245. プロピオン酸血症 Propionic acidemia Clinical trials / Disease details


臨床試験数 : 13 薬物数 : 17 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 6

  
1 trial found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
1NCT04732429
(ClinicalTrials.gov)
March 15, 202121/1/2021Study of HST5040 in Subjects With Propionic or Methylmalonic AcidemiaA Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension StudyMethylmalonic Acidemia;Propionic AcidemiaDrug: HST5040;Drug: PlaceboHemoShear TherapeuticsNULLRecruiting2 YearsN/AAll12Phase 2United States