Clinical Trial

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

Name: Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) — clinical trial record (NCT07530796)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Not Yet Recruiting Phase 1/2

View on ClinicalTrials.gov →

Summary

The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).

Trial Details

NCT Number	NCT07530796
Lead Sponsor	Rare Trait Hope
Conditions	Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
Enrollment	9 participants
Start Date	2026-05-01
Primary Completion	2029-05-01 (estimated)
Study Completion	2032-05-01 (estimated)
Updated on ClinicalTrials.gov	2026-04-15