Phase 2 Study of Rapcabtagene Autoleucel in Myositis

Novartis

Status and phase: Enrolling; Phase 2

Conditions: Idiopathic Inflammatory Myopathies

Treatments: Biological: Rapcabtagene autoleucel; Other: Active Comparator Option

Study type: Interventional

Funder types: Industry

Identifiers

NCT06665256

CYTB323L12201

Details and patient eligibility

About

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)

Full 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.

Enrollment: 123 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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
4. Participant must meet criteria for severe myositis

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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123 participants in 2 patient groups

Rapcabtagene autoleucel

Experimental group

Description:

Single infusion of rapcabtagene autoleucel (YTB323)

Treatment:

Biological: Rapcabtagene autoleucel

Comparator

Active Comparator group

Description:

Investigator choice of treatment as per protocol

Treatment:

Other: Active Comparator Option