Recently, the “Long-term Follow-up Study of Subjects in the NL003 Phase II Clinical Trial,” initiated by the team led by Professor Gu Yongquan from the Department of Vascular Surgery at Xuanwu Hospital, has been approved and officially entered the launch phase at Xuanwu Hospital of Capital Medical University. This project is conducted as a investigator-initiated trial, with the participation of nine research centers that took part in the Phase II clinical trial. The study is scheduled to be completed by December 2022.

This study was conducted among subjects enrolled in the Phase II clinical trial of NL003 (Recombinant Human Hepatocyte Growth Factor Naked Plasmid Injection). The Phase II clinical trial was a randomized, double-blind, placebo-controlled study involving patients with critical limb ischemia. The results preliminarily confirmed the safety and efficacy of NL003 for the treatment of severe lower-limb ischemia and were published in a peer-reviewed paper (Yongquan Gu, et al. Mol Ther. 2019 Dec 4;27(12):2158-2165).

This long-term follow-up study was designed in accordance with the "Good Clinical Practice for Drug Clinical Trials" and the "Technical Guidance Principles for Long-Term Follow-up Clinical Studies of Gene Therapy Products" issued by the NMPA. The clinical trial protocol employs a combination of on-site and remote visits to assess indicators such as amputation rates, mortality rates, and the incidence of major medical events among patients who received NL003 treatment in the Phase II clinical trial, thereby evaluating the long-term efficacy and safety over a period of 5 to 7 years.

Phase II of NL003 The clinical trial has initially evaluated the efficacy and safety of NL003 for the treatment of patients with severe lower-limb ischemia; however, the observation period was relatively short, lasting only 180 days. The purpose of this follow-up study is to assess the long-term efficacy of NL003 as well as any potential adverse reactions, thereby further elucidating the drug’s mechanism of action, its long-term effectiveness, and its possible risks. This study will also provide reference data for the new drug review process of NL003.