307. カナバン病 Canavan disease Clinical trials / Disease details


臨床試験数 : 5 薬物数 : 9 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

  
1 trial found
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1NCT04833907
(ClinicalTrials.gov)
April 1, 202124/3/2021rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan DiseasePhase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of AVASPA Gene Therapy for Treatment of Children With Typical Canavan DiseaseCanavan DiseaseDrug: rAAV-Olig001-ASPA;Drug: Levetiracetam;Drug: PrednisoloneMyrtelle Inc.NULLRecruiting3 Months60 MonthsAll24Phase 1/Phase 2United States