NCT07301164

Clinical Study of BCT301 Cell Injection Therapy for Refractory Autoimmune Diseases

A Study of BCT301 (Anti-CD19 Chemically Induced Pluripotent Stem Cell (CiPSC)-Derived CAR-iT Cells) Therapy for Refractory Autoimmune Diseases

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Peking University Third Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.

Detailed description

Autoimmune diseases are a class of disorders caused by abnormal immune responses against the body's own tissues or organs. Although significant therapeutic advances have been made, such as the use of biologics (e.g., rituximab), most available drugs can only alleviate symptoms or slow disease progression, rather than achieve a complete cure.BCT301 cell injection is a CD19-targeted CAR-iT (chemically induced T-cell) therapy derived from human chemically induced pluripotent stem cells (CiPSCs) through chemical reprogramming technology. Starting from healthy donor cells, human CiPSCs are generated via small molecule-based reprogramming, followed by induced hematopoietic differentiation, iT-cell differentiation and expansion, and finally CAR lentiviral transduction to produce the off-the-shelf, CD19-targeted CAR-iT cell product (BCT301 cell injection). This approach holds promise as a potentially effective treatment strategy.

Conditions

• System Lupus Erythematosus
• Systemic Sclerosis (SSc)
• Inflammatory Myositis
• Antiphospholipid Syndrome
• ANCA Associated Vasculitis
• Sjogren Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	BCT301	BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells.Participants will receive a single infusion of BCT301 cell injection at escalating dose levels.Prior to infusion, patients undergo a lymphodepleting conditioning regimen with fludarabine and cyclophosphamide given intravenously for three consecutive days.

Timeline

• Start date: 2025-12-11
• Primary completion: 2027-12-31
• Completion: 2028-12-31
• First posted: 2025-12-24
• Last updated: 2025-12-24