Phase 2 Study of Rapcabtagene Autoleucel in Myositis

Study Purpose

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)

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years - 65 Years
Gender	All

More Inclusion & 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 Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06665256
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Novartis Pharmaceuticals
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Novartis Pharmaceuticals
Principal Investigator Affiliation	Novartis Pharmaceuticals
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	France, Germany, Israel, Italy, Japan, Singapore, Spain, Taiwan, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Idiopathic Inflammatory Myopathies

Additional Details

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.

Arms & Interventions

Arms

Experimental: Rapcabtagene autoleucel

Single infusion of rapcabtagene autoleucel (YTB323)

Active Comparator: Comparator

Investigator choice of treatment as per protocol

Interventions

Biological: - Rapcabtagene autoleucel

Single infusion of rapcabtagene autoleucel

Other: - Active Comparator Option

Investigator choice of treatment as per protocol

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Northwestern University, Chicago 4887398, Illinois 4896861

Status

Recruiting

Address

Northwestern University

Chicago 4887398, Illinois 4896861, 60611

Site Contact

Matthew Selle

Clinical Trial Finder