RAAV9-CB6-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 : 6 薬物数 : 11 - (DrugBank : 5) / 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
1NCT05317780
(ClinicalTrials.gov)
February 10, 202024/3/2022Canavan-Single Patient INDExpanded Access Trial of Systemic Delivery of Aspartoacylase ASPA (rAAV9-CB6-AspA) Gene Vector in a Single Patient With Canavan DiseaseCanavan DiseaseDrug: rAAV9-CB6-AspABarry J. ByrneUniversity of Miami;University of Massachusetts, WorcesterAvailable18 Months24 MonthsMaleUnited States