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

Recruiting

• Conditions: severe refractory diffuse cutaneous systemic sclerosis

• Registration Number: jRCT2013240056
• Lead Sponsor: Novartis Pharma. K.K.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-11-28
• Last Update Posted: 2024-11-28

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 11

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

• 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.

Study & Design

• Study Type: Interventional
• Study Design: parallel assignment

Primary Outcome Measures

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