Clinical trial • Phase III • Immunology | Haematology | Oncology
rapcabtagene autoleucel for B-cell lymphoma | B-cell acute leukaemia | Multiple myeloma
Phase III trial of rapcabtagene autoleucel for B-cell lymphoma | B-cell acute leukaemia | Multiple myeloma. open-label. 952 participants.
Overview
- Trial Therapeutic Area
- Immunology | Haematology | Oncology
- Trial Disease
- B-cell lymphoma | B-cell acute leukaemia | Multiple myeloma
- Trial Stage
- Phase III
- Drug Modality
- Cell therapy
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 15-12-2023
- First CTIS Authorization Date
- 08-02-2024
Trial design
open-label Phase III trial across 53 sites in Germany, France, Greece and others.
- Open Label
- Yes
- Target Sample Size
- 952
- Trial Duration For Participant
- 7671
Eligibility
Recruits 952 paediatric patients.
- Vulnerable Population
- Includes vulnerable populations (minors). The protocol requires patients to provide informed consent or assent prior to entry; parental/guardian consent is required for children, with age-appropriate assent procedures. Country-specific subject information and informed consent/assent forms (for children, adolescents, parents/guardians and female partners) are provided.
Inclusion criteria
- {"criterion_text":"- Patients must have received CAR T-cell therapy within one of the following: •\tNovartis or Penn sponsored CAR T-cell treatment trials where CAR T-cell therapy was given as monotherapy or as combination therapy. •\tNovartis managed access programs (MAPs) outside of the commercial setting, i.e. where CAR T-cell therapy was intended to be given in the setting of a Novartis or Penn sponsored CAR T-cell treatment trial"}
- {"criterion_text":"- Patients must provide informed consent/assent prior to their entry into this study"}
Exclusion criteria
- {"criterion_text":"- There are no specific exclusion criteria for this study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Proportion of patients with events in each of the following categories: For participants treated for non-hematology/non-oncology indications: New primary malignancies•For participants with hematology/oncology indications: New secondary malignancies•New serious infections•New incidence of serious neurologic disorder•New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder•New incidence of a hematologic disorder•Other adverse events considered related to CAR T-cell thera","definition_or_measurement_approach":"Measured as the proportion of patients experiencing events in each listed category (new secondary malignancies, new serious infections, new serious neurologic disorders, new/exacerbated autoimmune disorders, new hematologic disorders, and other AEs considered related to prior CAR T-cell therapy)."}
Secondary endpoints
- {"endpoint_text":"- Proportion of patients with detectable CAR transgene levels in peripheral blood by qPCR at pre-specified time points","definition_or_measurement_approach":"Measured by quantitative PCR (qPCR) for CAR transgene in peripheral blood at pre-specified time points; reported as proportion of patients with detectable transgene."}
- {"endpoint_text":"- Proportion of patients with detectable RCL by VSV-G qPCR in peripheral blood at pre-specified time points","definition_or_measurement_approach":"Measured by VSV-G targeted qPCR to detect replication competent lentivirus (RCL) in peripheral blood at pre-specified time points; reported as proportion of patients with detectable RCL."}
- {"endpoint_text":"- •\tProportion of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry •\tIncidence of death Note: For non-hematology/non-oncology patients, though there will be no formal assessments for efficacy, data on disease indication management will be captured in the Concomitant Medication CRF (e.g., rescue medication including immunosuppressive medications).","definition_or_measurement_approach":"Proportion of patients with relapse or progression among those without relapse/progression at entry; incidence of death recorded. For non-hematology/non-oncology patients, disease management data captured in Concomitant Medication CRF."}
- {"endpoint_text":"- B and T lymphocyte counts","definition_or_measurement_approach":"Measured laboratory counts of B- and T-lymphocytes (timing/assay per protocol)."}
- {"endpoint_text":"- Height and weight, Tanner staging, menstruation status The pregnancy status for female patients will be captured in the CRF. For female partners of male patients if female partners provide consent, pregnancy outcome and data on live births for infants at birth, 3 months and 1 year of age is captured in Novartis safety forms.","definition_or_measurement_approach":"Anthropometric measures (height, weight), Tanner staging and menstrual status recorded; pregnancy status captured in CRF; pregnancy outcomes and live-birth data for consenting female partners captured in sponsor safety forms at birth, 3 months and 1 year."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 952
- Recruitment Window Months
- 252
- Consent Approach
- Informed consent (and assent where applicable) is required prior to study entry. Adults provide informed consent; for minors parental/guardian consent and age-appropriate assent forms are used (multiple ICF/assent templates exist for children and adolescents in age bands). Female partners (of male patients) have separate information/consent forms for pregnancy outcome data. Country-specific documents and translations are provided (languages and country-specific ICFs available for English, French, German, Spanish, Italian, Dutch, Swedish, Danish, Finnish, Greek, Norwegian and others). Remote/remote-visit consent and parent/guardian remote consent options are included in country-specific materials.
Methods
- Recruitment of patients previously treated with Novartis- or Penn-sponsored CAR T-cell treatment trials or Novartis managed access programs (MAPs) (i.e., identifying eligible patients from prior CAR-T trials and MAPs).
- Site-based recruitment at participating hospital/clinic centres listed in the CTIS Part II submissions (country-specific site lists).
Geography
- Total Number Of Sites
- 53
- Total Number Of Participants
- 222
Germany
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 13-03-2026
- Processing Time Days
- 792
- Number Of Sites
- 10
- Number Of Participants
- 17
Sites
- Site Name
- Universitaetsklinikum Wuerzburg AöR
- Department Name
- Oncology
- Contact Person Name
- Volker Kunzmann
- Contact Person Email
- kunzmann_v@ukw.de
- Site Name
- Medical Center - University Of Freiburg
- Department Name
- Rheumatology and Clinical Immunology
- Contact Person Name
- Stephanie Finzel
- Contact Person Email
- Stephanie.Finzel@uniklinik-freiburg.de
- Site Name
- Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
- Department Name
- Nephrology
- Contact Person Name
- Julia Weinmann-Menke
- Contact Person Email
- julia.weinmann-menke@unimedizin-mainz.de
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- Oncology
- Contact Person Name
- Francis Ayuk
- Contact Person Email
- ayuketang@uke.de
- Site Name
- University Hospital Cologne AöR
- Department Name
- Oncology
- Contact Person Name
- Peter Borchmann
- Contact Person Email
- peter.borchmann@uk-koeln.de
- Site Name
- Universitaetsklinikum Ulm AöR
- Department Name
- Oncology
- Contact Person Name
- Andreas Viardot
- Contact Person Email
- andreas.viardot@uniklinik-ulm.de
- Site Name
- Klinikum der Universitaet Muenchen AöR
- Department Name
- Oncology
- Contact Person Name
- Martin Dreyling
- Contact Person Email
- martin.dreyling@med.uni-muenchen.de
- Site Name
- Universitaetsklinikum Frankfurt AöR
- Department Name
- Oncology
- Contact Person Name
- Peter Bader
- Contact Person Email
- peter.bader@kgu.de
- Site Name
- Universitaetsklinikum Leipzig AöR
- Department Name
- Oncology
- Contact Person Name
- Vladan Vucinic
- Contact Person Email
- vladan.vucinic@medizin.uni-leipzig.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Oncology
- Contact Person Name
- Martin Janz
- Contact Person Email
- martin.janz@charite.de
France
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 09-03-2026
- Processing Time Days
- 788
- Number Of Sites
- 8
- Number Of Participants
- 55
Sites
- Site Name
- Hopital Saint Louis
- Department Name
- Hematology - Oncology
- Contact Person Name
- Catherine Thieblemont
- Contact Person Email
- catherine.thieblemont@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Hematology
- Contact Person Name
- Emmanuel Bachy
- Contact Person Email
- emmanuel.bachy@chu-lyon.fr
- Site Name
- Hopital Huriez
- Department Name
- Service des Maladies du Sang
- Contact Person Name
- Ibrahim Yakoub-Agha
- Contact Person Email
- Ibrahim.Yakoub-AGHA@chu-lille.fr
- Site Name
- Hopital Saint Louis
- Department Name
- Immuno-Hematology Department
- Contact Person Name
- Bertrand Arnulf
- Contact Person Email
- bertrand.arnulf@aphp.fr
- Site Name
- Robert Debre University Hospital
- Department Name
- Hematology
- Contact Person Name
- André Baruchel
- Contact Person Email
- andre.baruchel@rdb.aphp.fr
- Site Name
- Hopital Saint Louis
- Department Name
- Hematology
- Contact Person Name
- Nicolas Boissel
- Contact Person Email
- nicolas.boissel@aphp.fr
- Site Name
- Institut Gustave Roussy
- Department Name
- Department of Children and Adolescents Oncology
- Contact Person Name
- Charlotte Rigaud
- Contact Person Email
- charlotte.rigaud@gustaveroussy.fr
- Site Name
- Hospital Hotel Dieu
- Department Name
- Service d'Hematologie Clinique
- Contact Person Name
- Thomas Gastinne
- Contact Person Email
- thomas.gastinne@chu-nantes.fr
Greece
- Earliest CTIS Part Ii Submission Date
- 10-02-2025
- Latest Decision Or Authorization Date
- 11-03-2026
- Processing Time Days
- 394
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Evangelismos S.A.
- Department Name
- Hematology and Lymphoma Clinic - Bone Marrow Transplant Unit
- Contact Person Name
- Ioannis Baltadakis
- Contact Person Email
- ibaltadakis@icloud.com
Netherlands
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 13-04-2026
- Processing Time Days
- 823
- Number Of Sites
- 3
- Number Of Participants
- 15
Sites
- Site Name
- Utrecht University
- Department Name
- Oncology
- Contact Person Name
- Monique Minnema
- Contact Person Email
- m.c.minnema@umcutrecht.nl
- Site Name
- Prinses Maxima Centrum voor Kinderoncologie B.V.
- Department Name
- Oncology
- Contact Person Name
- Michel Zwaan
- Contact Person Email
- c.m.zwaan@prinsesmaximacentrum.nl
- Site Name
- Academisch Medisch Centrum
- Department Name
- Oncology
- Contact Person Name
- Marie Jose Kersten
- Contact Person Email
- m.j.kersten@amsterdamumc.nl
Denmark
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 09-03-2026
- Processing Time Days
- 788
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Rigshospitalet
- Department Name
- Oncology Department
- Contact Person Name
- Marianne Ifversen
- Contact Person Email
- ifversen@rh.dk
Sweden
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 10-03-2026
- Processing Time Days
- 790
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- Karolinska Institutet
- Department Name
- Oncology
- Contact Person Name
- Stephan Mielke
- Contact Person Email
- stephan.mielke@ki.se
- Site Name
- Sahlgrenska University Hospital-Vastra Gotalandsregionen
- Department Name
- Oncology
- Contact Person Name
- Karin Mellgren
- Contact Person Email
- karin.mellgren@vgregion.se
Norway
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 11-03-2026
- Processing Time Days
- 790
- Number Of Sites
- 2
- Number Of Participants
- 16
Sites
- Site Name
- Oslo University Hospital HF
- Department Name
- Oncology
- Contact Person Name
- Jochen Buchner
- Contact Person Email
- jocbuc@ous-hf.no
- Site Name
- Oslo University Hospital HF
- Department Name
- Oncology
- Contact Person Name
- Marianne Brodtkorb
- Contact Person Email
- meide@ous-hf.no
Finland
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 09-04-2026
- Processing Time Days
- 819
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- HUS-yhtymae
- Department Name
- Oncology
- Contact Person Name
- Samppa Ryhänen
- Contact Person Email
- samppa.ryhanen@hus.fi
Spain
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 13-03-2026
- Processing Time Days
- 792
- Number Of Sites
- 13
- Number Of Participants
- 51
Sites
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- Oncology
- Contact Person Name
- Joaquin Martinez Lopez
- Contact Person Email
- j.martinez@salud.madrid.org
- Site Name
- Hospital Clinico Universitario De Valencia
- Department Name
- Oncology
- Contact Person Name
- Carlos Solano Vercet
- Contact Person Email
- carlos.solano@uv.es
- Site Name
- Clinica Universidad De Navarra
- Department Name
- Hematology
- Contact Person Name
- Paula Rodríguez Otero
- Contact Person Email
- paurodriguez@unav.es
- Site Name
- Hospital Universitario La Paz
- Department Name
- Hematology and Hemotherapy
- Contact Person Name
- Berta Gonzalez Martinez
- Contact Person Email
- bertagonzalezm@gmail.com
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- Department of Rheumatology
- Contact Person Name
- José María Álvaro-Gracia
- Contact Person Email
- jalvarogracia@gmail.com
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Hematology
- Contact Person Name
- Gloria Inés Iacoboni García-Calvo
- Contact Person Email
- giacoboni@vhio.net
- Site Name
- Sant Joan De Deu Barcelona Hospital
- Department Name
- Oncology
- Contact Person Name
- Susana Rives Sola
- Contact Person Email
- srives@hsjdbcn.org
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- Oncology
- Contact Person Name
- Mi Kwon
- Contact Person Email
- mi.kwon@salud.madrid.org
- Site Name
- Hospital De La Santa Creu I Sant Pau
- Department Name
- Oncology
- Contact Person Name
- Javier Briones Meijide
- Contact Person Email
- jbriones@santpau.cat
- Site Name
- Institut Catala D'oncologia
- Department Name
- Oncology
- Contact Person Name
- Anna Sureda Balari
- Contact Person Email
- asureda@iconcologia.net
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Oncology
- Contact Person Name
- Jose Antonio Perez Simon
- Contact Person Email
- josea.perez.simon.sspa@juntadeandalucia.es
- Site Name
- Hospital Universitario De Salamanca
- Department Name
- Hematologia
- Contact Person Name
- Alejandro Martin Garcia-Sancho
- Contact Person Email
- amartingar@usal.es
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Department of Rheumatology
- Contact Person Name
- Josefina Cortes Hernández
- Contact Person Email
- fina.cortes@vhir.org
Italy
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 830
- Number Of Sites
- 7
- Number Of Participants
- 33
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- UOC Ematologia e Trapianti di CSE
- Contact Person Name
- Simona Sica
- Contact Person Email
- simona.sica@unicatt.it
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- U.O. di Ematologia e Trapianto di midollo osseo
- Contact Person Name
- Fabio Ciceri
- Contact Person Email
- ciceri.clinicaltrials@hsr.it
- Site Name
- Humanitas Mirasole S.p.A.
- Department Name
- UO di Oncologia Medica ed Ematologia
- Contact Person Name
- Armando Santoro
- Contact Person Email
- armando.santoro@cancercenter.humanitas.it
- Site Name
- Fondazione IRCCS Istituto Nazionale Dei Tumori
- Department Name
- Oncology
- Contact Person Name
- Paolo Corradini
- Contact Person Email
- paolo.corradini@istitutotumori.mi.it
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori
- Department Name
- Pediatric Bone Marrow Transplantation unit
- Contact Person Name
- Adriana Cristina Balduzzi
- Contact Person Email
- abalduzzi@fondazionembbm.it
- Site Name
- Ospedale Pediatrico Bambino Gesu'
- Department Name
- Oncology
- Contact Person Name
- Franco Locatelli
- Contact Person Email
- franco.locatelli@opbg.net
- Site Name
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
- Department Name
- Oncology
- Contact Person Name
- Pier Luigi Zinzani
- Contact Person Email
- pierluigi.zinzani@unibo.it
Austria
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 13-03-2026
- Processing Time Days
- 792
- Number Of Sites
- 3
- Number Of Participants
- 16
Sites
- Site Name
- St. Anna Kinderspital GmbH
- Department Name
- Division of Haematology and Oncology
- Contact Person Name
- Christina Peters
- Contact Person Email
- christina.peters@stanna.at
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Internal Medicine I, Division of Haematology and Haemostaseology
- Contact Person Name
- Ulrich Jaeger
- Contact Person Email
- ulrich.jaeger@meduniwien.ac.at
- Site Name
- Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH
- Department Name
- Department of Hematology, Medical Oncology, Hemostaseology, Infectious Disease and Rheumatology
- Contact Person Name
- Michael Leisch
- Contact Person Email
- m.leisch@salk.at
Belgium
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 10-03-2026
- Processing Time Days
- 790
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Oncology
- Contact Person Name
- Barbara De Moerloose
- Contact Person Email
- barbara.demoerloose@uzgent.be
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Oncology
- Contact Person Name
- Fritz Offner
- Contact Person Email
- fritz.offner@uzgent.be
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma Services AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- IQVIA Limited
- Responsibilities
- Ethic Committee notification/submission; Site management; Remote Monitoring; Investigator Payments; Decentralized Clinical Trials; Vendor management; Trial Master File
- Name
- Parexel International (IRL) Limited
- Responsibilities
- Regulatory/clinical trial services
- Name
- Pharmaceutical Research Associates Group B.V.
- Responsibilities
- Humoral IG (serum) analysis
- Name
- Perceptive Eclinical Limited
- Responsibilities
- IRT management of patient visits and enrollment
- Name
- Navigate Biopharma Services Inc.
- Responsibilities
- Biomarker sample analysis and related laboratory coordination
Third parties
- {"country":"United States","full_name":"Navigate Biopharma Services Inc.","duties_or_roles":"Biomarker samples analysis, Tumor Biopsy, Peripheral Blood, PBMC PK, Peripheral blood, bone marrow","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Ethic Committee notification/submission; Site management; Remote Monitoring; Investigator Payments; Decentralized Clinical Trials; Vendor management; Trial Master File","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Perceptive Eclinical Limited","duties_or_roles":"IRT will be used to manage patient visits and enrollment numbers; other data services","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Merative US LP","duties_or_roles":"Data services (site/system support)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"Specimen collection/management/repository/shipment; Lab kits/logistics; B cell flow cytometry","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"Humoral IG (serum) analysis","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Regulatory/clinical trial services (code 12 indicated)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Cellular immunogenicity, PBMC","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Reimbursement","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- YTB323
- Active Substance
- rapcabtagene autoleucel
- Modality
- Cell therapy
- Routes Of Administration
- INTRAVENOUS INFUSION
- Route
- INTRAVENOUS INFUSION
- Authorisation Status
- 1
- Investigational Product Name
- TISAGENLECLEUCEL
- Active Substance
- tisagenlecleucel
- Modality
- Cell therapy
- Routes Of Administration
- INTRAVENIOUS INFUSION
- Route
- INTRAVENIOUS INFUSION
- Authorisation Status
- 2
- Orphan Designation
- Yes
- Investigational Product Name
- PHE885
- Active Substance
- autologous t-cells encoding a chimeric antigen receptor targeting human b cell maturation antigen
- Modality
- Cell therapy
- Routes Of Administration
- INTRAVENOUS INFUSION
- Route
- INTRAVENOUS INFUSION
- Authorisation Status
- 1
Related trials
Other published trials that may interest you.