International Clinical Trial: Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

Geneva, April 27 -- International Clinical Trials Registry received information related to the study (NCT07530796) titled 'Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)' on April 1.

Study Type: Interventional

Study Design: Allocation: N/A. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).

Primary Sponsor: Rare Trait Hope

Condition: Aspartylglucosaminuria Aspartylglucosamidase (AGA) Deficiency

Intervention: Genetic: scAAV9/AGA

Recruitment Status: Not recruiting

Phase: Phase 1/Phase 2

Date of First Enrollment: May 1, 2026

Target Sample Size: 9

To know more, visit https://clinicaltrials.gov/study/NCT07530796

Disclaimer: Curat...