Clinical trial • Phase I/II • Immunology

RAPCABTAGENE AUTOLEUCEL for Rheumatoid arthritis | Sjögren's syndrome

Phase I/II trial of RAPCABTAGENE AUTOLEUCEL for Rheumatoid arthritis | Sjögren's syndrome. open-label, none/not specified-controlled. 27 participants.

Overview

Trial Therapeutic Area
Immunology
Trial Disease
Rheumatoid arthritis | Sjögren's syndrome
Trial Stage
Phase I/II
Drug Modality
Cell therapy | Small molecule | Monoclonal antibody

Key dates

Initial CTIS Submission Date
10-01-2025
First CTIS Authorization Date
02-05-2025

Trial design

open-label, none/not specified-controlled Phase I/II trial in Germany, France, Spain and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
27

Eligibility

Recruits 27 Vulnerable population selected. "Signed informed consent must be obtained prior to participation in the study." No details on assent or guardian consent for minors are provided in the source documents..

Pregnancy Exclusion
Female participants who are pregnant, breastfeeding or intending to conceive during the course of the study
Vulnerable Population
Vulnerable population selected. "Signed informed consent must be obtained prior to participation in the study." No details on assent or guardian consent for minors are provided in the source documents.

Inclusion criteria

  • {"criterion_text":"-Signed informed consent must be obtained prior to participation in the study"}
  • {"criterion_text":"-Men and women aged ≥ 18 years and ≤ 75 years at screening"}
  • {"criterion_text":"-Difficult-to-treat rheumatoid arthritis: Diagnosis of rheumatoid arthritis"}
  • {"criterion_text":"-Difficult-to-treat rheumatoid arthritis: XX"}
  • {"criterion_text":"-Difficult-to-treat rheumatoid arthritis: Treatment failure of standard of care therapies"}
  • {"criterion_text":"-Difficult-to-treat rheumatoid arthritis: Signs suggestive of active, progressive disease"}
  • {"criterion_text":"-Severe refractory Sjogren's Disease: Diagnosis of Sjogren’s disease"}
  • {"criterion_text":"-Severe refractory Sjogren's Disease: XX"}
  • {"criterion_text":"-Severe refractory Sjogren's Disease: Severe systemic, refractory disease with moderate to high disease activity"}

Exclusion criteria

  • {"criterion_text":"-Body Mass Index at screening of ≤17 or ≥40 kg/m2"}
  • {"criterion_text":"-Any XX that needs immediate treatment other than XX impossible"}
  • {"criterion_text":"-Clinically significant active, opportunistic, chronic or recurrent infection"}
  • {"criterion_text":"-Sexually active males unwilling to use a condom during intercourse from the time enrollment"}
  • {"criterion_text":"-Women of childbearing potential, unless they are using a highly effective method of contraception starting from the time of enrollment"}
  • {"criterion_text":"-Female participants who are pregnant, breastfeeding or intending to conceive during the course of the study"}
  • {"criterion_text":"-Inadequate organ function during screening"}
  • {"criterion_text":"-History of lymphoproliferative disease or any known malignancy or history of malignancy"}
  • {"criterion_text":"-History of bone marrow/hematopoietic stem cell or solid organ transplantation"}
  • {"criterion_text":"-Any psychiatric condition or disability making compliance with treatment or informed consent impossible"}
  • {"criterion_text":"-Any patients requiring medications prohibited by the protocol"}

Endpoints

Primary endpoints

  • {"endpoint_text":"-Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation","definition_or_measurement_approach":"Safety assessed by vital signs, adverse events, laboratory parameters and ECG evaluation (as listed in the primary endpoint text)."}

Secondary endpoints

  • {"endpoint_text":"-Rapcabtagene autoleucel transgene concentrations by qPCR over time in peripheral blood; cellular kinetics parameters (Cmax, AUC, Tmax, T1/2, Clast, Tlast)","definition_or_measurement_approach":"Transgene concentrations measured by quantitative PCR (qPCR) in peripheral blood over time; cellular kinetics parameters include Cmax, AUC, Tmax, T1/2, Clast, Tlast as specified."}

Recruitment

Planned Sample Size
27
Recruitment Window Months
41
Consent Approach
Signed informed consent must be obtained prior to participation in the study. Adult informed consent forms are provided (documents list main ICFs). Available ICF/document languages in the submission include German, French, Spanish, Italian, Hungarian and English. No assent/guardian consent procedures for minors are provided in the source.

Geography

Total Number Of Sites
19
Total Number Of Participants
27

Germany

Earliest CTIS Part Ii Submission Date
10-04-2025
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
299
Number Of Sites
6
Number Of Participants
8

Sites

Site Name
Universitaetsmedizin Goettingen
Department Name
2007: Klinik fuer Nephrologie und Rheumatologie
Principal Investigator Name
Jan Gerd Rademacher
Principal Investigator Email
jan-gerd.rademacher@med.uni-goettingen.de
Contact Person Name
Jan Gerd Rademacher
Site Name
Universitaetsklinikum Ulm AöR
Department Name
2006:Zentrum fuer Innere Medizin,Innere Medizin III
Principal Investigator Name
Anita Viardot
Principal Investigator Email
anita.viardot@uniklinik-ulm.de
Contact Person Name
Anita Viardot
Contact Person Email
anita.viardot@uniklinik-ulm.de
Site Name
Medical Center - University Of Freiburg
Department Name
2002:Klinik f Rheumatologie u Klinische Immunologie, Dep. Innere Medizin
Principal Investigator Name
Stephanie Finzel
Principal Investigator Email
stephanie.finzel@uniklinik-freiburg.de
Contact Person Name
Stephanie Finzel
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
2001:Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie
Principal Investigator Name
David Simon
Principal Investigator Email
david.simon@charite.de
Contact Person Name
David Simon
Contact Person Email
david.simon@charite.de
Site Name
University Hospital Cologne AöR
Department Name
2004:Klinik I für Innere Medizin
Principal Investigator Name
Philipp Köhler
Principal Investigator Email
philipp.koehler@uk-koeln.de
Contact Person Name
Philipp Köhler
Contact Person Email
philipp.koehler@uk-koeln.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
2005: I. Medizinische Klinik und Poliklinik
Principal Investigator Name
Julia Weinmann-Menke
Principal Investigator Email
Julia.weinmann-menke@unimedizin-mainz.de
Contact Person Name
Julia Weinmann-Menke

France

Earliest CTIS Part Ii Submission Date
14-04-2025
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
294
Number Of Sites
9
Number Of Participants
7

Sites

Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
1005:Rhumatologie
Principal Investigator Name
Christophe RICHEZ
Principal Investigator Email
christophe.richez@chu-bordeaux.fr
Contact Person Name
Christophe RICHEZ
Site Name
Centre Hospitalier Regional Et Universitaire De Brest
Department Name
1001:Rheumatologie
Principal Investigator Name
Valerie DEVAUCHELLE-PENSEC
Principal Investigator Email
Valerie.devauchelle-pensec@chu-brest.fr
Contact Person Name
Valerie DEVAUCHELLE-PENSEC
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
1003:Medecine Interne
Principal Investigator Name
Eric HACHULLA
Principal Investigator Email
Eric.hachulla@chu-lille.fr
Contact Person Name
Eric HACHULLA
Contact Person Email
Eric.hachulla@chu-lille.fr
Site Name
Institut Gustave Roussy
Department Name
1006:Rhumatologie
Principal Investigator Name
Xavier MARIETTE
Principal Investigator Email
Xavier.mariette@aphp.fr
Contact Person Name
Xavier MARIETTE
Contact Person Email
Xavier.mariette@aphp.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
1004:Rhumatologie
Principal Investigator Name
Jacques MOREL
Principal Investigator Email
j-morel@chu-montpellier.fr
Contact Person Name
Jacques MOREL
Contact Person Email
j-morel@chu-montpellier.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
1005:Rhumatologie
Principal Investigator Name
Christophe RICHEZ
Principal Investigator Email
christophe.richez@chu-bordeaux.fr
Contact Person Name
Christophe RICHEZ
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
1005:Rhumatologie
Principal Investigator Name
Christophe RICHEZ
Principal Investigator Email
christophe.richez@chu-bordeaux.fr
Contact Person Name
Christophe RICHEZ
Site Name
Assistance Publique Hopitaux De Paris
Department Name
1006:Rhumatologie
Principal Investigator Name
Xavier MARIETTE
Principal Investigator Email
Xavier.mariette@aphp.fr
Contact Person Name
Xavier MARIETTE
Contact Person Email
Xavier.mariette@aphp.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
1004:Rhumatologie
Principal Investigator Name
Jacques MOREL
Principal Investigator Email
j-morel@chu-montpellier.fr
Contact Person Name
Jacques MOREL
Contact Person Email
j-morel@chu-montpellier.fr

Spain

Earliest CTIS Part Ii Submission Date
10-04-2025
Latest Decision Or Authorization Date
05-02-2026
Processing Time Days
301
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
Hospital Universitari Vall D Hebron
Department Name
5002: Rheumatology
Principal Investigator Name
Sara Marsal Barril
Principal Investigator Email
sara.marsal@vhir.org
Contact Person Name
Sara Marsal Barril
Contact Person Email
sara.marsal@vhir.org
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
5001: Rheumatology
Principal Investigator Name
Francisco Javier Bachiller Corral
Principal Investigator Email
fbachiller@salud.madrid.org
Contact Person Name
Francisco Javier Bachiller Corral
Contact Person Email
fbachiller@salud.madrid.org

Hungary

Earliest CTIS Part Ii Submission Date
01-04-2026
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
40
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet
Department Name
#3001: Phase I Centre
Principal Investigator Name
Eva Vegh
Principal Investigator Email
evegh@dpckorhaz.hu
Contact Person Name
Eva Vegh
Contact Person Email
evegh@dpckorhaz.hu

Italy

Earliest CTIS Part Ii Submission Date
02-04-2026
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
40
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
IRCCS Azienda Ospedaliera Metropolitana
Department Name
# 4001; U.O. Ematologia e Centro Trapianti
Principal Investigator Name
Raffaele DE PALMA
Principal Investigator Email
raffaele.depalma@unige.it
Contact Person Name
Raffaele DE PALMA
Contact Person Email
raffaele.depalma@unige.it

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
IQVIA Limited
Responsibilities
code:1
Name
Pharma Bio-Research Group
Responsibilities
Analysis of humoral IG; code:4
Name
Parexel International (IRL) Limited
Responsibilities
Ancillary supplies for leukapheresis and infusion
Name
Icon Clinical Research Limited
Responsibilities
code:1
Name
RWS Life Sciences Inc.
Responsibilities
PRO translation services
Name
Labcorp Central Laboratory Services SARL
Responsibilities
code:4
Name
Bioagilytix Labs LLC
Responsibilities
Analysis of cellular IG; code:4
Name
Navigate Biopharma Services Inc.
Responsibilities
code:4
Name
Eurofins Genomics Europe AgriGenomics Products & Services A/S
Responsibilities
Pharmacogenetics; code:4
Name
Syneos Health Inc.
Responsibilities
code:1

Third parties

  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code:1","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Pharma Bio-Research Group","duties_or_roles":"Analysis of humoral IG; code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Ancillary supplies for leukapheresis and infusion","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code:1","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code:12","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"PRO translation services","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Analysis of cellular IG; code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Navigate Biopharma Services Inc.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"Denmark","full_name":"Eurofins Genomics Europe AgriGenomics Products & Services A/S","duties_or_roles":"Pharmacogenetics; code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code:1","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
YTB323
Active Substance
RAPCABTAGENE AUTOLEUCEL
Modality
Cell therapy
Routes Of Administration
INTRAVENOUS USE
Route
INTRAVENOUS USE
Authorisation Status
1
Investigational Product Name
FLUDARABINE PHOSPHATE
Active Substance
FLUDARABINE PHOSPHATE
Modality
Small molecule
Routes Of Administration
IV INFUSION
Route
IV INFUSION
Authorisation Status
2
Investigational Product Name
CYCLOPHOSPHAMIDE
Active Substance
CYCLOPHOSPHAMIDE
Modality
Small molecule
Routes Of Administration
IV INFUSION
Route
IV INFUSION
Authorisation Status
2
Investigational Product Name
TOCILIZUMAB
Active Substance
TOCILIZUMAB
Modality
Monoclonal antibody
Routes Of Administration
IV INFUSION
Route
IV INFUSION
Authorisation Status
2
Combination Treatment
Yes

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