Safety of PiggyBac Transposon CAR T-cells Targeting CD-19 in Refractory Lupus.

NCT06150651 | Completed Phase 1 DMC

• 1 other identifier: SLE-PB-CD19-CART
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 1 clinical trial to evaluate the safety and efficacy of PiggyBac transposon-mediated Chimeric Antigen Receptor(CAR) T-cells targeting CD19 in refractory Systemic Lupus Erythematosus (SLE) patients who have not responded to standard immunosuppressive treatments.

Conditions

SLE (Systemic Lupus)

Outcome Measures

Primary Outcomes (1)

• Safety of PiggyBac transposon-mediated CAR T-cell infusion targeting CD19 in adult patients with refractory SLE.

  The incidence of adverse events assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0

  Up to 28 days after CD-19 CAR-T cell infusion

Secondary Outcomes (3)

• Disease activity of SLE

  3, 6, and 12 months after CD-19 CAR-T cell infusion

• Complete response rate of lupus nephritis

  3, 6, and 12 months after CD-19 CAR-T cell infusion

• Partial response rate of lupus nephritis

  3, 6, and 12 months after CD-19 CAR-T cell infusion

Study Arms (1)

CAR T-cell therapy

EXPERIMENTAL

Subjects will receive a conditioning lymphodepletion chemotherapy regimen consisting of fludarabine and cyclophosphamide, followed by an infusion of CD19 CAR-T cells at doses of 1 × 10⁶ cells/kg (n = 3) and 2 × 10⁶ cells/kg (n = 3).

Other: PiggyBac Transposon-Mediated CD19 CAR-T Therapy

Interventions

PiggyBac Transposon-Mediated CD19 CAR-T Therapy OTHER

PiggyBac Transposon-Mediated CD19 CAR-T Therapy (1-2 x 10\^6 cells/kg)

CAR T-cell therapy

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age between 18 and 60 years.
• Diagnosis of Systemic Lupus Erythematosus (SLE), as defined by the American College of Rheumatology (ACR) 1997 criteria, The Systemic Lupus International Collaborating Clinics (SLICC) criteria, or the European Alliance of Associations for Rheumatology (EULAR)/ACR classification.
• Refractory SLE, defined by one or more of the following:
• Persistently active SLE requiring ongoing maintenance therapy (if not contraindicated) with:
• Antimalarial drug.
• Either mycophenolate (minimum daily dose of 1500 mg) or azathioprine (minimum daily dose of 1.5 mg/kg).
• Patients must also need a minimum daily dose of 7.5 mg prednisolone for lower disease activity maintenance, or have a SLEDAI score of 8 or higher.
• Biopsy-proven proliferative lupus nephritis after two standard induction therapies, including intravenous cyclophosphamide (cumulative dose of at least 1.5 g) and mycophenolate mofetil (administered for a minimum of 3 months), unless contraindicated.
• Worsening of biopsy-proven lupus nephritis (activity index \> 6 and chronicity index \< 6 within 6 months), indicated by increased proteinuria and/or decreased estimated glomerular filtration rate, despite treatment with high-dose corticosteroids (prednisolone at least 0.7 mg/kg/day or equivalent) and either mycophenolate mofetil or cyclophosphamide for a minimum of 14 days.
• Ability to understand and willingness to sign a written informed consent document.
• Participants of child-bearing or child-fathering potential must agree to practice birth control from enrollment until four months after receiving CAR T-cell infusion.

You may not qualify if:

• Pregnant or breastfeeding women.
• History of active malignancy, excluding non-melanoma skin cancer and carcinoma in situ (e.g., cervix, bladder, breast).
• History of vital organ transplantation (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/bone marrow transplantation.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, pulmonary abnormalities, cirrhosis, or psychiatric illness/social situations that limit compliance with study requirements.
• Any other clinically significant disease history or current disease that, in the judgment of the research physician, may pose a risk to the safety of the subjects or interfere with the research procedure, or the evaluation of safety and efficacy.
• Serologic status indicating active HIV, hepatitis B, or C infection. Participants positive for hepatitis B core antibody, hepatitis B surface antigen, or hepatitis C antibody must have a negative PCR prior to enrollment.
• History of severe adverse drug reaction to Cyclophosphamide or Fludarabine.
• Received a live vaccine within 30 days prior to CAR-T cell infusion.
• eGFR CKD-EPI \< 30 ml/min/1.73m\^2.
• Participation in other clinical investigations during the study period.
• Prior receipt of CAR-T cell therapy outside this protocol.

Sponsors & Collaborators

• Chulalongkorn University: lead
• King Chulalongkorn Memorial Hospital: collaborator
• Health Systems Research Institute,Thailand: collaborator

Study Sites (1)

King Chulalongkorn Memorial Hospital

Bangkok, Please Select, 10330, Thailand

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

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

Study Officials

• Wonngarm Kittanamongkolchai, MD

  Chulalongkorn University

  PRINCIPAL INVESTIGATOR

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: Principle Investigator

Model Details: Intervention: PiggyBac transposon-mediated CD-19 CAR T-cells (1x10\^6 cells/kg)

Study Record Dates

• First Submitted: November 13, 2023
• First Posted: November 29, 2023
• Study Start: December 1, 2023
• Primary Completion: March 1, 2026
• Study Completion: March 1, 2026
• Last Updated: March 23, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

TH (1)