Phase II investigator-initiated clinical trial of RE-01 cell therapy for refractory ischemic (non-arteriosclerotic) lower extremity ulcers caused by Buerger's disease and collagen diseases initiated.

当社は、リィエイル社との間で製造業務委受託基本契約を締結し、子会社である株式会社ロートセルファクトリー東京（本社：東京都墨田区、社長：杉本昇平、以下「ロートセルファクトリー東京」）にて、治験製品RE-01を製造します。本治験は、先行して実施された第I相医師主導治験に続く第II相試験です。バージャー病および膠原病により生じる難治性虚血性下肢潰瘍患者を対象に、RE-01を用いた治療の有効性および安全性を評価します。標準治療群を対照とする無作為化比較試験として、多施設共同で実施されます。

Background of Research and Development

Intractable ischemic lower limb ulcers caused by Buerger's disease and collagen diseases are primarily due to vasculitis and microvascular damage, and there is no effective treatment for them. If they worsen, lower limb amputation becomes unavoidable. Because the age of onset is young, many people experience significant difficulties in their daily lives during their prime working years. There is a great unmet medical need for the development of treatments that improve intractable ulcers and prevent amputation, in order to improve quality of life (QOL), reduce the burden of living difficulties and medical expenses, and enable early social reintegration.
Based on research results from Juntendo University, Reail Inc. is developing Regenerative medicine products using culture technology that amplifies functional regenerative cells from small amounts of peripheral blood. For RE-01, a Phase I investigator-initiated clinical trial (an exploratory study to examine the safety and efficacy of multiple administrations, jRCT2033220413) targeting patients with refractory ischemic lower limb ulcers caused by Buerger's disease and collagen diseases has been completed, and further development as a Regenerative medicine product is expected.

About the investigational product (RE-01)

RE-01 is a group of autologous peripheral blood cultured mononuclear cells produced using a culture method developed by a research group led by Professor Rika Tanaka of the Department of Regenerative Medicine, Graduate School of Medicine, Juntendo University. It uses only peripheral blood collected from the patient themselves as raw material, and does not use allogeneic cells or animal-derived cell lines. It is manufactured in a short-term process without medium changes or subculturing, and after manufacturing, it is transported to medical institutions under refrigeration. RE-01 is being developed by Re-Aile Co., Ltd. for the treatment of intractable ischemic lower limb ulcers caused by microvascular damage resulting from Buerger's disease and collagen diseases.
Our company has a manufacturing outsourcing agreement with Liayle Co., Ltd., and our subsidiary, Rohto Cell Factory Tokyo, manufactures the investigational products.

Overview of the Clinical Trial

• Target number of cases: 10 cases (RE-01 group: 5 cases, standard treatment group: 5 cases)
• Clinical trial period: June 2026 - September 2028
• Participating medical institutions: Juntendo University Hospital, Kobe University Hospital, Gunma University Hospital, Keiwakai Oita Oka Hospital

In this Phase II clinical trial, patients with Buerger's disease and refractory ischemic lower extremity ulcers caused by collagen diseases, for whom conventional treatments are insufficient and revascularization is difficult, will be randomly assigned to either a group that continues existing standard treatment and receives RE-01 three times at 4-week intervals, or a group that continues existing standard treatment. The efficacy and safety will be compared from baseline to approximately 5 months.
For details, please refer to the Japan Clinical Research Trials (jRCT) submission and publication system.
We express our sincere gratitude to everyone who is cooperating in this clinical trial.

Our role in this clinical trial

As the manufacturer of the investigational product, our subsidiary, Rohto Cell Factory Tokyo, is responsible for the planning, manufacturing, testing, packaging, storage, and shipping of the investigational product RE-01, and will continuously manufacture RE-01 throughout the trial period.

About Rohto Cell Factory Tokyo Co., Ltd.