Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.
J Inherit Metab DisSep 1, 2026
John J Mitchell, Jose E Abdenur, Foekje de Boer et al.
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In 30 seconds
This phase 3 randomized controlled trial evaluated the efficacy of DTX401 gene therapy in patients aged 8 years and older with glycogen storage disease type Ia (GSDIa). The study found a significant reduction in daily cornstarch intake, with a 41% decrease in the DTX401 group compared to a 10% decrease in the placebo group (p < 0.0001) after 48 weeks.
Key findings
- DTX401 treatment led to a 41% reduction in daily cornstarch intake compared to 10% for placebo (p < 0.0001).
- A mean desired percent reduction in daily cornstarch intake was 45% among participants at baseline.
- Greater reductions in cornstarch intake were observed at Week 96 for the crossover DTX401 group.
Why it matters
The findings suggest that DTX401 gene therapy may provide a significant therapeutic benefit for patients with GSDIa, potentially improving dietary management and quality of life. Understanding its efficacy and safety profile is crucial for clinicians managing this rare metabolic disorder.