Phase 2 Study of Rapcabtagene Autoleucel in Myositis

NCT ID: NCT06665256

Last Updated: 2026-02-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

123 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-17

Study Completion Date

2030-07-17

Brief Summary

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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

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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

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Idiopathic Inflammatory Myopathies

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Rapcabtagene autoleucel

Single infusion of rapcabtagene autoleucel (YTB323)

Group Type EXPERIMENTAL

Rapcabtagene autoleucel

Intervention Type BIOLOGICAL

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

Comparator

Investigator choice of treatment as per protocol

Group Type ACTIVE_COMPARATOR

Active Comparator Option

Intervention Type OTHER

Investigator choice of treatment as per protocol

Interventions

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Rapcabtagene autoleucel

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

Intervention Type BIOLOGICAL

Active Comparator Option

Investigator choice of treatment as per protocol

Intervention Type OTHER

Other Intervention Names

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YTB323

Eligibility Criteria

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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
4. Participant must meet criteria for severe myositis

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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University Of Alabama

Birmingham, Alabama, United States

Site Status RECRUITING

FL Medical Clinic Orlando Health

Zephyrhills, Florida, United States

Site Status RECRUITING

Northwestern University

Chicago, Illinois, United States

Site Status RECRUITING

Northwestern University

Chicago, Illinois, United States

Site Status RECRUITING

University Of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

LDS Hospital

Salt Lake City, Utah, United States

Site Status RECRUITING

Novartis Investigative Site

Brest, , France

Site Status RECRUITING

Novartis Investigative Site

Lille, , France

Site Status SUSPENDED

Novartis Investigative Site

Lyon, , France

Site Status RECRUITING

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Paris, , France

Site Status RECRUITING

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Rennes, , France

Site Status RECRUITING

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Cologne, North Rhine-Westphalia, Germany

Site Status RECRUITING

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Leipzig, Saxony, Germany

Site Status RECRUITING

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Jena, Thuringia, Germany

Site Status RECRUITING

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Aachen, , Germany

Site Status RECRUITING

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Hamburg, , Germany

Site Status RECRUITING

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Mainz, , Germany

Site Status RECRUITING

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Nuremberg, , Germany

Site Status RECRUITING

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Ulm, , Germany

Site Status RECRUITING

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Haifa, , Israel

Site Status RECRUITING

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Ramat Gan, , Israel

Site Status RECRUITING

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Tel Aviv, , Israel

Site Status RECRUITING

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Brescia, BS, Italy

Site Status RECRUITING

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Monza, MB, Italy

Site Status RECRUITING

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Milan, MI, Italy

Site Status RECRUITING

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Sapporo, Hokkaido, Japan

Site Status RECRUITING

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Kanazawa, Ishikawa-ken, Japan

Site Status RECRUITING

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Yokohama, Kanagawa-ku, Japan

Site Status RECRUITING

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Sendai, Miyagi, Japan

Site Status RECRUITING

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Suita, Osaka, Japan

Site Status RECRUITING

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Izumo, Shimane, Japan

Site Status RECRUITING

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Bunkyo-ku, Tokyo, Japan

Site Status RECRUITING

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Bunkyo-ku, Tokyo, Japan

Site Status RECRUITING

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Shinjuku-ku, Tokyo, Japan

Site Status RECRUITING

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Fukuoka, , Japan

Site Status RECRUITING

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Kyoto, , Japan

Site Status RECRUITING

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Amsterdam, North Holland, Netherlands

Site Status RECRUITING

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Riyadh, , Saudi Arabia

Site Status RECRUITING

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Singapore, , Singapore

Site Status RECRUITING

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Singapore, , Singapore

Site Status RECRUITING

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Singapore, , Singapore

Site Status RECRUITING

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Santiago Compostela, A Coruna, Spain

Site Status RECRUITING

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Santander, Cantabria, Spain

Site Status RECRUITING

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Pamplona, Navarre, Spain

Site Status RECRUITING

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Barcelona, , Spain

Site Status RECRUITING

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Córdoba, , Spain

Site Status RECRUITING

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Madrid, , Spain

Site Status RECRUITING

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Málaga, , Spain

Site Status RECRUITING

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Salamanca, , Spain

Site Status RECRUITING

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Geneva, , Switzerland

Site Status RECRUITING

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Kaohsiung City, , Taiwan

Site Status RECRUITING

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Taichung, , Taiwan

Site Status RECRUITING

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Taipei, , Taiwan

Site Status RECRUITING

Novartis Investigative Site

Sheffield, South Yorkshire, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Countries

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United States France Germany Israel Italy Japan Netherlands Saudi Arabia Singapore Spain Switzerland Taiwan United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

1-888-669-6682

Novartis Pharmaceuticals

Role: CONTACT

+41613241111

Facility Contacts

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Hannah Howell

Role: primary

205-996-7438

William Daily Johnston

Role: primary

Matthew Selle

Role: primary

Matthew Selle

Role: primary

Ashley Pieper

Role: primary

319-356-2197

Joshua Kunz

Role: primary

801-408-4724

Other Identifiers

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CYTB323L12201

Identifier Type: -

Identifier Source: org_study_id

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