-GO1   
   ALNYLAM PHARMACEUTICALS, INC.
      2021   Phase 3   EUCTR2019-001346-17-IT   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
   Alnylam Pharmaceuticals, Inc.
      2020   Phase 3   EUCTR2019-001346-17-GB   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      2019   Phase 3   EUCTR2019-001346-17-NL   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      2019   Phase 3   EUCTR2019-001346-17-BE   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-004014-17-GB   France;Germany;Israel;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-004014-17-FR   France;Germany;Israel;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-004014-17-DE   France;Germany;Israel;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001981-40-NL   France;Germany;Israel;Japan;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2018   Phase 3   EUCTR2018-001981-40-GB   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2018   Phase 3   EUCTR2018-001981-40-DE   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-003134-24-NL   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003134-24-GB   France;Germany;Israel;Netherlands;United Kingdom;
      2017   Phase 2   EUCTR2016-003134-24-FR   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003134-24-DE   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
      2017   Phase 1;Phase 2   EUCTR2015-004407-23-NL   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004407-23-GB   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
      -   Phase 3   EUCTR2019-001346-17-FR   Australia;Belgium;Egypt;France;Germany;India;Israel;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      -   Phase 3   EUCTR2019-001346-17-DE   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      -   Phase 1;Phase 2   EUCTR2015-004407-23-DE   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
CA   
   Department of Pediatric Gastroenterology, Academic Medical Centre
      -   Phase 2   EUCTR2010-022046-25-NL   Netherlands;
CALD HR-D (High-Risk, Regimen C)   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
CALD HR-D (High-Risk, Regimen D)   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
CALD SR-A (Standard-Risk, Regimen A)   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
CALD SR-B (Standard-Risk, Regimen B)   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Campath-1H   
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States;
Chenodeoxycholic acid   
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
Cholic Acids   
   Travere Therapeutics, Inc.
      1992   Phase 3   NCT00007020   United States;
Clofarabine   
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States;
Cyclosporine A   
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States;
DCR-1171X   
   Dicerna Pharmaceuticals, Inc.
      2016   Phase 1   EUCTR2015-003142-51-NL   France;Germany;Israel;Netherlands;United Kingdom;United States;
      2016   Phase 1   EUCTR2015-003142-51-GB   France;Germany;Israel;Netherlands;United Kingdom;United States;
DCR-L1360   
   Dicerna Pharmaceuticals Inc
      2021   Phase 2   EUCTR2020-002826-97-FR   Australia;France;Germany;Italy;Japan;Romania;Spain;United Kingdom;United States;
      2021   Phase 2   EUCTR2020-002826-97-DE   France;Germany;Italy;Romania;Spain;United Kingdom;United States;
   Dicerna Pharmaceuticals Inc.
      2021   Phase 2   EUCTR2020-002826-97-IT   Australia;France;Germany;Italy;Romania;Spain;United Kingdom;United States;
DCR-PH1   
   Dicerna Pharmaceuticals, Inc.
      2016   Phase 1   NCT02795325   Germany;Netherlands;
      2016   Phase 1   EUCTR2015-003142-51-NL   France;Germany;Israel;Netherlands;United Kingdom;United States;
      2016   Phase 1   EUCTR2015-003142-51-GB   France;Germany;Israel;Netherlands;United Kingdom;United States;
DCR-PHXC   
   Dicerna Pharmaceuticals Inc
      2021   Phase 2   EUCTR2020-002826-97-FR   Australia;France;Germany;Italy;Japan;Romania;Spain;United Kingdom;United States;
      2021   Phase 2   EUCTR2020-002826-97-DE   France;Germany;Italy;Romania;Spain;United Kingdom;United States;
   Dicerna Pharmaceuticals Inc.
      2021   Phase 2   EUCTR2020-002826-97-IT   Australia;France;Germany;Italy;Romania;Spain;United Kingdom;United States;
   Dicerna Pharmaceuticals, Inc.
      2021   Phase 2   NCT04580420   Spain;United Kingdom;United States;
      2019   Phase 3   NCT04042402   Australia;Canada;France;Germany;Italy;Japan;Lebanon;Netherlands;Spain;United Kingdom;United States;
      2019   Phase 2   NCT03847909   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States;
Hematopoietic stem cell transplantation   
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States;
Hydroxychloroquine   
   The Hospital for Sick Children
      2019   Phase 2   NCT03856866   Canada;
IMD Preparative Regimen   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Lumasiran   
   ALNYLAM PHARMACEUTICALS, INC.
      2021   Phase 3   EUCTR2019-001346-17-IT   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
   Alnylam Pharmaceuticals
      2020   Phase 3   NCT04152200   Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      2019   Phase 3   NCT03905694   France;Germany;Israel;United Kingdom;United States;
      2019   -   NCT04125472   Belgium;Canada;France;French Polynesia;Germany;Italy;Netherlands;Spain;Switzerland;United Kingdom;United States;
      2018   Phase 3   NCT03681184   France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2018   Phase 2   NCT03350451   France;Germany;Israel;Netherlands;United Kingdom;
      2016   Phase 1/Phase 2   NCT02706886   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States;
   Alnylam Pharmaceuticals, Inc.
      2020   Phase 3   EUCTR2019-001346-17-GB   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001981-40-NL   France;Germany;Israel;Japan;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2018   Phase 3   EUCTR2018-001981-40-GB   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2018   Phase 3   EUCTR2018-001981-40-DE   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003134-24-GB   France;Germany;Israel;Netherlands;United Kingdom;
      2017   Phase 2   EUCTR2016-003134-24-DE   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States;
Melphalan   
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States;
Mycophenolate mofetil   
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States;
Nedosiran   
   Dicerna Pharmaceuticals Inc
      2021   Phase 2   EUCTR2020-002826-97-FR   Australia;France;Germany;Italy;Japan;Romania;Spain;United Kingdom;United States;
      2021   Phase 2   EUCTR2020-002826-97-DE   France;Germany;Italy;Romania;Spain;United Kingdom;United States;
   Dicerna Pharmaceuticals Inc.
      2021   Phase 2   EUCTR2020-002826-97-IT   Australia;France;Germany;Italy;Romania;Spain;United Kingdom;United States;
   Dicerna Pharmaceuticals, Inc.
      2021   Phase 2   NCT05001269   -
Osteopetrosis Haploidentical Only Preparative Regimen   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Osteopetrosis Only Preparative Regimen   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Stem Cell Transplantation   
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Sterile Normal Saline ( NaCl)   
   Dicerna Pharmaceuticals, Inc.
      2019   Phase 2   NCT03847909   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States;
Ursodiol   
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -