severe refractory diffuse cutaneous systemic sclerosis
Conditions
Interventions
Experimental: infusion of rapcabtagene autoleucel
Active Comparator: rituximab intravenous infusion (i.v.) as per protocol
Participants in the SoC arm whose signs and symptoms are not fully controlle
Sponsors
Yamauchi Kyosuke
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Participant must fulfill the 2013 American College of Rheumatology/ European League Against Rheumatism classification criteria for systemic sclerosis and meet the diffuse cutaneous SSc (dcSSc) subset classification according to LeRoy. - Disease onset from the first non-Raynaud symptoms attributable to SSc (e.g., puffy hands, scleroderma, digital ulcers, arthralgia, dyspnea) within 7 years prior to the Screening visit. - Severe, progressive systemic sclerosis disease defined by at least one of the following: - Progressive systemic sclerosis-associated interstitial lung disease - Severe, progressive systemic sclerosis skin disease - Clinically significant systemic sclerosis-associated cardiac involvement at Screening
Exclusion criteria
Exclusion criteria: - Any condition during Screening that could prevent a complete washout of medications as required per protocol or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in the study, as judged by the Investigator. - Participants with history of hypersensitivity to excipients in rapcabtagene autoleucel or to rituximab. - Any participant for whom treatment with rituximab is clinically inappropriate in the opinion of the investigator. - Any medical conditions that are not related to SSc that, in the opinion of the Investigator, would jeopardize the ability of the participant to tolerate lymphodepletion and anti-CD19 CAR-T cell therapy. - Rheumatic disease other than dcSSc, (except secondary Sjogren's syndrome or scleroderma myopathy), limited cutaneous systemic sclerosis (lcSSc) or sine scleroderma at Screening. - Participants with pre-existing pulmonary hypertension. - Significant renal pathology at Screening, - Participants with uncontrolled stage II hypertension at Screening. - Vaccination with live attenuated vaccines within 6 weeks prior to randomization.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Achievement of a treatment response as per the Revised Composite Response Index in Systemic Sclerosis 50 (rCRISS50) definition at Week 52. | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Netherlands, Singapore, South Korea, Spain, Switzerland, Taiwan, UK, United States
Contacts
Public ContactKyosuke Yamauchi
Novartis Pharma. K.K.
Outcome results
None listed