Home › Trials › NCT06665256

Evaluating a new CAR-T therapy for myositis

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)

Phase 2 Interventional Novartis · NCT06665256

This study is testing a new CAR-T therapy to see if it can help people with severe myositis who haven't responded to other treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment123 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorNovartis Industry-sponsored
Drugs / interventionsCAR-T
Locations55 sites (Birmingham, Alabama and 54 other locations)
Trial IDNCT06665256 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 65 with a diagnosis of severe myositis who have not adequately responded to previous treatments.

Not a fit: Patients with other inflammatory myopathies or those with severe muscle damage or inadequate organ function may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could offer a new treatment option for patients with severe myositis who have not responded to existing therapies.

How similar studies have performed: While CAR-T therapies have shown promise in other conditions, this specific application in myositis is relatively novel and untested.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 this trial is running

Birmingham, Alabama and 54 other locations

+5 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Idiopathic Inflammatory MyopathiesChimeric Antigen Receptor T cellsrapcabtagene autoleucelidiopathic inflammatory myopathiesdermatomyositisAnti-Synthetase SyndromeImmune-Mediated Necrotizing MyopathyInterstitial Lung Disease
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.