Aortic & peripheral vascular disease
Anatomically Directed Lower Extremity Gene Therapy for Ulcer Healing: A Double-Blind, Randomized, Placebo-Controlled Study (LEGenD-1)
Authors' conclusion
Anatomically targeted intramuscular delivery of AMG0001 significantly accelerated healing in patients with moderate chronic limb-threatening ischemia and neuroischemic ulcers and may represent a promising nonsurgical therapeutic strategy.
Study context
- Population
- Patients with chronic limb-threatening ischemia and neuroischemic ulcers, toe pressure or transcutaneous oxygen pressure 30–59 mm Hg
- Participants
- 75 patients
- Comparison
- AMG0001 vs placebo
- Primary endpoint
- Coprimary: time to complete healing and proportion with ulcers healed (pooled AMG0001 analysis)
- Follow-up
- 6 months
Methodological detailsdouble-blind, placebo-controlled, phase II, 22 US sites
- Double-blind
- Placebo-controlled
- Phase II
- 22 US sites
Primary result
As reported in the abstract
Values
Healed by 6 months AMG0001 (pooled) 63.3% · Placebo 38.5% · P = 0.053
- AMG0001 (pooled) vs Placebo: P = 0.007 — median time to healing
- AMG0001 4 mg vs Placebo: P = 0.017 — median time to healing
- AMG0001 8 mg vs Placebo: P = 0.022 — median time to healing
Secondary outcomes
| Outcome | P value |
|---|---|
| Ulcer healing rate, AMG0001 (pooled) vs placebo · 12 monthsHealing rates 77.6% vs 46.2% | P = 0.010 |
Safety
- Adverse events similar across groups
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
Anatomically Directed Lower Extremity Gene Therapy for Ulcer Healing: A Double-Blind, Randomized, Placebo-Controlled Study (LEGenD-1). Circ Cardiovasc Interv. Published 4 November 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →