Clinical Research Alert: Phase 2 Study of Rapcabtagene Autoleucel in Individuals with IIM

Researchers at Novartis are working to better understand idiopathic inflammatory myopathies (IIMs) and to evaluate a potential new treatment for individuals living with IIM who have not responded to previous treatments (a condition known as refractory disease).

The study

Individuals with refractory IIM may be eligible to participate in a phase 2 clinical trial (AUTOGRAPH-IIM) evaluating the safety and effectiveness of the investigational drug rapcabtagene autoleucel. This drug is a cell-based therapy (a CAR T-cell therapy) designed to target and eliminate B-cells, which contribute to autoimmune activity in IIMs. The study aims to determine if rapcabtagene autoleucel is effective and safe compared to currently available treatment options in people with IIM who have not responded to previous treatment.

This is a randomized, open-label, controlled study, meaning that participants will know whether they have been randomly assigned to the group receiving rapcabtagene autoleucel or to the comparison group receiving standard-of-care treatments. Participants receiving the study drug will begin with a 2-week hospital stay, followed by frequent visits that gradually space out from weekly to monthly, then to every 7–12 weeks, and eventually every 6 months through year five. Those in the standard-of-care arm will follow the same long-term schedule but without the initial hospitalization and with fewer early visits.

Rapcabtagene autoleucel is administered by a single intravenous (in the vein) infusion. The effects of the drug will be evaluated using a number of assessments including but not limited to: physical examination, body weight, body temperature, blood pressure, pulse rate, respiratory rate, pulse oximetry, blood sample, urine sample, questionnaires, electrocardiogram (ECG), and pregnancy test.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:

• Men and women, aged >18 and ≤75 years, with a diagnosis of probable or definite myositis
• Inadequate response to prior therapy
• Diagnosed with active disease
• Meet criteria for severe myositis

Individuals may not be eligible to participate if they are affected by another condition or have received another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

To learn more about the study or inquire about participation, please contact the study coordinator at the closest study site listed here or visit the sponsor’s trial website.