Phase 2 Study of Rapcabtagene Autoleucel in Myositis

May 21, 2026 updated by: Novartis Pharmaceuticals

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)

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

123

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Australia
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Recruiting
        • Novartis Investigative Site
  • Brazil
    • São Paulo
      • Barretos, São Paulo, Brazil, 14784 400
        • Recruiting
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 01308-050
        • Recruiting
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 01232-010
        • Recruiting
        • Novartis Investigative Site
  • France
      • Brest, France, 29200
        • Recruiting
        • Novartis Investigative Site
      • Lille, France, 59037
        • Suspended
        • Novartis Investigative Site
      • Lyon, France, 69003
        • Recruiting
        • Novartis Investigative Site
      • Marseille, France, 13885
        • Recruiting
        • Novartis Investigative Site
      • Paris, France, 75013
        • Recruiting
        • Novartis Investigative Site
      • Rennes, France, 35033
        • Recruiting
        • Novartis Investigative Site
  • Germany
      • Aachen, Germany, 52074
        • Recruiting
        • Novartis Investigative Site
      • Hamburg, Germany, 20246
        • Recruiting
        • Novartis Investigative Site
      • Mainz, Germany, 55131
        • Recruiting
        • Novartis Investigative Site
      • Nuremberg, Germany, 90419
        • Recruiting
        • Novartis Investigative Site
      • Ulm, Germany, 89081
        • Recruiting
        • Novartis Investigative Site
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Germany, 50937
        • Recruiting
        • Novartis Investigative Site
    • Saxony
      • Leipzig, Saxony, Germany, 04103
        • Recruiting
        • Novartis Investigative Site
    • Thuringia
      • Jena, Thuringia, Germany, 07740
        • Recruiting
        • Novartis Investigative Site
  • Israel
      • Haifa, Israel, 3109601
        • Recruiting
        • Novartis Investigative Site
      • Ramat Gan, Israel, 5265601
        • Recruiting
        • Novartis Investigative Site
      • Tel Aviv, Israel, 6423906
        • Recruiting
        • Novartis Investigative Site
  • Italy
    • AN
      • Ancona, AN, Italy, 60126
        • Recruiting
        • Novartis Investigative Site
    • BS
      • Brescia, BS, Italy, 25123
        • Recruiting
        • Novartis Investigative Site
    • MB
      • Monza, MB, Italy, 20900
        • Recruiting
        • Novartis Investigative Site
    • MI
      • Milan, MI, Italy, 20122
        • Recruiting
        • Novartis Investigative Site
      • Rozzano, MI, Italy, 20089
        • Recruiting
        • Novartis Investigative Site
    • VR
      • Verona, VR, Italy, 37134
        • Recruiting
        • Novartis Investigative Site
  • Japan
      • Fukuoka, Japan, 8128582
        • Recruiting
        • Novartis Investigative Site
      • Ishikawa, Japan, 9208641
        • Recruiting
        • Novartis Investigative Site
      • Kyoto, Japan, 6068507
        • Recruiting
        • Novartis Investigative Site
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 060-8648
        • Recruiting
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama, Kanagawa, Japan, 236-0004
        • Recruiting
        • Novartis Investigative Site
    • Miyagi
      • Sendai, Miyagi, Japan, 9808574
        • Recruiting
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 565-0871
        • Recruiting
        • Novartis Investigative Site
    • Shimane
      • Izumo, Shimane, Japan, 6938501
        • Recruiting
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 113-8603
        • Recruiting
        • Novartis Investigative Site
      • Bunkyo-ku, Tokyo, Japan, 1138519
        • Recruiting
        • Novartis Investigative Site
      • Shinjuku-ku, Tokyo, Japan, 1608582
        • Recruiting
        • Novartis Investigative Site
  • Netherlands
    • North Holland
      • Amsterdam, North Holland, Netherlands, 1105 AZ
        • Recruiting
        • Novartis Investigative Site
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 11211
        • Recruiting
        • Novartis Investigative Site
  • Singapore
      • Singapore, Singapore, 169608
        • Recruiting
        • Novartis Investigative Site
      • Singapore, Singapore, 119074
        • Recruiting
        • Novartis Investigative Site
      • Singapore, Singapore, S308433
        • Recruiting
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain, 08035
        • Recruiting
        • Novartis Investigative Site
      • Córdoba, Spain, 14004
        • Recruiting
        • Novartis Investigative Site
      • Madrid, Spain, 28041
        • Recruiting
        • Novartis Investigative Site
      • Madrid, Spain, 28009
        • Recruiting
        • Novartis Investigative Site
      • Málaga, Spain, 29010
        • Recruiting
        • Novartis Investigative Site
      • Salamanca, Spain, 37007
        • Recruiting
        • Novartis Investigative Site
    • A Coruna
      • Santiago Compostela, A Coruna, Spain, 15706
        • Recruiting
        • Novartis Investigative Site
    • Cantabria
      • Santander, Cantabria, Spain, 39008
        • Recruiting
        • Novartis Investigative Site
    • Navarre
      • Pamplona, Navarre, Spain, 31008
        • Recruiting
        • Novartis Investigative Site
  • Switzerland
      • Geneva, Switzerland, 1211
        • Recruiting
        • Novartis Investigative Site
  • Taiwan
      • Kaohsiung City, Taiwan, 83301
        • Recruiting
        • Novartis Investigative Site
      • Taichung, Taiwan, 407219
        • Recruiting
        • Novartis Investigative Site
      • Taipei, Taiwan, 10002
        • Recruiting
        • Novartis Investigative Site
  • United Kingdom
      • London, United Kingdom, NW1 2BU
        • Recruiting
        • Novartis Investigative Site
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S10 2JF
        • Recruiting
        • Novartis Investigative Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Recruiting
        • University Of Alabama
        • Contact:
        • Principal Investigator:
          • James Andrews
    • Florida
      • Zephyrhills, Florida, United States, 33542
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Northwestern University
        • Principal Investigator:
          • George Georges
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa
        • Principal Investigator:
          • Hanna Zembrzuska
        • Contact:
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15261
        • Recruiting
        • University Of Pittsburgh Medical Center
        • Contact:
        • Principal Investigator:
          • Jeremy Tilstra
    • Texas
      • Dallas, Texas, United States, 75246
      • Houston, Texas, United States, 77030
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • Recruiting
        • LDS Hospital
        • Principal Investigator:
          • Dany Saad
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 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.
  4. Participant must meet criteria for severe myositis such as presence of active muscle weakness.

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Rapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel (YTB323)
Biological: Rapcabtagene autoleucel
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: Comparator
Investigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab)
Other: Active Comparator Option
Investigator choice of treatment as per protocol

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Week 52

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Week 52
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.
Baseline, Week 52
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.
Week 52
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.
Baseline, Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 17, 2024

Primary Completion (Estimated)

March 9, 2029

Study Completion (Estimated)

November 29, 2032

Study Registration Dates

First Submitted

October 29, 2024

First Submitted That Met QC Criteria

October 29, 2024

First Posted (Actual)

October 30, 2024

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 21, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CYTB323L12201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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