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

a study on Diffuse Scleroderma Systemic Sclerosis

Summary

Eligibility
for people ages 18-65 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around
Principal Investigator
by Emily Von Scheven
Headshot of Emily Von Scheven
Emily Von Scheven

Description

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.

Official Title

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

Details

This is a phase 2, multi-part, five-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. Participants in the rituximab arm whose disease is not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible per protocol After end of study, participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Keywords

Scleroderma, Diffuse, Diffuse cutaneous systemic sclerosis (dcSSc), revised Composite Response Index in Systemic Sclerosis (rCRISS), modified Rodnan skin score (mRSS), forced vital capacity (FVC), Systemic Scleroderma, Diffuse Scleroderma, Sclerosis, Rituximab, rapcabtagene autoleucel

Eligibility

You can join if…

Open to people ages 18-65

  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

You CAN'T join if...

  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.

Locations

  • UCSF accepting new patients
    San Francisco California 94115 United States
  • Avera Cancer accepting new patients
    Sioux Falls South Dakota 57105 United States

Lead Scientist at UCSF

  • Emily Von Scheven
    My long-term goal is to improve the care and outcomes of children with rheumatic diseases. My research has focused on defining and improving outcomes in pediatric-onset Lupus, evaluating and treating childhood osteoporosis and addressing the transition needs of patients as they transition from pediatric to adult providers.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Novartis Pharmaceuticals
ID
NCT06655896
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 86 study participants
Last Updated
Contact us about this trial