Evaluating a new CAR-T therapy for myositis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2 clinical trial aims to assess the efficacy and safety of rapcabtagene autoleucel, a chimeric antigen receptor T cell therapy, in patients with severe refractory idiopathic inflammatory myopathies (IIM). The study includes a lead-in cohort receiving the therapy and a randomized cohort comparing it to an active control. Participants will be monitored for two years, followed by a long-term follow-up period lasting up to 15 years. The trial is designed to provide insights into the potential benefits of CAR-T therapy for this challenging condition.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

1. Men and women, aged ≥ 18 and ≤75 years, with a diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria
2. Participants who had inadequate response to prior therapy
3. Diagnosed with active disease
4. Participant must meet criteria for severe myositis

Key Exclusion Criteria:

1. 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
2. BMI at Screening of ≤17 or ≥40 kg/m2
3. Severe muscle damage at Screening
4. Inadequate organ function
5. Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol
6. Other inflammatory and non-inflammatory myopathies
7. 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.

Where it's running

• University Of Alabama
  Birmingham, Alabama, United States Recruiting
• FL Medical Clinic Orlando Health
  Zephyrhills, Florida, United States Recruiting
• Northwestern University
  Chicago, Illinois, United States Recruiting
• Northwestern University
  Chicago, Illinois, United States Recruiting
• University Of Iowa
  Iowa City, Iowa, United States Recruiting
• LDS Hospital
  Salt Lake City, Utah, United States Recruiting

+5 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Investigating AZD5492 for treating systemic lupus erythematosus and inflammatory myopathies

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.