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OpenTrials
Active, Not Recruiting

NCT Number: NCT06665256

Phase 2 Study of Rapcabtagene Autoleucel in 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)

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Idiopathic Inflammatory Myopathies Connective Tissue Diseases Dermatomyositis Lung Diseases Lung Diseases, Interstitial Muscular Diseases Musculoskeletal Diseases Myositis Nervous System Diseases Neuromuscular Diseases Polymyositis Respiratory Tract Diseases Skin Diseases Skin and Connective Tissue Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, Melbourne, Victoria, Australia

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • 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
  • Participants who had inadequate response to prior therapy
  • Diagnosed with active disease such as presence of at least 1 of the following criteria: abnormal enzyme levels assessed as secondary to IIM, or EMG demonstrating active disease, DM skin rash, muscle biopsy demonstrating active IIM, or MRI demonstrating active inflammation.
  • Participant must meet criteria for severe myositis such as presence of active muscle weakness.

Key Exclusion Criteria:

  • 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 ≤17 or ≥40 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.

Treatment and study plan

Rapcabtagene autoleucel

Biological

Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.

Other names: YTB323

Active Comparator Option

Other

Investigator choice of treatment as per protocol

Primary outcomes

  1. Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at Week 52

    Time frame: 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.

Secondary outcomes

  1. Adjusted annual cumulative glucocorticoid dose up to Week 52

    Time frame: Week 52

    The total amount of glucocorticoids administered over the course of a year measured up to the 52nd week of treatment.

  2. Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52

    Time frame: 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.

  3. Proportion of participants achieving major improvement in TIS at Week 52

    Time frame: 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.

  4. Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52

    Time frame: Baseline, Week 52

    The difference in the fatigue levels reported by participants from the start of the study to the 52nd week.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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)

Important dates

Study start
2024
Primary completion
2029
Study completion
2032
First posted
Oct 30, 2024
Registry last updated
Jun 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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