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

Last updated: January 24, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Scar Tissue

Treatment

rapcabtagene autoleucel

rituximab

Clinical Study ID

NCT06655896
CYTB323K12201
2023-510380-34-00
  • Ages 18-65
  • All Genders

Study Summary

The purpose of this study is to evaluate the efficacy, safety and tolerability of rapcabtagene autoleucel (administered once following lymphodepletion) in participants with severe refractory diffuse cutaneous systemic sclerosis relative to rituximab.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Participant must fulfill the 2013 American College of Rheumatology/ European LeagueAgainst Rheumatism classification criteria for systemic sclerosis and meet thediffuse cutaneous SSc (dcSSc) subset classification according to LeRoy.

  2. Disease onset from the first non-Raynaud symptoms attributable to SSc (e.g., puffyhands, scleroderma, digital ulcers, arthralgia, dyspnea) within 7 years prior to theScreening visit.

  3. Severe, progressive systemic sclerosis disease defined by at least one of thefollowing:

  • Progressive systemic sclerosis-associated interstitial lung disease

  • Severe, progressive systemic sclerosis skin disease

  • Clinically significant systemic sclerosis-associated cardiac involvement atScreening

Exclusion

Exclusion Criteria:

  1. Any condition during Screening that could prevent a complete washout of medicationsas required per protocol or could otherwise make the participant ineligible foranti-CD19 CAR-T therapy and further participation in the study, as judged by theInvestigator.

  2. Participants with history of hypersensitivity to excipients in rapcabtageneautoleucel or to rituximab.

  3. Any participant for whom treatment with rituximab is clinically inappropriate in theopinion of the investigator.

  4. Any medical conditions that are not related to SSc that, in the opinion of theInvestigator, would jeopardize the ability of the participant to toleratelymphodepletion and anti-CD19 CAR-T cell therapy.

  5. Rheumatic disease other than dcSSc, (except secondary Sjogren's syndrome orscleroderma myopathy), limited cutaneous systemic sclerosis (lcSSc) or sinescleroderma at Screening.

  6. Participants with pre-existing pulmonary hypertension.

  7. Significant renal pathology at Screening, including:

  • Active SSc renal crisis

  • Confirmed diagnosis of glomerulonephritis

  1. Participants with uncontrolled stage II hypertension at Screening.

  2. Vaccination with live attenuated vaccines within 6 weeks prior to randomization.

Other protocol-defined inclusion/exclusion criteria may apply.

Study Design

Total Participants: 86
Treatment Group(s): 2
Primary Treatment: rapcabtagene autoleucel
Phase: 2
Study Start date:
October 29, 2024
Estimated Completion Date:
December 17, 2030

Study Description

This is a phase 2, multi-part, three-year, randomized, open-label, assessor-blinded, multicenter study to evaluate the efficacy and safety of rapcabtagene autoleucel versus rituximab in participants with severe refractory diffuse cutaneous systemic sclerosis (dcSSc) . This study comprises two cohorts:

  • A Lead-in Cohort enrolling participants to receive rapcabtagene autoleucel.

  • A Randomized Cohort enrolling participants to receive rapcabtagene autoleucel or rituximab.

After end of study, participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Connect with a study center

  • Novartis Investigative Site

    Darlinghurst, New South Wales 2010
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    Bordeaux Cedex, 33076
    France

    Active - Recruiting

  • Novartis Investigative Site

    Rennes, 35043
    France

    Active - Recruiting

  • Novartis Investigative Site

    Jena, 07740
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Haifa, 3109601
    Israel

    Active - Recruiting

  • Novartis Investigative Site

    Ramat Gan, 52621
    Israel

    Active - Recruiting

  • Novartis Investigative Site

    Ancona, AN 60126
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Genova, GE 16132
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Roma, RM 00168
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Sapporo city, Hokkaido 060 8648
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Kanazawa, Ishikawa 920 8641
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Singapore, 169608
    Singapore

    Active - Recruiting

  • Novartis Investigative Site

    Barcelona, 08041
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Madrid, 28041
    Spain

    Active - Recruiting

  • Northwestern University

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • University Of Iowa

    Iowa City, Iowa 52242
    United States

    Active - Recruiting

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