RN1201 Injection in the Treatment of Antibody-Mediated Diseases

NCT07072247 | Not Yet Recruiting Phase 1

• 1 other identifier: RN1201-Ab
• Study Type: interventional
• Target: 9
• Locations: 0 countries
• Sites: N/A

Brief Summary

This single-arm, open-label exploratory trial aims to evaluate the safety, feasibility, and preliminary efficacy of RN1201 Injection in patients with antibody-mediated diseases, including refractory immune-mediated platelet transfusion refractoriness (PTR) and relapsed or refractory immune thrombocytopenia (ITP). Patients will receive RN1201 cells infusion following lymphodepletion. The study will assess safety, response rates, B-cell depletion, and immune reconstitution. Exploratory analyses will examine in vivo persistence and activity of RN1201.

Conditions

Refractory Immune-mediated Platelet Transfusion Refractoriness; Relapsed or Refractory Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• The incidence and severity of treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs)

  To characterize the safety of RN1201 Cells for Antibody-mediated Diseases

  within 4 weeks after infusion

Secondary Outcomes (3)

• Objective Response Rate (ORR)

  From infusion up to 12 months post-treatment.

• Pharmacokinetic (PK) of RN1201

  From infusion up to 12 months post-treatment.

• Pharmacodynamic (PD)-B cell depletion

  From infusion up to 12 months post-treatment.

Study Arms (1)

Allogeneic CAR-T cell therapy

EXPERIMENTAL

Antibody-Mediated Diseases treated with RN1201 Injection

Genetic: CD19 and BCMA-targeted allogeneic CAR-T cells

Interventions

CD19 and BCMA-targeted allogeneic CAR-T cells GENETIC

Lymphodepletion chemotherapy followed by CD19 and BCMA-targeted allogeneic CAR-T cells infusion

Also known as: RN1201

Allogeneic CAR-T cell therapy

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 16-65 years
• Diagnosed with refractory immune-mediated PTR
• Resistant to at least 3 standard therapies
• Able to understand the study and consent
• Projected survival time exceeding three months
• Left Ventricular Ejection Fraction (LVEF) ≥0.5 (as measured by echocardiogram)
• Creatinine \<1.6 mg/dL
• Aspartate Aminotransferase (AST) \< three times the upper limit of normal
• Total bilirubin \<2.0 mg/dL
• Karnofsky Performance Status (KPS) score ≥60.
• Written informed consent obtained;
• Male or female patients aged 18 years or older on the day of informed consent signing;
• Left Ventricular Ejection Fraction (LVEF) ≥50% with no pericardial effusion;
• As assessed by the investigator, systemic treatment drugs (excluding supportive and symptomatic treatment, and prednisone at a daily dose of ≤10 mg or equivalent) can be discontinued prior to lymphodepletion preconditioning.
• Historically diagnosed with primary immune thrombocytopenia (ITP) (based on the 2019 International Working Group for ITP and the American Society of Hematology (ASH));

You may not qualify if:

• Uncontrolled active infection
• Active hepatitis B or C infection
• Patient has HIV or syphilis infection
• Patient is pregnant or breastfeeding
• Patient has a history of allogeneic hematopoietic stem cell transplantation (allo-HSCT)
• Conventional treatment for antibody-mediated disease is effective
• According to the New York Heart Association (NYHA) classification, patients with Class III/IV cardiovascular dysfunction
• Other contraindications that make participation in this study unsuitable.
• Patients with the following conditions at the screening visit: Neutrophil count \<1×10⁹/L; serum creatinine \>1.5× upper limit of normal (ULN); immunoglobulin G (IgG) \<5 g/L.
• Subjects with Class III or IV heart failure according to the NYHA classification (see Appendix I)
• Subjects with a history of epilepsy or other central nervous system diseases
• Patients with active viral, bacterial or other infections requiring systemic treatment at the screening visit (including active or latent tuberculosis (TB) or SARS-CoV-2), or with a history of clinically significant recurrent infections (e.g., Bacillus infection);
• Herpes or varicella-zoster virus infection within 12 weeks prior to the screening visit (particularly shingles);
• Patients positive for HCV or HBsAg are excluded. HBcAb-positive patients are eligible only if HBsAg (regardless of anti-HBs status) and HBV DNA are negative;
• Known history of primary or secondary immunodeficiency, or positive test results for HIV (ELISA and Western blot) at the screening visit;

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead
• Allorunning Therapeutics: collaborator

MeSH Terms

Conditions

Recurrence; Purpura, Thrombocytopenic, Idiopathic

Interventions

Antigens, CD19

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Antigens, CD; Antigens, Differentiation; Antigens, Surface; Antigens; Biological Factors; Antigens, Differentiation, B-Lymphocyte; Minor Histocompatibility Antigens; Histocompatibility Antigens; Isoantigens; Biomarkers

Central Study Contacts

Xiaowen Tang, PhD

CONTACT

+86-13913538266; tangxiaowen@suda.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2025
• First Posted: July 18, 2025
• Study Start: July 10, 2025
• Primary Completion (Estimated): July 30, 2026
• Study Completion (Estimated): July 30, 2027
• Last Updated: July 18, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share