NCT06665256

Brief 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)

Trial Health

88
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
123

participants targeted

Target at P75+ for phase_2

Timeline
51mo left

Started Dec 2024

Longer than P75 for phase_2

Geographic Reach
13 countries

55 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress25%
Dec 2024Jul 2030

First Submitted

Initial submission to the registry

October 29, 2024

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 30, 2024

Completed
2 months until next milestone

Study Start

First participant enrolled

December 17, 2024

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 17, 2029

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

July 17, 2030

Last Updated

February 5, 2026

Status Verified

February 1, 2026

Enrollment Period

4.6 years

First QC Date

October 29, 2024

Last Update Submit

February 4, 2026

Conditions

Idiopathic Inflammatory Myopathies

Keywords

Chimeric Antigen Receptor T cells (CAR-T)rapcabtagene autoleucelidiopathic inflammatory myopathies (IIM)dermatomyositisAnti-Synthetase SyndromeImmune-Mediated Necrotizing MyopathyInterstitial Lung Disease

Outcome Measures

Primary Outcomes (1)

  • 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.

    Week 52

Secondary Outcomes (4)

  • Adjusted annual cumulative glucocorticoid dose up to Week 52

    Week 52

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

    Baseline, Week 52

  • Proportion of participants achieving major improvement in TIS at Week 52

    Week 52

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

    Baseline, Week 52

Study Arms (2)

Rapcabtagene autoleucel

EXPERIMENTAL

Single infusion of rapcabtagene autoleucel (YTB323)

Biological: Rapcabtagene autoleucel

Comparator

ACTIVE COMPARATOR

Investigator choice of treatment as per protocol

Other: Active Comparator Option

Interventions

Rapcabtagene autoleucelBIOLOGICAL

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

Also known as: YTB323
Rapcabtagene autoleucel
Active Comparator OptionOTHER

Investigator choice of treatment as per protocol

Comparator

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • Participant must meet criteria for severe myositis

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (55)

University Of Alabama

Birmingham, Alabama, 35294, United States

RECRUITING

FL Medical Clinic Orlando Health

Zephyrhills, Florida, 33542, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

University Of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

LDS Hospital

Salt Lake City, Utah, 84143, United States

RECRUITING

Novartis Investigative Site

Brest, 29200, France

RECRUITING

Novartis Investigative Site

Lille, 59037, France

SUSPENDED

Novartis Investigative Site

Lyon, 69003, France

RECRUITING

Novartis Investigative Site

Paris, 75013, France

RECRUITING

Novartis Investigative Site

Rennes, 35033, France

RECRUITING

Novartis Investigative Site

Cologne, North Rhine-Westphalia, 50937, Germany

RECRUITING

Novartis Investigative Site

Leipzig, Saxony, 04103, Germany

RECRUITING

Novartis Investigative Site

Jena, Thuringia, 07740, Germany

RECRUITING

Novartis Investigative Site

Aachen, 52074, Germany

RECRUITING

Novartis Investigative Site

Hamburg, 20246, Germany

RECRUITING

Novartis Investigative Site

Mainz, 55131, Germany

RECRUITING

Novartis Investigative Site

Nuremberg, 90419, Germany

RECRUITING

Novartis Investigative Site

Ulm, 89081, Germany

RECRUITING

Novartis Investigative Site

Haifa, 3109601, Israel

RECRUITING

Novartis Investigative Site

Ramat Gan, 5265601, Israel

RECRUITING

Novartis Investigative Site

Tel Aviv, 6423906, Israel

RECRUITING

Novartis Investigative Site

Brescia, BS, 25123, Italy

RECRUITING

Novartis Investigative Site

Monza, MB, 20900, Italy

RECRUITING

Novartis Investigative Site

Milan, MI, 20122, Italy

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608648, Japan

RECRUITING

Novartis Investigative Site

Kanazawa, Ishikawa-ken, 920 8641, Japan

RECRUITING

Novartis Investigative Site

Yokohama, Kanagawa-ku, 236-0004, Japan

RECRUITING

Novartis Investigative Site

Sendai, Miyagi, 9808574, Japan

RECRUITING

Novartis Investigative Site

Suita, Osaka, 565 0871, Japan

RECRUITING

Novartis Investigative Site

Izumo, Shimane, 693 8501, Japan

RECRUITING

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8603, Japan

RECRUITING

Novartis Investigative Site

Bunkyo-ku, Tokyo, 1138519, Japan

RECRUITING

Novartis Investigative Site

Shinjuku-ku, Tokyo, 1608582, Japan

RECRUITING

Novartis Investigative Site

Fukuoka, 8128582, Japan

RECRUITING

Novartis Investigative Site

Kyoto, 6068507, Japan

RECRUITING

Novartis Investigative Site

Amsterdam, North Holland, 1105 AZ, Netherlands

RECRUITING

Novartis Investigative Site

Riyadh, 11211, Saudi Arabia

RECRUITING

Novartis Investigative Site

Singapore, 119074, Singapore

RECRUITING

Novartis Investigative Site

Singapore, 169608, Singapore

RECRUITING

Novartis Investigative Site

Singapore, S308433, Singapore

RECRUITING

Novartis Investigative Site

Santiago Compostela, A Coruna, 15706, Spain

RECRUITING

Novartis Investigative Site

Santander, Cantabria, 39008, Spain

RECRUITING

Novartis Investigative Site

Pamplona, Navarre, 31008, Spain

RECRUITING

Novartis Investigative Site

Barcelona, 08035, Spain

RECRUITING

Novartis Investigative Site

Córdoba, 14004, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28041, Spain

RECRUITING

Novartis Investigative Site

Málaga, 29010, Spain

RECRUITING

Novartis Investigative Site

Salamanca, 37007, Spain

RECRUITING

Novartis Investigative Site

Geneva, 1211, Switzerland

RECRUITING

Novartis Investigative Site

Kaohsiung City, 83301, Taiwan

RECRUITING

Novartis Investigative Site

Taichung, 407219, Taiwan

RECRUITING

Novartis Investigative Site

Taipei, 10002, Taiwan

RECRUITING

Novartis Investigative Site

Sheffield, South Yorkshire, S10 2JF, United Kingdom

RECRUITING

Novartis Investigative Site

London, NW1 2BU, United Kingdom

RECRUITING

MeSH Terms

Conditions

MyositisDermatomyositisLung Diseases, Interstitial

Condition Hierarchy (Ancestors)

Muscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesPolymyositisConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin DiseasesLung DiseasesRespiratory Tract Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111novartis.email@novartis.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 29, 2024

First Posted

October 30, 2024

Study Start

December 17, 2024

Primary Completion (Estimated)

July 17, 2029

Study Completion (Estimated)

July 17, 2030

Last Updated

February 5, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will share

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

Locations

NL(1)IL(3)SA(1)CH(1)DE(8)FR(5)SG(3)TW(3)ES(8)US(6)GB(2)JP(11)IT(3)