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

Launched by NOVARTIS PHARMACEUTICALS · Oct 22, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment called rapcabtagene autoleucel for people with a severe form of scleroderma known as diffuse cutaneous systemic sclerosis. The study aims to see how effective and safe this treatment is compared to an existing medication called rituximab. Participants in the trial will receive rapcabtagene autoleucel after a process called lymphodepletion, which helps prepare their body for the treatment.

To join the study, participants need to have been diagnosed with diffuse cutaneous scleroderma within the last seven years and show signs of severe disease, such as lung or heart involvement. The trial is open to adults aged 65 to 74 and younger adults aged 23 to 740. Those interested will undergo careful screening to ensure they meet all the necessary criteria. If eligible, participants can expect close monitoring throughout the trial to assess how well the treatment works and how they tolerate it. It's an exciting opportunity to contribute to research that may lead to better options for managing this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. 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.
• • 2. 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.
• 3. 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:
• • 1. 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.
• • 2. Participants with history of hypersensitivity to excipients in rapcabtagene autoleucel or to rituximab.
• • 3. Any participant for whom treatment with rituximab is clinically inappropriate in the opinion of the investigator.
• • 4. 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.
• • 5. Rheumatic disease other than dcSSc, (except secondary Sjogren's syndrome or scleroderma myopathy),including limited cutaneous systemic sclerosis (lcSSc) or sine scleroderma at Screening.
• • 6. Participants with pre-existing pulmonary hypertension.
• • 7. Significant renal pathology at Screening.
• • 8. Participants with uncontrolled stage II hypertension at Screening.
• • 9. Vaccination with live attenuated vaccines within 6 weeks prior to randomization.
• • Other protocol-defined inclusion/exclusion criteria may apply.