307. Canavan disease Clinical trials / Disease details


Clinical trials : 6 Drugs : 11 - (DrugBank : 5) / Drug target genes : 2 - Drug target pathways : 2

  
1 trial found
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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