Phase 2 Study of Rapcabtagene Autoleucel in Myositis

June 22, 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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Australia
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Novartis Investigative Site
  • Brazil
    • São Paulo
      • Barretos, São Paulo, Brazil, 14784 400
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 01232-010
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 01308-050
        • Novartis Investigative Site
  • France
      • Brest, France, 29200
        • Novartis Investigative Site
      • Lille, France, 59037
        • Novartis Investigative Site
      • Lyon, France, 69003
        • Novartis Investigative Site
      • Marseille, France, 13885
        • Novartis Investigative Site
      • Paris, France, 75013
        • Novartis Investigative Site
      • Rennes, France, 35033
        • Novartis Investigative Site
  • Germany
      • Aachen, Germany, 52074
        • Novartis Investigative Site
      • Hamburg, Germany, 20246
        • Novartis Investigative Site
      • Mainz, Germany, 55131
        • Novartis Investigative Site
      • Nuremberg, Germany, 90419
        • Novartis Investigative Site
      • Ulm, Germany, 89081
        • Novartis Investigative Site
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Germany, 50937
        • Novartis Investigative Site
    • Saxony
      • Leipzig, Saxony, Germany, 04103
        • Novartis Investigative Site
    • Thuringia
      • Jena, Thuringia, Germany, 07740
        • Novartis Investigative Site
  • Israel
      • Haifa, Israel, 3109601
        • Novartis Investigative Site
      • Ramat Gan, Israel, 5265601
        • Novartis Investigative Site
      • Tel Aviv, Israel, 6423906
        • Novartis Investigative Site
  • Italy
    • AN
      • Ancona, AN, Italy, 60126
        • Novartis Investigative Site
    • BS
      • Brescia, BS, Italy, 25123
        • Novartis Investigative Site
    • MB
      • Monza, MB, Italy, 20900
        • Novartis Investigative Site
    • MI
      • Milan, MI, Italy, 20122
        • Novartis Investigative Site
      • Rozzano, MI, Italy, 20089
        • Novartis Investigative Site
    • VR
      • Verona, VR, Italy, 37134
        • Novartis Investigative Site
  • Japan
      • Fukuoka, Japan, 8128582
        • Novartis Investigative Site
      • Ishikawa, Japan, 9208641
        • Novartis Investigative Site
      • Kyoto, Japan, 6068507
        • Novartis Investigative Site
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 060-8648
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama, Kanagawa, Japan, 236-0004
        • Novartis Investigative Site
    • Miyagi
      • Sendai, Miyagi, Japan, 9808574
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 565-0871
        • Novartis Investigative Site
    • Shimane
      • Izumo, Shimane, Japan, 6938501
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 113-8603
        • Novartis Investigative Site
      • Bunkyo-ku, Tokyo, Japan, 1138519
        • Novartis Investigative Site
      • Shinjuku-ku, Tokyo, Japan, 1608582
        • Novartis Investigative Site
  • Netherlands
    • North Holland
      • Amsterdam, North Holland, Netherlands, 1105 AZ
        • Novartis Investigative Site
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 11211
        • Novartis Investigative Site
  • Singapore
      • Singapore, Singapore, 119074
        • Novartis Investigative Site
      • Singapore, Singapore, 169608
        • Novartis Investigative Site
      • Singapore, Singapore, S308433
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain, 08035
        • Novartis Investigative Site
      • Córdoba, Spain, 14004
        • Novartis Investigative Site
      • Madrid, Spain, 28041
        • Novartis Investigative Site
      • Madrid, Spain, 28009
        • Novartis Investigative Site
      • Málaga, Spain, 29010
        • Novartis Investigative Site
      • Salamanca, Spain, 37007
        • Novartis Investigative Site
    • A Coruna
      • Santiago Compostela, A Coruna, Spain, 15706
        • Novartis Investigative Site
    • Cantabria
      • Santander, Cantabria, Spain, 39008
        • Novartis Investigative Site
    • Navarre
      • Pamplona, Navarre, Spain, 31008
        • Novartis Investigative Site
  • Switzerland
      • Geneva, Switzerland, 1211
        • Novartis Investigative Site
  • Taiwan
      • Kaohsiung City, Taiwan, 83301
        • Novartis Investigative Site
      • Taichung, Taiwan, 407219
        • Novartis Investigative Site
      • Taipei, Taiwan, 10002
        • Novartis Investigative Site
  • United Kingdom
      • London, United Kingdom, NW1 2BU
        • Novartis Investigative Site
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S10 2JF
        • Novartis Investigative Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama
    • Florida
      • Zephyrhills, Florida, United States, 33542
        • FL Medical Clinic Orlando Health
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Augusta University Georgia
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke Clinical Research Institute
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15261
        • University of Pittsburgh Medical Center
    • Texas
      • Dallas, Texas, United States, 75246
        • Baylor University Medical Center
      • Houston, Texas, United States, 77030
        • Houston Methodist Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • LDS Hospital

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 17, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 9, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 29, 2032

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 29, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 29, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 30, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 23, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 22, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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