Phase 2 Study of Rapcabtagene Autoleucel in Myositis
- Conditions
- Idiopathic Inflammatory Myopathies
- Interventions
- Other: Active Comparator Option
- Registration Number
- NCT06665256
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
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)
- Detailed Description
This is a Phase 2, two-year, randomized, assessor- blinded, 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.
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 123
- Men and women, aged >18 and ≤65 years, with a diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria
- Participants who had inadequate response to prior therapy
- Diagnosed with active disease
- Participant must meet criteria for severe myositis
Key
- Any condition during Screening that could prevent a complete washout of medications or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in the study
- BMI at Screening of ≤18.5 or ≥35 kg/m2
- Severe muscle damage at Screening
- Inadequate organ function
- Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol
- Other inflammatory and non-inflammatory myopathies
- Any medical conditions that are not related to IIM that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy
Other protocol-defined inclusion/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Rapcabtagene autoleucel Rapcabtagene autoleucel Single infusion of rapcabtagene autoleucel (YTB323) Comparator Active Comparator Option Investigator choice of treatment as per protocol
- Primary Outcome Measures
Name Time Method Proportion of participants achieving at least moderate improvement in Total Improvement Score (TIS) at Week 52 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 at least moderate improvement.
- Secondary Outcome Measures
Name Time Method Adjusted annual cumulative glucocorticoid dose up to Week 52 Week 52 The total amount of glucocorticoids administered over the course of a year measured up to the 52nd week of treatment.
Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52 Baseline, Week 52 The difference in the percentage of the predicted Forced Vital Capacity (FVC) from the start of the study to the 52nd week.
Proportion of participants achieving major improvement in TIS at Week 52 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.
Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52 Baseline, Week 52 The difference in the fatigue levels reported by participants from the start of the study to the 52nd week.
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) Week 104 The distribution of adverse events will be done via the analysis of frequencies for Adverse Event (AEs) and Serious Adverse Event (SAEs) through the monitoring of relevant clinical and laboratory safety parameters.
Trial Locations
- Locations (3)
Northwestern University
🇺🇸Chicago, Illinois, United States
University Of Iowa
🇺🇸Iowa City, Iowa, United States
Novartis Investigative Site
🇨🇳Taichung, Taiwan