AAV9 BBP-812 ( DrugBank: BBP-812 )


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
1NCT04998396
(ClinicalTrials.gov)
September 8, 20215/8/2021A Study of AAV9 Gene Therapy in Participants With Canavan DiseaseA Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA GeneCanavan DiseaseBiological: AAV9 BBP-812Aspa TherapeuticsNULLRecruitingN/A30 MonthsAll18Phase 1/Phase 2United States