344. Very long-chain acyl-CoA dehydrogenase deficiency Disease details / Clinical trials / Drug dev / DR info /

Clinical trials : 15 /  Drugs : 25 - (DrugBank : 4) / Drug target genes : 2 - Drug target pathways : 11

Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
Bezafibrate
   Rigshospitalet, Denmark
      2009   Phase 2   NCT00983788   Denmark
Exercise
   University Medical Center Groningen
      2016   -   NCT03531554   Netherlands
Glycerol/saline
   Oregon Health and Science University
      2016   -   NCT02517307   United States
Hyperinsulinemic euglycemic clamp
   Oregon Health and Science University
      2016   -   NCT02517307   United States
Intralipid/heparin
   Oregon Health and Science University
      2016   -   NCT02517307   United States
Ketone ester drink
   University Medical Center Groningen
      2016   -   NCT03531554   Netherlands
Magnetic resonance imaging
   University Medical Center Groningen
      2016   -   NCT03531554   Netherlands
MCT OIL
   Kajiwara Makoto
      2025   Phase 3   JPRN-jRCT2031240472   Czech Republic;Germany;Japan;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
   Ultragenyx Pharmaceutical Inc
      2023   Phase 3   NCT05933200   Czechia;Germany;Japan;Poland;Saudi Arabia;Spain;Turkey (Türkiye);United Kingdom
Medium-chain triglycerides
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2022-001539-10-PL   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Türkiye;United Kingdom
      2023   Phase 3   EUCTR2022-001539-10-CZ   Czech Republic;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2022   Phase 3   EUCTR2022-001539-10-ES   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      -   Phase 3   EUCTR2022-001539-10-DE   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
Miglyol® 812 N
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2022-001539-10-PL   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Türkiye;United Kingdom
      2023   Phase 3   EUCTR2022-001539-10-CZ   Czech Republic;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2022   Phase 3   EUCTR2022-001539-10-ES   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      -   Phase 3   EUCTR2022-001539-10-DE   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
Muscle biopsy
   University Medical Center Groningen
      2016   -   NCT03531554   Netherlands
Nutritional ketone supplement
   Oregon Health and Science University
      2022   Early Phase 1   NCT05411835   United States
Obtaining DRY blood spots from healthy children aged 8 days - 7 years
   West Kazakhstan Medical University
      2022   -   NCT05910151   Kazakhstan
Obtaining DRY blood spots from healthy children aged 8-18 years
   West Kazakhstan Medical University
      2022   -   NCT05910151   Kazakhstan
Triglycerides, medium chain
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2022-001539-10-PL   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Türkiye;United Kingdom
      2023   Phase 3   EUCTR2022-001539-10-CZ   Czech Republic;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2022   Phase 3   EUCTR2022-001539-10-ES   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      -   Phase 3   EUCTR2022-001539-10-DE   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
Triheptanoin
   Kajiwara Makoto
      2025   Phase 3   JPRN-jRCT2031240472   Czech Republic;Germany;Japan;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
   Ultragenyx Pharmaceutial Inc.
      2016   Phase 2   EUCTR2016-000322-19-GB   United Kingdom;United States
   Ultragenyx Pharmaceutical Inc
      2023   Phase 3   NCT05933200   Czechia;Germany;Japan;Poland;Saudi Arabia;Spain;Turkey (Türkiye);United Kingdom
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2022-001539-10-PL   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Türkiye;United Kingdom
      2023   Phase 3   EUCTR2022-001539-10-CZ   Czech Republic;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2022   Phase 3   EUCTR2022-001539-10-ES   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2014   Phase 2   EUCTR2013-004830-14-GB   United Kingdom;United States
      -   Phase 3   EUCTR2022-001539-10-DE   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
UX007
   Ultragenyx Pharmaceutial Inc.
      2016   Phase 2   EUCTR2016-000322-19-GB   United Kingdom;United States
   Ultragenyx Pharmaceutical Inc
      2014   Phase 2   NCT02214160   United Kingdom;United States
      2014   Phase 2   NCT01886378   United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2022-001539-10-PL   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Türkiye;United Kingdom
      2023   Phase 3   EUCTR2022-001539-10-CZ   Czech Republic;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2022   Phase 3   EUCTR2022-001539-10-ES   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom
      2014   Phase 2   EUCTR2013-004830-14-GB   United Kingdom;United States
      -   Phase 3   EUCTR2022-001539-10-DE   Czech Republic;Czechia;Germany;Poland;Saudi Arabia;Spain;Turkey;United Kingdom