Clinical trial • Phase II • Immunology|Rare Disease
RAPCABTAGENE AUTOLEUCEL for Idiopathic inflammatory myopathies (severe refractory) | Immune-mediated myositis
Phase II trial of RAPCABTAGENE AUTOLEUCEL for Idiopathic inflammatory myopathies (severe refractory) | Immune-mediated myositis.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Idiopathic inflammatory myopathies (severe refractory) | Immune-mediated myositis
- Trial Stage
- Phase II
- Drug Modality
- Cell therapy|Small molecule|Monoclonal antibody
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 29-08-2024
- First CTIS Authorization Date
- 20-12-2024
Trial design
Randomised, open-label, investigator's choice of treatment (comparator arm). comparator products listed in part i include: mycophenolate mofetil (oral, hard capsules), cyclophosphamide (powder for solution for injection/infusion, iv), mycophenolic acid (gastro-resistant tablet, oral), tacrolimus (capsule, oral), rituximab (concentrate for infusion, iv). dose and schedule details are not specified in the provided ctis json.-controlled Phase II trial in Germany, Spain, France and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Investigator's choice of treatment (comparator arm). Comparator products listed in Part I include: MYCOPHENOLATE MOFETIL (oral, hard capsules), CYCLOPHOSPHAMIDE (powder for solution for injection/infusion, IV), MYCOPHENOLIC ACID (gastro-resistant tablet, oral), TACROLIMUS (capsule, oral), RITUXIMAB (concentrate for infusion, IV). Dose and schedule details are not specified in the provided CTIS JSON.
- Target Sample Size
- 45
- Trial Duration For Participant
- 364
Eligibility
Recruits 45 The application indicates isVulnerablePopulationSelected = true. Signed informed consent must be obtained prior to participation in the study. Subject information and informed consent forms (L1_ICF) and procedure documents (L2_ICF) are provided for adults in multiple languages (see documents: German, English, Spanish, French, Italian, Dutch). There are also specific follow-up ICFs for pregnant participants and for pregnant partners/parent legal guardian information sheets, indicating additional procedures for pregnant participants or partners; consent is required from the participant (or legal guardian where applicable) per the provided ICFs..
- Vulnerable Population
- The application indicates isVulnerablePopulationSelected = true. Signed informed consent must be obtained prior to participation in the study. Subject information and informed consent forms (L1_ICF) and procedure documents (L2_ICF) are provided for adults in multiple languages (see documents: German, English, Spanish, French, Italian, Dutch). There are also specific follow-up ICFs for pregnant participants and for pregnant partners/parent legal guardian information sheets, indicating additional procedures for pregnant participants or partners; consent is required from the participant (or legal guardian where applicable) per the provided ICFs.
Inclusion criteria
- {"criterion_text":"- Signed informed consent must be obtained prior to participation in the study"}
- {"criterion_text":"- Male or female participants xx on the day of signing informed consent."}
- {"criterion_text":"- A diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria."}
- {"criterion_text":"- Participant must be xx based on an historical result xx"}
- {"criterion_text":"- Participants must have refractory xx:a. xx b. xx i. xx ii.xx iii. Xx iv. xx xx"}
- {"criterion_text":"- Diagnosed with active disease such as presence of at least 1 of the following criteria and confirmed by an adjudication committee for criteria b through e prior to randomization: xx"}
- {"criterion_text":"- (any additional inclusion lines present as 'xx' placeholders in the source)"}
Exclusion criteria
- {"criterion_text":"- Have severe muscle damage at Screening, as defined xx"}
- {"criterion_text":"- Participants treated with RTX xx prior to Screening or CYC xx prior to Baseline."}
- {"criterion_text":"- Inadequate organ function (one retest during Screening of any of the below is permitted): Inadequate renal function (performed by central laboratory) defined as: • eGFR < 45 ml/min/1.73m2 using the 2021 CKD-EPI formula. Inadequate hepatic function defined as any of the following: • ALT and AST >1.5 × ULN (performed by central laboratory), except if clinically assessed as secondary to IIM • Total Bilirubin >1.5 × ULN (performed by central laboratory). Participants with Gilbert's syndrome may be included if their total bilirubin is ≤ 3.0 × ULN and direct bilirubin ≤1.5 × ULN. • International normalized ratio (INR) >1.5 (performed by central laboratory) Inadequate cardiac function defined as: • Left ventricular ejection fraction (LVEF) <50% as determined by echocardiogram (ECHO) Inadequate hematologic function (performed by central laboratory) defined as: • Absolute neutrophil count (ANC) <1500/mm3 • Platelets <100,000/μL • White blood cells count (WBC) <3,000 cells/μL • Absolute lymphocyte count <700/μL • Hemoglobin < 8.0 g/dL (< 4.9 mmol/L) Inadequate pulmonary function defined by ANY of the following: • Oxygen saturation measured by pulse xx • Hemoglobin corrected DLCO xx • FVC% xx"}
- {"criterion_text":"- Any participant who failed all available treatment options in the comparator arm or for whom these options are clinically inappropriate in the opinion of the Investigator."}
- {"criterion_text":"- Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol (e.g., rapcabtagene autoleucel, comparator arm treatments, tocilizumab, xx, etc.), to the excipients of rapcabtagene autoleucel (e.g., xx), or to any other drug product as advised for administration in the study protocol (e.g., xx, G-CSF , tocilizumab)."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Achieving moderate to major improvement in TIS (TIS ≥ 40) at Week 52 (Yes/No)","definition_or_measurement_approach":"Total Improvement Score (TIS); moderate to major improvement defined as TIS ≥ 40 at Week 52 (binary Yes/No)"}
Secondary endpoints
- {"endpoint_text":"- Achieving moderate to major improvement in TIS (≥ 40) at Week 52 (Yes/No)","definition_or_measurement_approach":"Total Improvement Score (TIS); improvement defined as TIS ≥ 40 at Week 52 (binary Yes/No)"}
- {"endpoint_text":"- Adjusted annual cumulative GC dose up to Week 52","definition_or_measurement_approach":"Cumulative glucocorticoid (GC) dose from baseline to Week 52, adjusted annualised dose (measurement of cumulative GC exposure)"}
- {"endpoint_text":"- Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52","definition_or_measurement_approach":"Change from baseline in percent predicted Forced Vital Capacity (FVC%) measured at Week 52"}
- {"endpoint_text":"- Achieving major improvement in TIS (≥ 60) at Week 52 (Yes/No)","definition_or_measurement_approach":"Total Improvement Score (TIS); major improvement defined as TIS ≥ 60 at Week 52 (binary Yes/No)"}
- {"endpoint_text":"- Change from baseline in Patient-Reported Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52","definition_or_measurement_approach":"Change from baseline in PROMIS-Fatigue 7a score at Week 52 (patient-reported outcome measure)"}
- {"endpoint_text":"- Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation","definition_or_measurement_approach":"Safety assessed by monitoring vital signs, recording adverse events, laboratory parameter results and ECG evaluations"}
Recruitment
- Planned Sample Size
- 45
- Recruitment Window Months
- 94
- Consent Approach
- Signed informed consent must be obtained prior to participation. Subject information and consent documents (L1_ICF, L2_ICF and additional info sheets) are available in multiple languages (documents present for German, English, Spanish, French, Italian, Dutch). Specific ICFs and follow-up sheets are provided for pregnant participants, pregnant partners and for female partners, and there are addenda/optional ICFs and biomarker information sheets as indicated in the uploaded documents.
Geography
- Total Number Of Sites
- 37
- Total Number Of Participants
- 45
Germany
- Earliest CTIS Part Ii Submission Date
- 19-11-2024
- Latest Decision Or Authorization Date
- 17-04-2026
- Processing Time Days
- 514
- Number Of Sites
- 10
- Number Of Participants
- 15
Sites
- Site Name
- Universitaetsklinikum Jena KöR
- Department Name
- #3106: Klinik für Innere Medizin III, Rheumatologie/Osteologie
- Principal Investigator Name
- Alexander Pfeil
- Principal Investigator Email
- Alexander.Pfeil@med.uni-jena.de
- Contact Person Name
- Alexander Pfeil
- Contact Person Email
- Alexander.Pfeil@med.uni-jena.de
- Site Name
- Fraunhofer Institute For Translational Medicine And Pharmacology ITMP
- Department Name
- #3104: Fraunhofer Institute For Translational Medicine And Pharmacology ITMP
- Principal Investigator Name
- Michaela Koehm
- Principal Investigator Email
- michaela.koehm@itmp.fraunhofer.de
- Contact Person Name
- Michaela Koehm
- Contact Person Email
- michaela.koehm@itmp.fraunhofer.de
- Site Name
- Universitaetsklinikum Aachen AöR
- Department Name
- #3101: Medizinische Klinik II, Sektion Rheumatologie
- Principal Investigator Name
- Thomas Rauen
- Principal Investigator Email
- trauen@ukaachen.de
- Contact Person Name
- Thomas Rauen
- Contact Person Email
- trauen@ukaachen.de
- Site Name
- Universitaetsklinikum Leipzig AöR
- Department Name
- #3108: Klinik und Poliklinik für Endokrinologie, Nephrologie, Rheumatolgie
- Principal Investigator Name
- Ulf Wagner
- Principal Investigator Email
- Ulf.Wagner@medizin.uni-leipzig.de
- Contact Person Name
- Ulf Wagner
- Contact Person Email
- Ulf.Wagner@medizin.uni-leipzig.de
- Site Name
- University Hospital Cologne AöR
- Department Name
- #3107: 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
- Universitaetsklinikum Ulm AöR
- Department Name
- #3112: 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
- Klinikum Nuernberg
- Department Name
- #3111: Klinikum für Innere Medizin 5 Schwerpunkt Onkologie / Hämatologie
- Principal Investigator Name
- Axel Hueber
- Principal Investigator Email
- Axel.Hueber@klinikum-nuernberg.de
- Contact Person Name
- Axel Hueber
- Contact Person Email
- Axel.Hueber@klinikum-nuernberg.de
- Site Name
- Universitaetsklinikum Schleswig-Holstein AöR
- Department Name
- 3113: Klinik für Rheumatologie
- Principal Investigator Name
- Gabriela Riemekasten
- Principal Investigator Email
- gabriela.riemekasten@uksh.de
- Contact Person Name
- Gabriela Riemekasten
- Contact Person Email
- gabriela.riemekasten@uksh.de
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- #3105: III. Medizinische Klinik und Poliklinik Nephrologie, Rheumatologie und Endokrinologie
- Principal Investigator Name
- Martin Krusche
- Principal Investigator Email
- m.krusche@uke.de
- Contact Person Name
- Martin Krusche
- Contact Person Email
- m.krusche@uke.de
- Site Name
- Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
- Department Name
- #3109: 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
- Contact Person Email
- Julia.weinmann-menke@unimedizin-mainz.de
Spain
- Earliest CTIS Part Ii Submission Date
- 22-11-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 514
- Number Of Sites
- 11
- Number Of Participants
- 12
Sites
- Site Name
- Clinica Universidad De Navarra
- Department Name
- #3410:Nefrología
- Principal Investigator Name
- Jose Maria Mora Gutierrez
- Principal Investigator Email
- jmora@unav.es
- Contact Person Name
- Jose Maria Mora Gutierrez
- Contact Person Email
- jmora@unav.es
- Site Name
- Hospital Universitario Reina Sofia
- Department Name
- #3407:Reumatología
- Principal Investigator Name
- Alejandro Escudero Contreras
- Principal Investigator Email
- alejandro.escudero.sspa@juntadeandalucia.es
- Contact Person Name
- Alejandro Escudero Contreras
- Contact Person Email
- alejandro.escudero.sspa@juntadeandalucia.es
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- #3403:Reumatología
- Principal Investigator Name
- Eva María Pérez Pampín
- Principal Investigator Email
- eva.perez.pampin@sergas.es
- Contact Person Name
- Eva María Pérez Pampín
- Contact Person Email
- eva.perez.pampin@sergas.es
- Site Name
- Hospital Universitario Marques De Valdecilla
- Department Name
- #3406:Reumatología
- Principal Investigator Name
- Ricardo Blanco Alonso
- Principal Investigator Email
- ricardo.blanco@scsalud.es
- Contact Person Name
- Ricardo Blanco Alonso
- Contact Person Email
- ricardo.blanco@scsalud.es
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- #3402:Medicina Interna
- Principal Investigator Name
- Albert Selva O’Callaghan
- Principal Investigator Email
- albert.selva@vallhebron.cat
- Contact Person Name
- Albert Selva O’Callaghan
- Contact Person Email
- albert.selva@vallhebron.cat
- Site Name
- Hospital Universitario De Salamanca
- Department Name
- #3408:Reumatología
- Principal Investigator Name
- Susana Gómez Castro
- Principal Investigator Email
- sgomezc@saludcastillayleon.es
- Contact Person Name
- Susana Gómez Castro
- Contact Person Email
- sgomezc@saludcastillayleon.es
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- #3404:Reumatología
- Principal Investigator Name
- José María Álvaro Gracia
- Principal Investigator Email
- josemaria.alvarogracia@salud.madrid.org
- Contact Person Name
- José María Álvaro Gracia
- Contact Person Email
- josemaria.alvarogracia@salud.madrid.org
- Site Name
- Bellvitge University Hospital
- Department Name
- #3401:Reumatología
- Principal Investigator Name
- Francisco Javier Narváez García
- Principal Investigator Email
- fjnarvaez@bellvitgehospital.cat
- Contact Person Name
- Francisco Javier Narváez García
- Contact Person Email
- fjnarvaez@bellvitgehospital.cat
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- #3405:Reumatología
- Principal Investigator Name
- Patricia Esmeralda Carreira Delgado
- Principal Investigator Email
- carreira@h12o.es
- Contact Person Name
- Patricia Esmeralda Carreira Delgado
- Contact Person Email
- carreira@h12o.es
- Site Name
- Clinica Universidad De Navarra (Madrid site)
- Department Name
- #3410:Nefrología
- Principal Investigator Name
- Jose Maria Mora Gutierrez
- Principal Investigator Email
- jmora@unav.es
- Contact Person Name
- Jose Maria Mora Gutierrez
- Contact Person Email
- jmora@unav.es
- Site Name
- Hospital Universitario Regional De Malaga
- Department Name
- #3409:Reumatología
- Principal Investigator Name
- Natalia Mena Vázquez
- Principal Investigator Email
- natalia.mena.sspa@juntadeandalucia.es
- Contact Person Name
- Natalia Mena Vázquez
- Contact Person Email
- natalia.mena.sspa@juntadeandalucia.es
France
- Earliest CTIS Part Ii Submission Date
- 06-12-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 500
- Number Of Sites
- 7
- Number Of Participants
- 9
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- #3002: Medecine Interne
- Principal Investigator Name
- Eric HACHULLA
- Principal Investigator Email
- Eric.hachulla@chru-lille.fr
- Contact Person Name
- Eric HACHULLA
- Contact Person Email
- Eric.hachulla@chru-lille.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- #3009: Medecine Interne
- Principal Investigator Name
- Nicolas SCHLEINITZ
- Principal Investigator Email
- Nicolas.SCHLEINITZ@ap-hm.fr
- Contact Person Name
- Nicolas SCHLEINITZ
- Contact Person Email
- Nicolas.SCHLEINITZ@ap-hm.fr
- Site Name
- Centre Hospitalier Universitaire De Rennes
- Department Name
- #3005: Medecine Interne
- Principal Investigator Name
- Eric MENSI
- Principal Investigator Email
- Eric.MENSI@chu-rennes.fr
- Contact Person Name
- Eric MENSI
- Contact Person Email
- Eric.MENSI@chu-rennes.fr
- Site Name
- Centre Hospitalier Regional Et Universitaire De Brest
- Department Name
- #3008: Rhumatologie
- Principal Investigator Name
- Dewi GUELLEC
- Principal Investigator Email
- Dewi.guellec@chu-brest.fr
- Contact Person Name
- Dewi GUELLEC
- Contact Person Email
- Dewi.guellec@chu-brest.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #3001: Medecine Interne et Immunologie clinique
- Principal Investigator Name
- Yves ALLENBACH
- Principal Investigator Email
- yves.allenbach@aphp.fr
- Contact Person Name
- Yves ALLENBACH
- Contact Person Email
- yves.allenbach@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- #3007: Medecine Interne
- Principal Investigator Name
- Laure GALLAY
- Principal Investigator Email
- laure.gallay01@chu-lyon.fr
- Contact Person Name
- Laure GALLAY
- Contact Person Email
- laure.gallay01@chu-lyon.fr
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- #3003: Rhumatologie
- Principal Investigator Name
- Alain MEYER
- Principal Investigator Email
- Alain.meyer@chru-strasbourg.fr
- Contact Person Name
- Alain MEYER
- Contact Person Email
- Alain.meyer@chru-strasbourg.fr
Netherlands
- Earliest CTIS Part Ii Submission Date
- 09-12-2024
- Latest Decision Or Authorization Date
- 16-04-2026
- Processing Time Days
- 493
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- #3302:Internal Medicine
- Principal Investigator Name
- Zana Brkic
- Principal Investigator Email
- z.brkic@erasmusmc.nl
- Contact Person Name
- Zana Brkic
- Contact Person Email
- z.brkic@erasmusmc.nl
- Site Name
- Amsterdam UMC Stichting
- Department Name
- #3301: Neurology
- Principal Investigator Name
- Joost Raaphorst
- Principal Investigator Email
- j.raaphorst@amsterdamumc.nl
- Contact Person Name
- Joost Raaphorst
- Contact Person Email
- j.raaphorst@amsterdamumc.nl
Italy
- Earliest CTIS Part Ii Submission Date
- 19-11-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 517
- Number Of Sites
- 7
- Number Of Participants
- 7
Sites
- Site Name
- Azienda Ospedaliero Universitaria Delle Marche
- Department Name
- 3207:S.O.D. Clinica Medica
- Principal Investigator Name
- Gianluca Moroncini
- Principal Investigator Email
- g.moroncini@staff.univpm.it
- Contact Person Name
- Gianluca Moroncini
- Contact Person Email
- g.moroncini@staff.univpm.it
- Site Name
- Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
- Department Name
- #3201: U.O.C. Reumatologia e Immunologia Clinica
- Principal Investigator Name
- Paola Toniati
- Principal Investigator Email
- palola1@virgilio.it
- Contact Person Name
- Paola Toniati
- Contact Person Email
- palola1@virgilio.it
- Site Name
- Azienda Ospedaliera Universitaria Integrata Verona
- Department Name
- #3202: U.O.C. di Reumatologia
- Principal Investigator Name
- Giovanni Orsolini
- Principal Investigator Email
- giovanniorsolini@gmail.com
- Contact Person Name
- Giovanni Orsolini
- Contact Person Email
- giovanniorsolini@gmail.com
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- #3204: Medicina Generale - Immunologia e Allergologia
- Principal Investigator Name
- Nicola Montano
- Principal Investigator Email
- nicola.montano@policlinico.mi.it
- Contact Person Name
- Nicola Montano
- Contact Person Email
- nicola.montano@policlinico.mi.it
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori
- Department Name
- #3203: SSD Reumatologia
- Principal Investigator Name
- Carlo Alberto Scirè
- Principal Investigator Email
- carlo.scire@unimib.it
- Contact Person Name
- Carlo Alberto Scirè
- Contact Person Email
- carlo.scire@unimib.it
- Site Name
- ASST Grande Ospedale Metropolitano Niguarda
- Department Name
- 3206: Epis S.C. Reumatologia
- Principal Investigator Name
- Oscar Massimiliano Epis
- Principal Investigator Email
- oscar.epis@ospedaleniguarda.it
- Contact Person Name
- Oscar Massimiliano Epis
- Contact Person Email
- oscar.epis@ospedaleniguarda.it
- Site Name
- Humanitas Mirasole S.p.A.
- Department Name
- 3205:U.O. Reumatologia ed Immunologia
- Principal Investigator Name
- Carlo Selmi
- Principal Investigator Email
- carlo.selmi@humanitas.it
- Contact Person Name
- Carlo Selmi
- Contact Person Email
- carlo.selmi@humanitas.it
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- PAREXEL International GmbH
- Responsibilities
- Ancillary supplies for infusion
- Name
- Parexel International (IRL) Limited
- Name
- Icon Clinical Research Limited
- Name
- Syneos Health Inc.
- Name
- IQVIA Limited
- Name
- Iqvia Biotech LLC
- Responsibilities
- Site support
- Name
- Veeda Clinical Research Limited
Third parties
- {"country":"United States","full_name":"Somalogic Operating Co. Inc.","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Cristcot HCA LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ePRO/eCOA","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"GuruKool LLC","duties_or_roles":"Rater’s trainings","organisation_type":"SME"}
- {"country":"Italy","full_name":"Phardis S.r.l.","duties_or_roles":"Local drug supply","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Travel concierge and patient re-imbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"PAREXEL International GmbH","duties_or_roles":"Ancillary supplies for infusion","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Rules Based Medicine Inc.","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"Site support","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Diagnostic Services Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"eResearchTechnology GmbH","duties_or_roles":"Spirometry review, ECG central reading and storage","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Creapharm Clinical Supplies","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"TMF archive","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Imaging storage and reading – HRCT scans","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Navigate Biopharma Services Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient facing materials (videos, print material)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Denmark","full_name":"Clinigma ApS","duties_or_roles":"In-trial interviews","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Irepertoire Inc.","duties_or_roles":"","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
- MYCOPHENOLATE MOFETIL
- Active Substance
- MYCOPHENOLATE MOFETIL
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- 2
- Investigational Product Name
- CYCLOPHOSPHAMIDE
- Active Substance
- CYCLOPHOSPHAMIDE
- Modality
- Small molecule
- Routes Of Administration
- INTRAVENOUS USE / IV INFUSION
- Route
- INTRAVENOUS USE
- Authorisation Status
- 2
- Investigational Product Name
- TACROLIMUS
- Active Substance
- TACROLIMUS
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Authorisation Status
- 2
- Investigational Product Name
- RITUXIMAB
- Active Substance
- RITUXIMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- IV INFUSION
- Route
- IV INFUSION
- Authorisation Status
- 2
- Orphan Designation
- Yes
- 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
- MYCOPHENOLIC ACID
- Active Substance
- MYCOPHENOLIC ACID
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- 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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