Study Description
The purpose of this study is to evaluate the efficacy and safety of rapcabtagene
autoleucel (administered once following lymphodepletion) versus Standard of Care (SOC) in
patients with systemic lupus erythematosus (SLE) with active, refractory lupus nephritis
(LN). This is a Phase 2, adaptive, two-year, randomized, assessor-blinded, active controlled
study:
- Part A: Participants suffering from systemic lupus erythematosus (SLE) with active,
refractory LN will be randomized to Regimen 1, Regimen 2, or SOC.
- Part B: Participants suffering from SLE with active, refractory LN will be
randomized to the selected regimen from Part A or SOC.
The study will consist of two periods:
- A screening period lasting up to 6 weeks, and
- A randomized treatment period and primary follow-up period lasting up to 104 weeks.
After end of study (EOS), participants who received rapcabtagene autoleucel infusion will
enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene
autoleucel infusion. This LTFU will be described in a separate study protocol.
Interventions
rapcabtagene autoleucel Regimen 1
rapcabtagene autoleucel Regimen 2
Standard of Care
Eligibility Criteria
Key Inclusion Criteria:
- Men and women with SLE, aged >= 18 years and =< 65 years at screening, fulfilling
the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology
(ACR) classification criteria for SLE at screening.
- Participant must be positive for at least one of the following autoantibodies at
screening: antinuclear antibodies (ANA) at a titer of >= 1:80 (on HEp-2 cells or an
equivalent positive test), or anti-dsDNA (above the ULN); or anti-Sm (above the ULN)
as determined by a central laboratory.
- Active lupus nephritis without signs of significant chronicity
- SLEDAI-2K Criteria at screening: SLEDAI-2K score >= 6 points (Gladman et al 2002,
Touma et al 2011), excluding points attributed to "fever", "lupus headache",
"alopecia", and "organic brain syndrome".
- Inadequate response at screening to at least two LN treatment regimens
Key Exclusion Criteria:
- Any acute, severe lupus related-flare at screening that needs immediate treatment
other than pulse GCs and/or makes the immunosuppressive washout impossible and,
thus, makes the participant ineligible for CD19 CAR-T therapy
- Inadequate organ function during screening and prior to randomization
- History or current diagnosis of ECG or cardiac abnormalities indicating significant
risk of safety for participants prior to randomization
- Human immunodeficiency virus (HIV) positivity at screening.
- Acute or chronic infection with hepatitis B (HBV) or hepatitis C (HCV) at screening.
- Evidence of active or latent tuberculosis.
- Grade 2 or higher thromboembolic event in the past 4 weeks prior to screening.
- Vaccination (including with live attenuated vaccines) not completed at least 6 weeks
prior to randomization.
Other protocol-defined inclusion/exclusion criteria may apply.
Study Location
Novartis Investigative Site
Recruiting
Singapore,119074,Singapore
Northwestern University Research
Recruiting
Chicago,Illinois,60611,United States
George Georges
Komel Safdar
University Of Iowa
Recruiting
Iowa City,Iowa,52242,United States
Hanna Zembrzuska
Worldwide Contacts
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