CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus

NCT06839976 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 24-022668
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-center, single-arm, open-label phase 1/2 study of CART19 in children and young adults with refractory Systemic lupus erythematosus (SLE), including both patients diagnosed with lupus nephritis (LN) and patients with non-renal Systemic lupus erythematosus (SLE). Phase 1 will evaluate the safety of CART19 in 6-12 patients with Systemic lupus erythematosus (SLE). There is no planned dose escalation, but a dose de-escalation will be made based on the incidence of Dose Limiting Toxicities. Phase 2 will evaluate the efficacy and further evaluate the safety of CART19 in this population.

Conditions

SLE; Systemic Lupus Erythematosus (SLE); CAR T Cell; CART19; Cell Therapy; Lupus; Lupus Nephritis (LN)

Outcome Measures

Primary Outcomes (1)

• Frequency of the dose limiting toxicities of CART19

  Frequency of the dose limiting toxicities of CART19

  up to 24 months post infusion

Secondary Outcomes (9)

• Rate of childhood SLE Clinical Remission off steroids (cCR-0) at 3 months

  3 months post treatment

• 2-year overall survival rate

  24 months post infusion

• 2-year flare free survival rate

  up to 24 months post infusion

• Feasibility of manufacturing CART19 for participants with SLE

  up to 24 months post infusion

• Proportion of patients achieving a complete renal response

  up to 24 months post infusion

Study Arms (1)

CART19

EXPERIMENTAL

Participants will receive the study product. CART19 cells transduced with a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ, administered by IV injection.

Biological: CART19

Interventions

CART19 BIOLOGICAL

CART19 cells transduced with a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ, administered by IV injection.

CART19

Eligibility Criteria

• Age: 12 Years - 29 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Signed informed consent form must be obtained prior to any study procedure. Labs or other procedures obtained during routine clinical care may be used for eligibility if obtained within the protocol required window.
• Patient age must be 12-29 years, inclusive, at time of enrollment.
• Meeting ACR/EULAR Classification Criteria for SLE
• ANA positive \> 1:80 and/or double-stranded DNA (dsDNA) positive
• Active (refractory) disease, defined as follows:
• a. Lupus nephritis subjects must meet both the following criteria: i. ISN/RPS active nephritis Class III/IV +/- V lupus nephritis diagnosed by biopsy within past 12 months.
• ii. Persistent and clinically significant: ≥2 measurements with urine protein with either of the following:
• \> 1mg/mg creatinine
• \> 0.5 mg/mg creatinine associated with renal dysfunction or low albumin.
• \> 0.5 mg/mg creatinine in a patient with rising proteinuria after prior complete renal response b. Non-renal SLE subjects must meet either of the following criteria: i. SLEDAI-2K ≥ 8 and clinical SLEDAI-2K ≥ 6 ii. Inability to decrease prednisone ≤7.5mg/day or 0.15mg/kg/day, whichever is lower, due to active disease.
• \. Patients must have had at least 3 months of cumulative conventional therapy defined as:
• Conventional induction immunosuppressive agent(s) (e.g., mycophenolate mofetil, cyclophosphamide), and
• At least one additional therapy:
• i. B-cell directed biologic therapy (e.g., rituximab, belimumab, ofatumumab, obinutuzumab) ii. Calcineurin inhibitor (e.g., tacrolimus, cyclosporine, voclosporin) iii. Other immunosuppressive medication for SLE (e.g., anifrolumab, abatacept, JAK inhibitor) 7. Adequate organ function status
• Renal: eGFR must be ≥30 and subject cannot be receiving dialysis.

You may not qualify if:

• Active, untreated infections
• HIV infection
• Active Hepatitis B
• a. Patients must have a negative hepatitis B surface antigen to be enrolled on this study.
• Active Hepatitis C
• Patients with severe neuropsychiatric lupus or neurologic manifestations of SLE (e.g. stroke, seizure, psychosis, demyelinating syndromes, organic brain syndrome, or lupus related headaches)
• Monogenic lupus (known)
• Previous autologous or allogenic stem cell transplant
• Previous kidney transplant
• History of seizure disorder
• Patients who are on anti-epileptic therapy
• Participation in a clinical trial in which the patient receives an investigational drug within a time period equal or less than 5.5 half-lives of the investigational agent prior to study enrollment.
• Subjects who are unwilling or unable to discontinue immunosuppressive medications at the times of CART19 infusion will be excluded from the trial
• Any comorbidity that in the opinion of the investigators would jeopardize the ability of the subject to tolerate therapy.
• Pregnant patients. All participants of childbearing potential must have negative pregnancy test.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Lupus Nephritis

Interventions

CTL019 chimeric antigen receptor

Condition Hierarchy (Ancestors)

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

Study Officials

• Caitlin Elgarten, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Caitlin Elgarten, MD

CONTACT

267-425-7964; elgartenc@chop.edu

Melissa Varghese

CONTACT

845-553-5358; verghesem@chop.edu

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: February 12, 2025
• First Posted: February 21, 2025
• Study Start: May 6, 2025
• Primary Completion (Estimated): February 28, 2030
• Study Completion (Estimated): February 28, 2030
• Last Updated: February 23, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)