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Evaluating a new treatment for severe scleroderma

A Phase II, Multi-part, Randomized, Open-label, Assessor-blinded, Active-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Rituximab Treatment in Participants With Severe Refractory Diffuse Cutaneous Systemic Sclerosis

Phase 2 Interventional Novartis · NCT06655896

This study is testing a new treatment called rapcabtagene autoleucel for people with severe scleroderma to see if it works better than the standard treatment, rituximab.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment96 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorNovartis Industry-sponsored
Drugs / interventionsrituximab, CAR-T
Locations87 sites (Los Angeles, California and 86 other locations)
Trial IDNCT06655896 on ClinicalTrials.gov

What this trial studies

This phase 2 study aims to assess the efficacy, safety, and tolerability of rapcabtagene autoleucel in patients with severe refractory diffuse cutaneous systemic sclerosis (dcSSc) compared to rituximab. The study is designed as a randomized, open-label, multicenter trial that includes a lead-in cohort and a randomized cohort. Participants will receive either rapcabtagene autoleucel or rituximab, with the option for those on rituximab to switch to rapcabtagene autoleucel if their disease is not adequately controlled. Following treatment, participants will enter a long-term follow-up period to monitor outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals diagnosed with severe refractory diffuse cutaneous systemic sclerosis within 7 years of symptom onset.

Not a fit: Patients with conditions that prevent them from receiving anti-CD19 CAR-T therapy or those who do not meet the eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from severe scleroderma, potentially improving their quality of life and disease management.

How similar studies have performed: While this approach is novel in the context of scleroderma, similar CAR-T therapies have shown promise in other autoimmune conditions, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion 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
4. All recommended vaccinations received according to institutional, local or global guidelines for immuno-compromised patients.

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.

Where this trial is running

Los Angeles, California and 86 other locations

+37 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Scleroderma, DiffuseDiffuse cutaneous systemic sclerosisrevised Composite Response Index in Systemic Sclerosismodified Rodnan skin scoreforced vital capacity
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.