Clinical trial • Phase II • Immunology|Rare Disease
RAPCABTAGENE AUTOLEUCEL for Diffuse cutaneous systemic sclerosis (dcSSc)
Phase II trial of RAPCABTAGENE AUTOLEUCEL for Diffuse cutaneous systemic sclerosis (dcSSc).
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Diffuse cutaneous systemic sclerosis (dcSSc)
- Trial Stage
- Phase II
- Drug Modality
- Cell therapy|Monoclonal antibody
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 07-06-2024
- First CTIS Authorization Date
- 02-10-2024
Trial design
Randomised, open-label, rituximab (comparator). dose and schedule not specified in the provided data.-controlled Phase II trial in Czechia, Hungary, Belgium and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Rituximab (comparator). Dose and schedule not specified in the provided data.
- Target Sample Size
- 61
- Trial Duration For Participant
- 1825
Eligibility
Recruits 61 Vulnerable population selected. Informed consent is obtained from adult participants via the Main ICF (Adult). The documentation set includes language-specific ICFs and separate information/consent documents for pregnant participants, female partners, follow-up for pregnant participants, data protection consent and optional assessments. No specific assent or minor consent procedures are provided in the available documents..
- Vulnerable Population
- Vulnerable population selected. Informed consent is obtained from adult participants via the Main ICF (Adult). The documentation set includes language-specific ICFs and separate information/consent documents for pregnant participants, female partners, follow-up for pregnant participants, data protection consent and optional assessments. No specific assent or minor consent procedures are provided in the available documents.
Inclusion criteria
- {"criterion_text":"- Participant must fulfill the 2013 American College of Rheumatology/ European League Against Rheumatism classification criteria for systemic sclerosis (van den Hoogen et al 2013) and meet the diffuse cutaneous SSc (dcSSc) subset classification according to LeRoy (LeRoy et al 1988)"}
- {"criterion_text":"- 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"}
- {"criterion_text":"- 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
- {"criterion_text":"- 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"}
- {"criterion_text":"- Participants with history of hypersensitivity to excipients in rapcabtagene autoleucel or to rituximab"}
- {"criterion_text":"- Any participant for whom treatment with rituximab is clinically inappropriate in the opinion of the investigator"}
- {"criterion_text":"- 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"}
- {"criterion_text":"- Rheumatic disease other than dcSSc, (except secondary Sjogren’s syndrome or scleroderma myopathy), including limited cutaneous systemic sclerosis (lcSSc) or sine scleroderma at Screening"}
- {"criterion_text":"- Participants with pre-existing pulmonary hypertension."}
- {"criterion_text":"- Significant renal pathology at Screening, including: • Scleroderma renal crisis • Confirmed diagnosis of glomerulonephritis"}
- {"criterion_text":"- Participants with uncontrolled stage II hypertension at Screening."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Achievement of a treatment response as per the rCRISS50 definition at Week 52","definition_or_measurement_approach":"Proportion of participants achieving a Revised Composite Response Index in Systemic Sclerosis 50 (rCRISS50) response at Week 52 (as per the rCRISS50 definition)"}
Secondary endpoints
- {"endpoint_text":"- Change from baseline in FVC% predicted at Week 52","definition_or_measurement_approach":"Change from baseline in forced vital capacity percent predicted (FVC% predicted) at Week 52"}
- {"endpoint_text":"- Change from baseline in mRSS at Week 52","definition_or_measurement_approach":"Change from baseline in Modified Rodnan Skin Score (mRSS) at Week 52"}
- {"endpoint_text":"- Change from baseline in HAQ-DI at Week 52","definition_or_measurement_approach":"Change from baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 52"}
- {"endpoint_text":"- Safety evaluation includes adverse events, laboratory parameters, vital signs, ECGs, and mortality","definition_or_measurement_approach":"Safety assessed by monitoring adverse events, laboratory parameters, vital signs, ECGs, and mortality"}
Recruitment
- Planned Sample Size
- 61
- Recruitment Window Months
- 94
- Consent Approach
- Informed consent is obtained from adult participants using the Main ICF (Adult). Consent documentation is provided in multiple country languages (examples in the documents list: English, German, French, Spanish, Italian, Hungarian, Czech, Dutch, Danish). Separate ICFs/info sheets are provided for pregnant participants, female partners, follow-up procedures, molecular pre-screening, and optional assessments. No pediatric assent processes are included in the available documents.
Methods
- Site-based recruitment at participating hospitals/clinics across the listed Member States (country-specific site lists and contact points provided in Part II submissions).
- Use of recruitment arrangements and advertisement materials (documents K1_Recruitment Arrangements and K2_Advertisements are listed per country) for outreach and participant identification.
- Patient-facing materials and site training materials produced by third parties (e.g., Publicis Healthcare Communications Group Limited) to support recruitment and information.
- Patient retention support (hotel and travel reimbursement) provided by Greenphire LLC as noted in sponsor third-party duties.
Geography
Czechia
- Earliest CTIS Part Ii Submission Date
- 29-01-2025
- Latest Decision Or Authorization Date
- 02-04-2026
- Processing Time Days
- 428
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Revmatologicky Ustav
- Department Name
- #2751: Revmatologicky Ustav
- Contact Person Name
- Michal Tomcik
- Contact Person Email
- michal.tomcik@lf1.cuni.cz
- Site Name
- University Hospital Olomouc
- Department Name
- #2752: III. Interni klinika nefrologicka, revmatologicka a endokrinologicka
- Contact Person Name
- Martina Skacelova
- Contact Person Email
- martina.skacelova@fnol.cz
Hungary
- Earliest CTIS Part Ii Submission Date
- 21-02-2025
- Latest Decision Or Authorization Date
- 01-04-2026
- Processing Time Days
- 404
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- University Of Debrecen
- Department Name
- 3052: Department of Internal Medicine
- Contact Person Name
- Arpad Illes
- Contact Person Email
- llesarpaddr@gmail.com
- Site Name
- Semmelweis University
- Department Name
- 3051: Department of Rheumatology and Immunology
- Contact Person Name
- Gyorgy Nagy
- Contact Person Email
- gyorgyngy@gmail.com
Belgium
- Earliest CTIS Part Ii Submission Date
- 08-11-2024
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 507
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- #2951: Rheumatology
- Contact Person Name
- Vanessa Smith
- Contact Person Email
- Vanessa.smith@ugent.be
France
- Earliest CTIS Part Ii Submission Date
- 09-09-2024
- Latest Decision Or Authorization Date
- 03-04-2026
- Processing Time Days
- 571
- Number Of Sites
- 18
- Number Of Participants
- 8
Sites
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- 2054: Immunologie Clinique
- Contact Person Name
- Thierry MARTIN
- Contact Person Email
- Thierry.martin@chru-strasbourg.fr
Netherlands
- Earliest CTIS Part Ii Submission Date
- 05-09-2024
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 571
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- 1002: Rheumatology
- Contact Person Name
- Julia Spierings
- Contact Person Email
- J.Spierings@umcutrecht.nl
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- #1001: Hematology
- Contact Person Name
- Joost Vermaat
- Contact Person Email
- j.s.p.vermaat@lumc.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 06-09-2024
- Latest Decision Or Authorization Date
- 02-04-2026
- Processing Time Days
- 573
- Number Of Sites
- 16
- Number Of Participants
- 16
Sites
- Site Name
- Universitaetsmedizin Goettingen
- Department Name
- 2164: Klinik fuer Nephrologie und Rheumatologie
- Contact Person Name
- Viktor Korendovych
- Contact Person Email
- Viktor.korendovych@med.uni-goettingen.de
Austria
- Earliest CTIS Part Ii Submission Date
- 03-02-2025
- Latest Decision Or Authorization Date
- 31-03-2026
- Processing Time Days
- 421
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Medical University Of Graz
- Department Name
- Department of rheumatology
- Contact Person Name
- Jens Thiel
- Contact Person Email
- jens.thiel@medunigraz.at
Denmark
- Earliest CTIS Part Ii Submission Date
- 21-01-2025
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 433
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Aarhus University Hospital
- Department Name
- #3151: Led- og Bindevævssygdomme Klinik
- Contact Person Name
- Ellen-Margrethe Hauge
- Contact Person Email
- ellen.hauge@aarhus.rm.dk
Spain
- Earliest CTIS Part Ii Submission Date
- 06-09-2024
- Latest Decision Or Authorization Date
- 06-04-2026
- Processing Time Days
- 577
- Number Of Sites
- 18
- Number Of Participants
- 18
Sites
- Site Name
- Hospital Universitario Reina Sofia
- Department Name
- 2457: Reumatología
- Contact Person Name
- Alejandro Escudero Contreras
- Contact Person Email
- alejandro.escudero.sspa@juntadeandalucia.es
Italy
- Earliest CTIS Part Ii Submission Date
- 19-09-2024
- Latest Decision Or Authorization Date
- 02-04-2026
- Processing Time Days
- 560
- Number Of Sites
- 17
- Number Of Participants
- 17
Sites
- Site Name
- Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
- Department Name
- 2252 U.O.C. Reumatologia e Immunologia Clinica
- Contact Person Name
- Paola Toniati
- Contact Person Email
- paola.toniati@icloud.com
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Parexel International Corp.
- Responsibilities
- Ancillary supplies for infusion; clinical operations support
- Name
- IQVIA Limited
- Responsibilities
- Patient profiles; clinical trial information support
- Name
- Syneos Health Inc.
- Responsibilities
- Operational support / CRO-type responsibilities
Third parties
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Electronic Clinical Outcome Assessments (eCOA, ePRO, ClinRO); Medical Image Analysis/Review – CT, ECHO, x-ray, MRI, ultrasound etc; ECG Analysis/Review, Spirometry, DLCO (various entries across third party records)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient retention; hotel and travel reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"Aliquoting","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Parexel International Corp.","duties_or_roles":"Ancillary supplies for infusion","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Patient profiles; general clinical trial information support","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Clinical trial enquiries / support (role code 12 listed)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Somalogic Operating Co. Inc.","duties_or_roles":"Analysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Central laboratory services","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Navigate Biopharma Services Inc.","duties_or_roles":"PK Analysis","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Pharmacokinetics (PK) IG (Cellular) analysis; Flow sample testing","organisation_type":"Pharmaceutical company"}
- {"country":"Denmark","full_name":"Clinigma ApS","duties_or_roles":"Patient experience exit interview","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"Operational support (listed duty code 1)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient facing materials (informative videos, print material), Site training materials","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc. (additional entry)","duties_or_roles":"Medical Image Analysis/Review (CT, ECHO, x-ray, MRI, ultrasound etc)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc. (ECG/Spirometry entry)","duties_or_roles":"ECG Analysis/Review, Spirometry, DLCO","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- YTB323
- Active Substance
- RAPCABTAGENE AUTOLEUCEL
- Modality
- Cell therapy
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- prodAuthStatus:1
- Investigational Product Name
- RITUXIMAB
- Active Substance
- RITUXIMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- prodAuthStatus:2
- Orphan Designation
- Yes
- Investigational Product Name
- CYCLOPHOSPHAMIDE
- Active Substance
- CYCLOPHOSPHAMIDE
- Modality
- Small molecule
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- prodAuthStatus:2
- Investigational Product Name
- FLUDARABINE PHOSPHATE
- Active Substance
- FLUDARABINE PHOSPHATE
- Modality
- Small molecule
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- prodAuthStatus:2
- Investigational Product Name
- TOCILIZUMAB
- Active Substance
- TOCILIZUMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- prodAuthStatus:2
- Combination Treatment
- Yes
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