NCT07530796

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

An Open-Label, Single Center, Phase 1/2 Study to Evaluate the Safety and Efficacy of DANAGALEX (scAAV9/AGA) in Participants With Aspartylglucosaminuria (AGU)

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Rare Trait Hope · Academic / Other
• Sex: All
• Age: 4 Years – 45 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This study is a first in human (FIH) open-label study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding aspartylglucosaminidase (AGA) in participants with Aspartylglucosaminuria (AGU). Safety will be monitored continuously throughout the study for adverse / serious adverse events and dose limiting toxicities. All outcomes (primary, secondary, exploratory) will be assessed at 52 and 104 weeks.

Conditions

• Aspartylglucosaminuria
• Aspartylglucosamidase (AGA) Deficiency

Interventions

Type	Name	Description
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

Timeline

• Start date: 2026-05-01
• Primary completion: 2029-05-01
• Completion: 2032-05-01
• First posted: 2026-04-15
• Last updated: 2026-04-15

Regulatory

• FDA-regulated drug study