Anti-CD19 Chimeric Antigen Receptor T Cells for Refractory Autoimmune Diseases

NCT06688799 | Recruiting Phase 1 DMC

• 1 other identifier: BJGBYY-IIT-LCYJ-2024-046
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this study is to evaluate the safety and effi cacy of CD19 CAR T cells in the treatment of Refractory Autoimmune Diseases.

Conditions

Autoimmune Diseases

Keywords

Refractory Autoimmune Diseases; CD19 CAR-T

Outcome Measures

Primary Outcomes (3)

• Phase I:Dose-limiting toxicity (DLT)

  The incidence and type of dose-limiting toxicity (DLT).

  28 days post infusion

• Phase I： Adverse events (AEs)

  The incidence and severity of adverse events (AE).

  30 days post infusion

• Phase II: Objective response rate (ORR)

  Proportions of subjects achieving Autoimmune Diseases response

  3 months and 6 months post infusion]

Secondary Outcomes (9)

• Phase I: Objective response rate (ORR)

  3 months and 6 months

• Phase I: Long-term serious adverse events (SAEs)

  From 30 days after CD19 CAR T infusion to 2 years

• Phase I: Pharmacokinetics(PK)

  Up to 2 years post infusion

• Phase I: Pharmacodynamic(PD)

  Up to 2 years post infusion

• Phase II: Adverse events (AEs)

  2 years post infusion

Study Arms (1)

Experimental : CD19 CAR

EXPERIMENTAL

Following the lymphodepleting treatment, patients will be treated with CD19 Chimeric Antigen Receptor (CAR) positive T cells as a single dose.

Drug: CD19 CAR-T cells

Interventions

CD19 CAR-T cells DRUG

Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with CD19 Chimeric Antigen Receptor (CAR) positive T cells as a single dose.

Experimental : CD19 CAR

Eligibility Criteria

• Age: 3 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Systemic lupus erythematosus (SLE) 1.1. Refractory systemic lupus erythematosus (SLE) and / or refractory lupus nephritis (LN)
• Male or female patients, aged 3-65 years (including 3 and 65 years);
• Systemic lupus erythematosus that meets the 2019 American Society of Rheumatology (ACR) / European Association of Rheumatology Consortium (EULAR) classification criteria (see Annex 2);
• Nuclear antibody (ANA) test is clearly positive, namely ANA titer 1:80 (based on the equivalent results of Hep-2 immunofluorescence or enzyme immunoassay) and / or the test at the screening visit (based on ELISA test, 30 IU / mL).
• Refractory systemic lupus erythematosus (SLE) and / or refractory lupus nephritis (LN):
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• Patients take at least 7.5 mg of prednisolone daily to maintain low disease activity or a SLEDAI 2K score (see attachment 3) of 8 or higher.
• Recurrence of disease activity after failure of conventional therapy or after remission. Definition of conventional treatment: using corticosteroids (1 mg / kg / day) and cyclophosphamide for 6 months; or any of the following immunomodulatory drugs for more than 3 months: antimalarial drugs, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, and biological agents such as rituximab, belimumab, and teacept.
• \) Physical strength status score (ECOG): 0-2 points; 6) The estimated survival period of 90 days; 1.2. Immune thrombocytopenia in refractory lupus (SLE-ITP)
• Age range: 18-70 years (including 18 and 70 years), gender limitation.
• Patients with refractory systemic lupus erythematosus (immune thrombocytopenia): meeting SLE 2019 ACR / EULAR classification criteria, with at least 2 consecutive routine blood tests showing platelets lower ; no abnormal morphology of blood cells in peripheral blood smear; morphological characteristics of bone marrow cells meeting immune thrombocytopenia. Refractory systemic lupus erythematosus (immune thrombocytopenia) was defined as at least 1 course of MP shock (1g 3 days) or high-dose hormone (1mg / kg d equivalent dose of glucocorticoid) combined with 1 or more immunosuppressive agents. Thrombocytopenia except for other than non-SLE causes, such as infection, myelosuppression, macropleic, hypersplenism, etc.
• Clinician assessment of the patients condition allowed the use of 10mg prednisone or its equivalent dose during the study and allowed the discontinuation of all immunosuppressive agents (excluding hydroxychloroquine).
• refractory lupus thrombotic microangiopathy (SLE-TMA)
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• Age range: 18-70 years (including 18 and 70 years), gender limitation.

You may not qualify if:

• \. intracranial hypertension or cerebral consciousness disorder: Intracranial pressure is kept above 15mmHg; Organic encephalopathy syndrome, cerebrovascular accident, encephalitis or central nervous system vasculitis, visual impairment and other brain lesions that require intervention.
• \. Symptomatic heart failure or severe arrhythmia: Left ventricular ejection fraction (LVEF) \<45% within 12 months prior to screening; Abnormal electrocardiogram (ECG): left bundle branch, double bundle branch block or other clinically significant abnormal electrocardiogram; Congenital long QT interval (QT) syndrome or Fridericia correction formula (QTcF) 470 ms; Congestive heart failure (New York Heart Association Class III or IV); 3. Severe respiratory failure or other respiratory symptoms that are difficult to control: 4. Along with other types of malignant tumors; 5. Diffuse endovascular coagulation; 6. Sepsis or other infections that are difficult to control: uncontrolled active systemic bacterial, viral, fungal or parasitic infections (except nail fungal infections) or other clinically significant active diseases; 7. Uncontrolled diabetes: fasting blood glucose (FBG)≥8.0mmol/L, 2 hours postprandial blood glucose (PBG) 15 mmol/L, glycated hemoglobin (HbA 1 c) after at least 3 months of diet, exercise or related treatment; combined with diabetic ketoacidosis or other uncontrollable diabetic complications; 8. Received organ transplantation (excluding bone marrow transplantation); 9. eGFR CKD-EPI \< 30 ml/min/1.73m\^2； 10. Patients who cannot continue the immunosuppressive agents for 7 days, or repeat the disease and the investigators evaluate the risk of serious adverse reactions.

Sponsors & Collaborators

• Beijing GoBroad Hospital: lead

Study Sites (1)

Beijing GoBroad Hospital

Beijing, Beijing Municipality, China, China

RECRUITING

MeSH Terms

Conditions

Autoimmune Diseases

Condition Hierarchy (Ancestors)

Immune System Diseases

Central Study Contacts

Tengyu Wang

CONTACT

18333186020; tengyu.wang@gohealtharo.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Dept of Hemato-Oncology and Immunotherapy

Study Record Dates

• First Submitted: November 12, 2024
• First Posted: November 14, 2024
• Study Start: November 23, 2024
• Primary Completion: March 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: March 10, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)