U.S., April 16 -- ClinicalTrials.gov registry received information related to the study (NCT07530796) titled 'Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)' on April 01.
Brief 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).
Study Start Date: May 01
Study Type: INTERVENTIONAL
Condition:
Aspartylglucosaminuria
Aspartylglucosamidase (AGA) Deficiency
Intervention:
GENETIC: scAAV9/AGA
Danagalex, a self-complementary adeno-associated Virus Serotype 9 (AAV9) vector-mediated gene transfer of codon optimized human AGA gene, administered via intrathecal injection
R...