Idiopathic Inflammatory MyopathiesPhase 2NCT06665256
Phase 2 Study of Rapcabtagene Autoleucel in Myositis
A Phase 2 study testing Rapcabtagene autoleucel and Active Comparator Option in people with Idiopathic Inflammatory Myopathies, run by Novartis Pharmaceuticals at 68 sites in 15 countries.
Trial summary
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) Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Idiopathic Inflammatory Myopathies
- What is being tested?
- Rapcabtagene autoleucel and Active Comparator Option
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 68 sitesin 15 countries
- Study dates
- 17 Dec 2024to 29 Nov 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
68 sites in 15 countries.
68 sites in 15 countries
Japan11 sites
Novartis Investigative Site
Bunkyo-ku, Tokyo, 113-8603
Novartis Investigative Site
Bunkyo-ku, Tokyo, 1138519
Novartis Investigative Site
Fukuoka, 8128582
Novartis Investigative Site
Ishikawa, 9208641
Novartis Investigative Site
Izumo, Shimane, 6938501
Novartis Investigative Site
Kyoto, 6068507
Novartis Investigative Site
Sapporo, Hokkaido, 060-8648
Novartis Investigative Site
Sendai, Miyagi, 9808574
Novartis Investigative Site
Shinjuku-ku, Tokyo, 1608582
Novartis Investigative Site
Suita, Osaka, 565-0871
Novartis Investigative Site
Yokohama, Kanagawa, 236-0004
United States10 sites
Augusta University Georgia
Augusta, Georgia, 30912
University Of Alabama
Birmingham, Alabama, 35294
Northwestern University
Chicago, Illinois, 60611
Baylor University Medical Center
Dallas, Texas, 75246
Duke Clinical Research Institute
Durham, North Carolina, 27710
Houston Methodist Hospital
Houston, Texas, 77030
University Of Iowa
Iowa City, Iowa, 52242
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15261
LDS Hospital
Salt Lake City, Utah, 84143
FL Medical Clinic Orlando Health
Zephyrhills, Florida, 33542
Spain9 sites
Novartis Investigative Site
Barcelona, 08035
Novartis Investigative Site
Córdoba, 14004
Novartis Investigative Site
Madrid, 28009
Novartis Investigative Site
Madrid, 28041
Novartis Investigative Site
Málaga, 29010
Novartis Investigative Site
Pamplona, Navarre, 31008
Novartis Investigative Site
Salamanca, 37007
Novartis Investigative Site
Santander, Cantabria, 39008
Novartis Investigative Site
Santiago Compostela, A Coruna, 15706
Germany8 sites
Novartis Investigative Site
Aachen, 52074
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Hamburg, 20246
Novartis Investigative Site
Jena, Thuringia, 07740
Novartis Investigative Site
Leipzig, Saxony, 04103
Novartis Investigative Site
Mainz, 55131
Novartis Investigative Site
Nuremberg, 90419
Novartis Investigative Site
Ulm, 89081
France6 sites
Novartis Investigative Site
Brest, 29200
Novartis Investigative Site
Lille, 59037
Novartis Investigative Site
Lyon, 69003
Novartis Investigative Site
Marseille, 13885
Novartis Investigative Site
Paris, 75013
Novartis Investigative Site
Rennes, 35033
Italy6 sites
Novartis Investigative Site
Ancona, AN, 60126
Novartis Investigative Site
Brescia, BS, 25123
Novartis Investigative Site
Milan, MI, 20122
Novartis Investigative Site
Monza, MB, 20900
Novartis Investigative Site
Rozzano, MI, 20089
Novartis Investigative Site
Verona, VR, 37134
Brazil3 sites
Novartis Investigative Site
Barretos, São Paulo, 14784 400
Novartis Investigative Site
São Paulo, São Paulo, 01232-010
Novartis Investigative Site
São Paulo, São Paulo, 01308-050
Israel3 sites
Novartis Investigative Site
Haifa, 3109601
Novartis Investigative Site
Ramat Gan, 5265601
Novartis Investigative Site
Tel Aviv, 6423906
Singapore3 sites
Novartis Investigative Site
Singapore, 119074
Novartis Investigative Site
Singapore, 169608
Novartis Investigative Site
Singapore, S308433
Taiwan3 sites
Novartis Investigative Site
Kaohsiung City, 83301
Novartis Investigative Site
Taichung, 407219
Novartis Investigative Site
Taipei, 10002
United Kingdom2 sites
Novartis Investigative Site
London, NW1 2BU
Novartis Investigative Site
Sheffield, South Yorkshire, S10 2JF
Netherlands1 site
Novartis Investigative Site
Amsterdam, North Holland, 1105 AZ
About this study
Official 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)
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)
Read the 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.
- Conditions
- Idiopathic Inflammatory Myopathies
- Being tested
- Rapcabtagene autoleucel, Active Comparator Option
- Keywords
- Chimeric Antigen Receptor T cells (CAR-T), rapcabtagene autoleucel, idiopathic inflammatory myopathies (IIM), dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy, Interstitial Lung Disease
- Medical subject headings
- Myositis, Dermatomyositis, Lung Diseases, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rapcabtagene autoleucelBiological
Also called YTB323
Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.
Active Comparator OptionOther
Investigator choice of treatment as per protocol
| Study group | Role | What they receive |
|---|---|---|
| Rapcabtagene autoleucel | Experimental | Single infusion of rapcabtagene autoleucel (YTB323) Receives: Biological: Rapcabtagene autoleucel |
| Comparator | Active comparator | Investigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab) Receives: Other: Active Comparator Option |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at 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.
Measured: Week 52
Secondary outcomes (4)
Adjusted annual cumulative glucocorticoid dose up to Week 52
The total amount of glucocorticoids administered over the course of a year measured up to the 52nd week of treatment.
Measured: Week 52
Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52
The difference in the percentage of the predicted Forced Vital Capacity (FVC) from the start of the study to the 52nd week.
Measured: Baseline, Week 52
Proportion of participants achieving major improvement in TIS at 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.
Measured: Week 52
Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52
The difference in the fatigue levels reported by participants from the start of the study to the 52nd week.
Measured: Baseline, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2024
- Study start17 Dec 2024
- Primary completion9 Mar 2029 (estimated)
- Study completion29 Nov 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Inflammatory MyopathiesStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06665256
- Sponsor's protocol ID
- CYTB323L12201
- Status last verified
- Jun 2026
- First submitted
- 29 Oct 2024
- Last updated
- 23 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT06665256
- Acronym / sponsor ID
- CYTB323L12201
- Conditions
- Idiopathic Inflammatory Myopathies
- Keywords
- Chimeric Antigen Receptor T cells (CAR-T), rapcabtagene autoleucel, idiopathic inflammatory myopathies (IIM), dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy, Interstitial Lung Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 17 Dec 2024 (actual)
- Primary completion
- 9 Mar 2029 (estimated)
- Study completion
- 29 Nov 2032 (estimated)
- First posted
- 30 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rapcabtagene autoleucel | Experimental | Biological: Rapcabtagene autoleucelSingle infusion of rapcabtagene autoleucel (YTB323) |
| Comparator | Active comparator | Other: Active Comparator OptionInvestigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rapcabtagene autoleucelAlso: YTB323Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days. | Biological | Rapcabtagene autoleucel |
| Active Comparator OptionInvestigator choice of treatment as per protocol | Other | Comparator |
Primary outcome
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.
4 secondary outcomes
Adjusted annual cumulative glucocorticoid dose up to Week 52
Time frame: Week 52
Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52
Time frame: Baseline, Week 52
Proportion of participants achieving major improvement in TIS at Week 52
Time frame: Week 52
Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52
Time frame: Baseline, Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
68 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rapcabtagene autoleucel and Active Comparator Option in people with Idiopathic Inflammatory Myopathies, run by Novartis Pharmaceuticals at 68 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 68 sites in Japan, United States, Spain, Germany, France and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at Week 52 (time frame: Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2026.