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Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Diffuse Cutaneous Systemic Sclerosis (CYTB323K12201)

Phase II study evaluating rapcabtagene autoleucel in participants with diffuse cutaneous systemic sclerosis (CYTB323K12201)

Yamauchi Kyosuke

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku2@novartis.com

Yamauchi Kyosuke

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku2@novartis.com

Recruiting

Dec. 10, 2024

Interventional

randomized controlled trial

open(masking not used)

active control

parallel assignment

treatment purpose

- 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

- 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, including:
- Active SSc renal crisis
- Confirmed diagnosis of glomerulonephritis
- Participants with uncontrolled stage II hypertension at Screening.
- Vaccination with live attenuated vaccines within 6 weeks prior to randomization.

Both

severe refractory diffuse cutaneous systemic sclerosis

Experimental: Single infusion of rapcabtagene autoleucel (YTB323)
Active Comparator: rituximab intravenous infusion (i.v.) as per protocol

Achievement of a treatment response as per the Revised Composite Response Index in Systemic Sclerosis 50 (rCRISS50) definition at Week 52.

+81-11-706-7061

Aug. 22, 2024

Yes

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Singapore/United States