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, two-year, randomized, assessor- blinded, 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.

After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Enrollment: 123 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Men and women, aged >18 and ≤65 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 ≤18.5 or ≥35 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: Single Blind

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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