Umbilical Cord Blood CD19-BCMA CART Cell Therapy for SLE-LN, SSc, andpSS PAH.

NCT06947473 | Recruiting Phase 1 DMC

• 1 other identifier: BJGBYY-IIT-LCYJ-2025-025
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-center, open-label, non-randomized, single-arm clinical trial. Patients with refractory lupus neritis (SLE-LN), systemic sclerosis (SSc), and primary Sjogren syndrome combined with pulmonary artery hypertension (pSS-PAH receive umbilical cord blood CD19-BCMA CAR T cell therapy. The primary objective is to prospectively assess the safety of umbilical cord blood CD19BCMA CAR T cell therapy in patients with refractory lupus nephritis (SLE-LN), systemic sclerosis (SSc), and primaryjogren syndrome combined with pulmonary artery hypertension. The primary endpoint is the type and incidence of dose-limiting toxicity (DLT) within 28 days after theusion of umbilical cord blood CD19-BCMA CAR T cells. It is anticipated that 45-54 participants will be recruited.

Conditions

Refractory Lupus Nephritis; Systemic Sclerosis; Primary Sjogren's Syndrome Combined With Pulmonary Hypertension

Outcome Measures

Primary Outcomes (2)

• Dose-limiting toxicity (DLT)

  Incidence and type of dose-limiting toxicity(DLT) within 28 days of umbilical cord blood CD19-BCMA CAR-T infusion.

  28 days

• Adverse events (AEs）

  Total number, incidence and severity of adverse events (AEs) within 30 days of umbilical cord blood CD19-BCMA CAR-T infusion

  30 days

Secondary Outcomes (2)

• Overall remission rate (ORR)

  90,120 days

• Serious adverse event（SAE)

  from 30 days to 2 years

Study Arms (1)

umbilical cord blood CD19-BCMA CAR T cell therapy.

EXPERIMENTAL

Patients were treated with umbilical cord blood CD19-BCMA CAR T cell therapy.

Drug: umbilical cord blood CD19-BCMA CAR-T cells infusion

Interventions

umbilical cord blood CD19-BCMA CAR-T cells infusion DRUG

Approximately 3-5 days prior to umbilical cord blood CD19-BCMA CAR-T cell infusion, subjects are treated with FC regimen (fludarabine and cyclophosphamide) for lymphodepletion. CAR-T cell infusion are performed 48 h after completion of chemotherapy.

umbilical cord blood CD19-BCMA CAR T cell therapy.

Eligibility Criteria

• Age: 6 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Refractory lupus nephritis (LN):
• According to the 2019 American College of Rheumatology (ACR) criteria and the 2018 International Society of Nephrology (ISN)/Society of Nephrology (RPS) criteria (see Appendix 3), confirmed by renal tissue biopsy as active, proliferative lupus nephritis, type III or IV \[excluding type III (C), IV-S (C), and IV-G (C)\], or type III/IV combined with type V. Biopsy must be performed within 6 months prior to the screening visit or during the screening period.
• Male or female patients aged 3-65 years old;
• Systemic lupus erythematosus that meets the 2019 American College of Rheumatology (ACR)/European Union of Rheumatology Associations (EULAR) classification criteria (see Annex 5);
• The result of the anti nuclear antibody (ANA) test is clearly positive, that is, the ANA titer is ≥ 1:80 (equivalent test result based on Hep-2 immunofluorescence detection method or enzyme immunoassay method), and/or at the screening visit, the anti dsDNA serum antibody test is positive (≥ 30 IU/mL based on ELISA detection method) according to the test results of the research center laboratory.
• B cell CD19+expression and discontinuation of immunosuppressive agents for more than 1 week;
• No severe allergic constitution;
• Physical condition score (ECOG) 0-2 points;
• Expected survival period ≥ 90 days;
• The subjects and/or their guardians are able to understand and willing to sign a written informed consent form; 2. Systemic sclerosis (SSc):
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• The selected patient or their legal representative voluntarily signs the informed consent form.
• Age range: 6-65 years old (including 6 and 65 years old), gender is not limited.
• According to the SSc classification criteria proposed by the American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) in 2013, the highest score under the same item, ≥ 9 points, can be classified as SSc (Attachment 6).
• Or if one sufficient condition is met, that is, the skin of both fingers thickens and extends to the proximal end of the metacarpophalangeal joint, it can be directly classified as SSc.

You may not qualify if:

• \. Refractory lupus nephritis (LN):
• \) Intracranial hypertension or cerebral consciousness disorders; 2) Symptomatic heart failure or severe arrhythmia; 3) Symptoms of severe respiratory failure; 4) Accompanied by other types of malignant tumors; 5) Diffuse intravascular coagulation; 6) Suffering from sepsis or other difficult to control infections; 7) Have uncontrollable diabetes and other endocrine diseases; 8) Serious mental disorder; 9) Head MRI examination shows obvious lesions in the skull; 10) Received organ transplantation (excluding bone marrow transplantation); 11) Female patients (those with fertility) have a positive HCG test in their blood; 12) Hepatitis (including hepatitis B and C), AIDS and syphilis screening positive persons; 13）eGFR CKD-EPI \< 30 ml/min/1.73m\^2； 14) Any active skin disease that may interfere with the evaluation of systemic lupus erythematosus research,Including but not limited to psoriasis, dermatomyositis, systemic sclerosis, and non systemic diseases Skin manifestations of lupus erythematosus (such as skin vascular disease, pericapillary dilation)Zhang, fingertip sclerosis, rheumatoid nodules, erythema multiforme, leg ulcers)Or drug-induced lupus.
• \) Subjects who were not successfully matched during the screening period; 16) Previously received CART treatment other than CD19-CART. 2. Primary Sjogren's syndrome with pulmonary hypertension (pSS PAH):
• \) Overlapping connective tissue diseases; Rheumatoid arthritis, systemic lupus erythematosus, or inflammatory myopathy 2) The clinical manifestations can be explained by similar diseases of SSc, such as nephrogenic systemic fibrosis, generalized scleroplakia, eosinophilic fasciitis, diabetes scleroma, scleromyedema, erythematous acrodynia, porphyria, sclerotic lichen, graft-versus-host disease, diabetes and other endocrine diseases related hand joint diseases.

Sponsors & Collaborators

• Beijing GoBroad Hospital: lead

Study Sites (1)

Beijing GoBroad Hospital

Beijing, Beijing Municipality, 102206, China

RECRUITING

MeSH Terms

Conditions

Lupus Nephritis; Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Lupus Erythematosus, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases

Central Study Contacts

ZHOU

CONTACT

86+18511772961; zhousn@gobroadhealthcare.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 the department of Immunotherapy for Hematopoietic Malignancies

Study Record Dates

• First Submitted: April 21, 2025
• First Posted: April 27, 2025
• Study Start: March 18, 2026
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: March 3, 2026

Record last verified: 2025-05

Locations

CN (1)