Study comparing rapcabtagene autoleucel and rituximab in patients with severe refractory diffuse cutaneous systemic sclerosis

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What is this study about?

This study focuses on people with diffuse cutaneous systemic sclerosis (dcSSc), a severe autoimmune disease that causes hardening of the skin and can affect internal organs. The main treatment being tested is rapcabtagene autoleucel (also known as YTB323), which is a type of cell therapy where a patient’s own immune cells are modified to fight the disease. This treatment will be compared to rituximab, which is an existing medication used to treat autoimmune conditions.

The purpose of this research is to determine if rapcabtagene autoleucel is more effective than rituximab in treating severe dcSSc that has not responded well to previous treatments. Before receiving the main treatment, patients may receive preparation medications including cyclophosphamide, fludarabine phosphate, and tocilizumab. All medications will be given through an intravenous line directly into the bloodstream.

Throughout the study, which lasts for 52 weeks, doctors will monitor changes in lung function, skin thickness, and ability to perform daily activities. They will also track any side effects that may occur during or after treatment. The study will continue to follow participants for several years to understand the long-term effects of the treatment.

1 Initial Treatment Phase

You will receive lymphodepletion therapy consisting of fludarabine phosphate and cyclophosphamide through intravenous infusion

This preparatory treatment helps your body receive the main therapy

2 Main Treatment Administration

You will receive a single infusion of rapcabtagene autoleucel (YTB323) through intravenous administration

This is a specialized treatment that uses modified immune cells to target your condition

3 Monitoring Period

Regular assessments will track your progress over 52 weeks

Medical staff will measure changes in your lung function, skin condition, and overall health status

Your ability to perform daily activities will be evaluated throughout this period

4 Safety Evaluations

Regular health checks will include blood tests, vital signs monitoring, and heart activity recordings (ECG)

If needed, you may receive tocilizumab through intravenous infusion to manage specific side effects

Any health changes or side effects will be documented and addressed

5 Final Assessment

At week 52, a comprehensive evaluation will determine the effectiveness of the treatment

This includes measuring improvements in your condition using specialized assessment tools

Long-term follow-up will continue to monitor your health status

Who Can Join the Study?

  • You must be diagnosed with diffuse cutaneous systemic sclerosis (dcSSc) according to specific medical criteria established by rheumatology experts
  • Your disease symptoms (not including Raynaud’s phenomenon – a condition where fingers and toes feel numb and cold) must have started within the last 7 years. These symptoms may include:
    • Swollen hands
    • Hardening of the skin
    • Sores on fingers or toes
    • Joint pain
    • Difficulty breathing
  • You must have severe, progressive disease with at least one of these conditions:
    • Progressive lung involvement (breathing problems related to systemic sclerosis)
    • Severe, worsening skin involvement (skin hardening that is getting worse)
    • Heart problems related to systemic sclerosis
  • Both men and women can participate in this study
  • Adults of all ages can participate

Who Cannot Join the Study?

  • Age below 18 years or above 65 years
  • Previous treatment with rituximab (a type of medication that affects immune system cells)
  • Active or chronic infections, including hepatitis B, hepatitis C, or HIV
  • History of severe allergic reactions to biological treatments
  • Current pregnancy or breastfeeding
  • Significant heart problems or uncontrolled high blood pressure
  • Severe kidney or liver problems
  • Active cancer or history of cancer in the past 5 years (except for successfully treated skin cancer)
  • Major surgery within 8 weeks before starting the study
  • Participation in other clinical trials within 30 days before this study
  • Mental conditions that could interfere with following study procedures
  • Substance abuse within the past year
  • Any condition that, in the opinion of study doctors, would make participation unsafe
  • Unable to provide informed consent

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Revmatologicky Ustav Prague Czechia
Klinikum Nuernberg Nürnberg Germany
Universita Cattolica Del Sacro Cuore Rome Italy
Universitair Ziekenhuis Gent Gent Belgium
Azienda Sanitaria Universitaria Friuli Centrale Udine Italy
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital Universitario 12 De Octubre Madrid Spain
Hospital Santa Maria Della Misericordia Perugia Italy
Azienda Sanitaria Locale Di Pescara Pescara Italy
Universitaetsklinikum Schleswig-Holstein AöR Kiel Germany
University Of Debrecen Debrecen Hungary
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ospedale San Raffaele S.r.l. Milan Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Semmelweis University Budapest Hungary
IRCCS Ospedale Policlinico San Martino Genoa Italy
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse Vienna Austria
Centre Hospitalier Universitaire De Montpellier Montpellier France
Universita’ Politecnica Delle Marche Ancona Italy
Universidade De Santiago De Compostela Santiago De Compostela Spain
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Centre Hospitalier Universitaire De Rennes Rennes France
University Hospital Olomouc Olomouc Czechia
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Universita Degli Studi Di Brescia Brescia Italy
Universitaetsklinikum Leipzig AöR Leipzig Germany
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP Frankfurt Germany
Azienda Ospedaliero Universitaria Pisana Pisa Italy
Lhpxv Uxfqscxmneph Mmvcaqx Crjbcyt (httbv Leiden The Netherlands
Hadttese Uacflnlfdphsk Mqnurdf Dy Vuglfunvxn Santander Spain
Upagynefqc Hnovxkqe Cdcpiuh Cologne Germany
Crzvmj Hpsnakrwaxs Uitsqaaxtocjx Db Dnrrq Dijon France
Ayrqtk Uufzzczsso Hlutzlfo Aarhus Denmark
Hwxluhyd De Lz Sdqsd Crvo I Sttq Pov Barcelona Spain
Aujvhud Opoejokwihn Pkhi Gialtssh Xbcnb Bergamo Italy
Mdbesrbttecqvupttimpcpprlu Hzfpwafmbcffyssa Halle (Saale) Germany
Hsnpybaw Vhfa dckaaonb Barcelona Spain
Hxpnqxxk Upjcgfupbxfjoz Spooaqgjsq &kxarsi Hfrsoxp dy Hqtlhjbgbko STRASBOURG, Alsace France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Recruiting
02.12.2024
Belgium Belgium
Recruiting
02.12.2024
Czechia Czechia
Recruiting
02.12.2024
Denmark Denmark
Recruiting
02.12.2024
France France
Recruiting
02.12.2024
Germany Germany
Recruiting
02.12.2024
Hungary Hungary
Recruiting
02.12.2024
Italy Italy
Recruiting
02.12.2024
Spain Spain
Recruiting
02.12.2024
The Netherlands The Netherlands
Recruiting
02.12.2024

Trial locations

Rapcabtagene autoleucel is a type of cell therapy where a patient’s own T cells (a type of immune cell) are modified in a laboratory. These modified cells are designed to target specific cells in the body that are involved in systemic sclerosis, a serious autoimmune condition affecting the skin and internal organs. The therapy is given as a single infusion into the bloodstream.

Rituximab is an antibody medication that works by targeting and reducing certain types of B cells (immune cells) in the body. It is commonly used to treat various autoimmune conditions and certain types of cancer. The medication is given through an infusion into the bloodstream and works by modifying the immune system’s response.

Diffuse cutaneous systemic sclerosis (dcSSc) – A rare autoimmune disease that causes hardening and tightening of the skin and connective tissues throughout the body. The condition begins with inflammation and swelling of the skin, which later leads to excessive collagen production causing skin thickening. It affects large areas of the skin, particularly on the trunk, face, and limbs. The disease typically progresses relatively quickly over the first few years after onset. Internal organs may also be affected as the condition develops. The term “diffuse” refers to the widespread nature of skin involvement, distinguishing it from other forms of systemic sclerosis.

Trial ID:
2023-510380-34-00
Protocol code:
CYTB323K12201
Trial Phase:
Therapeutic exploratory (Phase II)

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