RAAV-Olig001-ASPA ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
307カナバン病1

307. カナバン病


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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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