Study of CD19 CAR-T Therapy for Refractory SLE

NCT07403097 | Recruiting Phase 1 DMC

• 1 other identifier: PBC098
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

This is an investigator-initiated trial aimed at assessing the safety and efficacy of PTOC1 cells Injection in the treatment of refractory systemic lupus erythematosus.

Conditions

Systemic Lupus Erythematosus

Keywords

CD19; CAR-T

Outcome Measures

Primary Outcomes (7)

• The safety of PTOC1 cell therapy in patients with refractory SLE [Safety]

  Types, frequency and severity of adverse events.

  3 months

• The changes in SLEDAI-2K from baseline [efficacy]

  Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) ranges from 0 to 105 points. The higher the score, the higher the disease activity.

  6 months

• The changes in PGA from baseline [efficacy]

  Physician Global Assessment(PGA) is a continuous visual analogue scale with 0, 1, 2, and 3 scores. "0" indicates no disease activity and "3" indicates the most severe disease activity.

  6 months

• The changes in BILAG-2004 from baseline [efficacy]

  British Isles Lupus Assessment Group Index 2004(BILAG-2004) consists of 8 systems, each of which is graded as A, B, C and D. "A" indicates that the condition is highly active and requires active treatment. "B" indicates that the condition is active and requires close monitoring or symptomatic treatment. "C" indicates a stable condition. "D" indicates that the system is uninvolved.

  6 months

• The number of patients with SRI-4 response [efficacy]

  The definition of SRI-4 response: SLEDAI-2K ≥ 4 points improvement; PGA \<0.3 points increase; BILAG 2004 with no new A grade score and no more than 1 new B grade score.

  3 months

• The number of patients with LLDAS [efficacy]

  The definition of LLDAS: SLEDAI-2K ≤ 4 points and no disease activity in major organs (kidney, central nervous system, heart and lung), and no vasculitis or fever; no new disease activity symptoms compared with previous disease assessments; PGA ≤ 1; serological parameters not required; with the permitted use of low-dose glucocorticoids (prednisolone ≤ 7.5 mg/day), and/or stable immunosuppressives and biologics.

  6 months

• The number of patients with DORIS [efficacy]

  The definition of DORIS: SLEDAI-2K = 0 points; PGA \< 0.5 points; serological parameters not required; with the permitted use of antimalarials, low-dose glucocorticoids (prednisolone ≤ 5 mg/day), and/or stable immunosuppressives and biologics.

  6 months

Secondary Outcomes (9)

• Cmax of PTOC1 cells [PK parameter]

  3 months

• Tmax of PTOC1 cells [PK parameter]

  3 months

• AUC28d/90d of PTOC1 cells [PK parameter]

  3 months

• The degree of B cell depletion [PD parameter]

  3 months

• The concentration of IL-6 [PD parameter]

  3 months

Study Arms (1)

PTOC1

EXPERIMENTAL

This trial was designed as an open, single-arm, single-center, dose-exploration trial.

Biological: PTOC1

Interventions

PTOC1 BIOLOGICAL

Three dose groups (1.5×10\^5/kg, 5×10\^5/kg,1×10\^6/kg) were set up, starting from the low dose group climbing to explore the safe and effective dose.

PTOC1

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 5 years, no gender limitation;
• Diagnosed with SLE according to the 2019 EULAR/ACR classification criteria, and still in moderate to severe disease activity despite ≥ 3 months of high dose glucocorticoids(prednisone≥1mg/kg/d or other equivalent amount of other steroid), combined with hydroxychloroquine, and at least 2 Immunosuppressants or biologics (including cyclophosphamide, mycophenolate mofetil, azathioprine, methotrexate, cyclosporin, tacrolimus, sirolimus, leflunomide, telitacicept, belimumab, and rituximab) or intolerant to standard treatments;
• SLEDAI-2K score ≥ 8 points;
• The functions of vital organs must meet the following requirements:
• cardiac function: left ventricular ejection fraction (LVEF) ≥ 50%, with no obvious abnormalities on electrocardiogram (ECG);
• renal function: eGFR ≥ 30 mL/min/1.73m2;
• hepatic function: AST and ALT ≤ 3.0×ULN, total bilirubin ≤ 2.0×ULN;
• pulmonary function: no severe pulmonary lesions; blood oxygen saturation ≥ 92% under non-oxygen supplementation conditions.
• Meet the criteria of leukapheresis or intravenous blood collection, and no contraindication for leukapheresis;
• Negative pregnancy test for female subjects of childbearing age, and agree to take effective contraceptive measures until one year after infusion;
• Participant or his/her guardians agree to participate in the clinical trial and sign the informed consent form indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study.

You may not qualify if:

• Central nervous system (CNS) diseases: presence of CNS lupus symptoms requiring intervention within 60 days, including epilepsy, confusion, cerebrovascular events, etc;
• Congenital heart disease or severe arrhythmia before screening: Including multifocal and frequent supraventricular tachycardia, ventricular tachycardia, etc.; or complicated with moderate to large pericardial effusion, severe myocarditis, etc.; or patients with unstable vital signs who require vasopressors to maintain blood pressure;
• Presence of active infections requiring systemic treatment or uncontrolled infections within 3 months prior to screening;
• Having received solid organ transplantation or hematopoietic stem cell transplantation within 3 months prior to screening; or having grade 2 or above acute graft-versus-host disease (GVHD) within 2 weeks prior to screening;
• Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood hepatitis B virus (HBV) DNA level exceeding the normal range; positive for hepatitis C virus (HCV) antibody with peripheral blood hepatitis C virus (HCV) RNA level exceeding the normal range; positive for human immunodeficiency virus (HIV) antibody; positive for treponema pallidum antibody;
• History of macrophage activation syndrome within 1 month prior to screening (except for those for whom the investigator has determined that safety risks are excluded after treatment);
• History of previous CAR-T therapy (except for those for whom the investigator has determined that safety risks are excluded after treatment);
• Presence of active pulmonary tuberculosis at the time of screening;
• Having received any vaccination within 4 weeks prior to screening;
• Positive result of blood pregnancy test;
• A confirmed diagnosis of malignant diseases such as tumors prior to screening;
• Participation in other clinical trials within 3 months prior to enrollment;
• Other circumstances that the investigator deems inappropriate for participation in this study.

Sponsors & Collaborators

• Chongqing Precision Biotech Co., Ltd: lead

Study Sites (1)

Children's Hospital of Chongqing Medical University

Chongqing, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Xiaodong Zhao

CONTACT

+8613896187845; zhaoxd530@aliyun.com

Yunfei An

CONTACT

+8618623070626; anyf82@aliyun.com

Study Design

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

Study Record Dates

• First Submitted: February 4, 2026
• First Posted: February 11, 2026
• Study Start: February 1, 2026
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: February 11, 2026

Record last verified: 2026-02

Locations

CN (1)