A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)
This is a Phase 2, randomized, active-controlled study. This study comprises two cohorts: * A lead-in cohort enrolling participants to receive rapcabtagene autoleucel * A randomized cohort with participants receiving either rapcabtagene autoleucel or a comparator option. Participants in the comparator arm whose signs and symptoms are not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
123
Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.
Investigator choice of treatment as per protocol
Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at Week 52
The percentage of participants with a TIS of at least 40 at the 52nd week after the start of the study, corresponding to moderate-to-major improvement.
Time frame: Week 52
Adjusted annual cumulative glucocorticoid dose up to Week 52
The total amount of glucocorticoids administered over the course of a year measured up to the 52nd week of treatment.
Time frame: Week 52
Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52
The difference in the percentage of the predicted Forced Vital Capacity (FVC) from the start of the study to the 52nd week.
Time frame: Baseline, Week 52
Proportion of participants achieving major improvement in TIS at Week 52
The percentage of participants with a TIS of at least 60 at the 52nd week after the start of the study, corresponding to at least major improvement.
Time frame: Week 52
Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52
The difference in the fatigue levels reported by participants from the start of the study to the 52nd week.
Time frame: Baseline, Week 52
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University Of Alabama
Birmingham, Alabama, United States
RECRUITINGFL Medical Clinic Orlando Health
Zephyrhills, Florida, United States
RECRUITINGNorthwestern University
Chicago, Illinois, United States
RECRUITINGNorthwestern University
Chicago, Illinois, United States
RECRUITINGUniversity Of Iowa
Iowa City, Iowa, United States
RECRUITINGLDS Hospital
Salt Lake City, Utah, United States
RECRUITINGNovartis Investigative Site
Brest, France
RECRUITINGNovartis Investigative Site
Lille, France
SUSPENDEDNovartis Investigative Site
Lyon, France
RECRUITINGNovartis Investigative Site
Paris, France
RECRUITING...and 45 more locations