RECRUITINGPhase 2INTERVENTIONAL
Phase 2 Study of Rapcabtagene Autoleucel in Myositis
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)
About This Trial
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)
Who May Be Eligible (Plain English)
Key Who May Qualify:
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 Who Should NOT Join This Trial:
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. Inyour organs (liver, kidneys, etc.) are working well enough based on blood tests
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.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
BIOLOGICAL
Rapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.
OTHER
Active Comparator Option
Investigator choice of treatment as per protocol
Locations (20)
University Of Alabama
Birmingham, Alabama, United States
FL Medical Clinic Orlando Health
Zephyrhills, Florida, United States
Northwestern University
Chicago, Illinois, United States
Northwestern University
Chicago, Illinois, United States
University Of Iowa
Iowa City, Iowa, United States
LDS Hospital
Salt Lake City, Utah, United States
Novartis Investigative Site
Brest, France
Novartis Investigative Site
Lille, France
Novartis Investigative Site
Lyon, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Rennes, France
Novartis Investigative Site
Cologne, North Rhine-Westphalia, Germany
Novartis Investigative Site
Leipzig, Saxony, Germany
Novartis Investigative Site
Jena, Thuringia, Germany
Novartis Investigative Site
Aachen, Germany
Novartis Investigative Site
Hamburg, Germany
Novartis Investigative Site
Mainz, Germany
Novartis Investigative Site
Nuremberg, Germany
Novartis Investigative Site
Ulm, Germany
Novartis Investigative Site
Haifa, Israel