Clinical trial • Phase III • Oncology|Rare Disease
TOVORAFENIB for Pediatric low-grade glioma (RAF-altered)
Phase III trial of TOVORAFENIB for Pediatric low-grade glioma (RAF-altered).
Overview
- Trial Therapeutic Area
- Oncology|Rare Disease
- Trial Disease
- Pediatric low-grade glioma (RAF-altered)
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 25-09-2024
- First CTIS Authorization Date
- 29-10-2024
Trial design
Randomised, open-label, standard of care (soc) chemotherapy comparator arms including vincristine-based regimens and vinblastine or carboplatin regimens. comparator products listed: vincrisin 1 mg/ml solution injectable (vincristine sulfate) iv; cellcristin® 1 mg/ml (vincristine sulfate) iv; vincristinesulfaat teva 1 mg/ml iv; velbe 10 mg (vinblastine sulfate) iv; vinblastin stada 10 mg powder for injection (vinblastine sulfate) iv; carboplatin hikma 10 mg/ml concentrate for infusion (carboplatin) iv. regimen durations described in protocol: 60 weeks for cog-vc, 81 weeks for siope-lgg-v/c, 70 weeks for vbl, or 72 weeks for monthly carboplatin. test arm: day101 (tovorafenib) monotherapy (oral tablet or powder for oral suspension) until progression/unacceptable toxicity/withdrawal/end of study.-controlled Phase III trial in Denmark, Spain, Netherlands and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Standard of Care (SoC) chemotherapy comparator arms including vincristine-based regimens and vinblastine or carboplatin regimens. Comparator products listed: VINCRISIN 1 mg/ml solution injectable (vincristine sulfate) IV; cellcristin® 1 mg/ml (vincristine sulfate) IV; Vincristinesulfaat Teva 1 mg/ml IV; VELBE 10 mg (vinblastine sulfate) IV; Vinblastin STADA 10 mg powder for injection (vinblastine sulfate) IV; Carboplatin Hikma 10 mg/ml concentrate for infusion (carboplatin) IV. Regimen durations described in protocol: 60 weeks for COG-VC, 81 weeks for SIOPe-LGG-V/C, 70 weeks for VBL, or 72 weeks for monthly carboplatin. Test arm: DAY101 (tovorafenib) monotherapy (oral tablet or powder for oral suspension) until progression/unacceptable toxicity/withdrawal/end of study.
- Target Sample Size
- 155
- Trial Duration For Participant
- 567
Eligibility
Recruits 155 paediatric patients.
- Vulnerable Population
- The study enrols children and adolescents (patients <25 years) and explicitly includes minors. The documentation package contains age‑appropriate assent forms (examples in documents: Assent 11 years and younger; Assent 12-14 years; Assent 15-17 years; Assent 7-12 years; Assent 13 years and older, etc.) and parental/guardian main consent forms (Parental Main, Parental Optional Tumor, Parental Optional Biobank, Power of Attorney Between Parents). Site-specific 'Scout' procedures and site information are provided to support identification and approach of potential pediatric participants. Consent/assent materials are translated/localised for participating countries (multiple language versions available: Danish, Spanish, Dutch, Hungarian, Greek, Italian, French, German, Czech, Swedish, Finnish, Slovenian, etc.).
Inclusion criteria
- {"criterion_text":"- Less than 25 years of age with LGG with known activating RAF alteration\n- Histopathologic diagnosis of glioma or glioneuronal tumor\n- At least one measurable lesion\n- Meet indication for first-line systemic therapy"}
Exclusion criteria
- {"criterion_text":"- Patient has any of the following tumor-histological findings: a. \tSchwannoma b. \tSubependymal giant cell astrocytoma (Tuberous Sclerosis) c. \tDiffuse intrinsic pontine glioma, even if histologically diagnosed as WHO Grade I-II\n- Patient's tumor has additional pathogenic molecular alterations\n- Known or suspected diagnosis of neurofibromatosis Type 1 or 2 (NF1/NF-2)\n- Prior or ongoing nonsurgical anticancer therapy for this indication (eg, chemotherapy, oral/IV targeted therapy) including radiation."}
Endpoints
Primary endpoints
- {"endpoint_text":"- ORR, per RAPNO criteria, defined as the proportion of patients with overall confirmed response of complete response (CR), partial response (PR), or minor response (MR).","definition_or_measurement_approach":"ORR assessed per RAPNO criteria by Independent Review Committee (IRC); defined as proportion of patients with confirmed overall response of CR, PR, or MR."}
Secondary endpoints
- {"endpoint_text":"- PFS per RAPNO criteria, defined as time from randomization to progressive disease (PD) or death from any cause, whichever comes first.","definition_or_measurement_approach":"PFS assessed per RAPNO criteria by IRC; time from randomization to PD or death (any cause)."}
- {"endpoint_text":"- EFS per RAPNO criteria, defined as time from randomization to PD, death from any cause, or initiation of any new anticancer therapy, whichever comes first.","definition_or_measurement_approach":"EFS assessed per RAPNO criteria by IRC; time from randomization to PD, death, or start of new anticancer therapy, whichever occurs first."}
- {"endpoint_text":"- OS, defined as time from randomization up to death from any cause.","definition_or_measurement_approach":"Overall survival measured as time from randomization to death from any cause."}
Recruitment
- Planned Sample Size
- 155
- Recruitment Window Months
- 89
- Consent Approach
- Informed consent is obtained from parents/legal guardians for minors; age‑appropriate assent is obtained from children/adolescents with dedicated assent documents (multiple age bands: e.g., assent for 11 years and younger, 12-14 years, 15-17 years, 7-12 years, 13 years and older, etc.). There are parental main consent forms and participant main consent forms, plus addenda for optional tumor specimen collection, optional biobank, pregnant partner data collection, continuation, and treatment‑beyond‑progression. A Power of Attorney Between Parents document is available where relevant. Consent/assent materials and supporting documents are provided in local languages for participating countries; site-specific procedures (K1 recruitment procedure documents) and Scout agreements describe how potential participants are approached. Participant safety follow-up (30-day safety follow-up) and long-term follow-up procedures are included in the protocol and consent materials.
Methods
- Physician referral letters and HCP outreach (documents: Physician Referral Letter; country-specific K1/K2 recruitment procedures) — channel: hospital clinicians and pediatric oncology departments; target audience: treating pediatric oncologists; country-specific procedures present (examples: K1/K2 documents for NL, ES, IT, DE, DK, HU, NO, IE, EL, BE, FR, FI, SE, CZ, SI).
- Patient-facing trial cards and informational materials (Trial Card / Patient Facing Trial Card) in local languages for direct patient/parent information — channel: printed materials given in clinic; target audience: patients/parents (pediatric patients) and guardians; country-specific translations present.
- Healthcare professional trial cards (HCP Trial Card) to inform clinicians about trial eligibility and contact information — channel: HCP-targeted materials; target audience: referring physicians and hospital staff.
- Scout program / scout agreements (Scout Clinical; Scout Agreement documents) to support identification of potential participants in the community and by site-designated scouts.
- Site-based recruitment via pediatric oncology clinics and tumor boards (sites listed per country with pediatric oncology departments).
- Use of standard site communications / translations support (Transperfect Translations) and EDC contact (Medidata Rave) to manage and support trial communications and enrollment logistics.
Geography
- Total Number Of Sites
- 57
- Total Number Of Participants
- 245
Denmark
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 04-11-2024
- Processing Time Days
- 24
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Rigshospitalet
- Department Name
- Department of Pediatric Hematology and Oncology Department of Pediatric and Adolescence Medicine
- Contact Person Name
- Astrid Sehested
- Contact Person Email
- astrid.marie.sehested@regionh.dk
- Site Name
- Aarhus Universitetshospital
- Department Name
- Department of Pediatrics and Adolescent Medicine
- Contact Person Name
- Louise Henriksen
- Contact Person Email
- louhen@rm.dk
Spain
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 31-10-2024
- Processing Time Days
- 20
- Number Of Sites
- 7
- Number Of Participants
- 35
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Pediatric Oncology Department
- Contact Person Name
- Anna Llort
- Contact Person Email
- anna.llort@vallhebron.cat
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Pediatric Oncology Department
- Contact Person Name
- Miriam Pavón Mengual
- Contact Person Email
- miriam.pavon@sjd.es
- Site Name
- Hospital Universitario De Cruces
- Department Name
- Pediatric Hemato-Oncology Department
- Contact Person Name
- Miguel Alejandro Garcia-Ariza
- Contact Person Email
- miguelalejandro.garciaariza@osakidetza.eus
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Pediatric Hemato-Oncology Department
- Contact Person Name
- Adela Cañete
- Contact Person Email
- canyete_ade@gva.es
- Site Name
- Hospital Universitario La Paz
- Department Name
- Pediatric Hemato-Oncology Department
- Contact Person Name
- Diego Plaza López de Sabando
- Contact Person Email
- diego.plaza@salud.madrid.org
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Pediatric Oncology Department
- Contact Person Name
- Palma Solano Paez
- Contact Person Email
- palma.solano.sspa@juntadeandalucia.es
- Site Name
- Hospital Infantil Universitario Nino Jesus
- Department Name
- Pediatric Hemato-Oncology Department
- Contact Person Name
- Alvaro Lassaletta Atienza
- Contact Person Email
- alvaro.lassaletta@salud.madrid.org
Netherlands
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 29-10-2024
- Processing Time Days
- 18
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Prinses Maxima Centrum voor Kinderoncologie B.V.
- Department Name
- Pediatric hemato-Oncology
- Contact Person Name
- Jasper van der Lugt
- Contact Person Email
- j.vanderlugt@prinsesmaximacentrum.nl
Slovenia
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 08-11-2024
- Processing Time Days
- 28
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- University Medical Center Ljubljana
- Department Name
- Department of Hematology and Oncology
- Contact Person Name
- Barbara Faganel Kotnik
- Contact Person Email
- barbara.faganel@gmail.com
Austria
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 05-11-2024
- Processing Time Days
- 25
- Number Of Sites
- 2
- Number Of Participants
- 12
Sites
- Site Name
- Medizinische Universitaet Innsbruck
- Department Name
- Department für Kinder- und Jugendheilkunde
- Contact Person Name
- Roman Crazzolara
- Contact Person Email
- roman.crazzolara@i-med.ac.at
- Site Name
- Medical University Of Vienna
- Department Name
- Universitätsklinik für Neurologie
- Contact Person Name
- Amedeo Azizi
- Contact Person Email
- amedeo.azizi@meduniwien.ac.at
Hungary
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 30-10-2024
- Processing Time Days
- 19
- Number Of Sites
- 1
- Number Of Participants
- 9
Sites
- Site Name
- Semmelweis University
- Department Name
- Oncology
- Contact Person Name
- Miklós Garami
- Contact Person Email
- miklos.garami@gmail.com
Norway
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 30-10-2024
- Processing Time Days
- 19
- Number Of Sites
- 2
- Number Of Participants
- 12
Sites
- Site Name
- Oslo University Hospital HF
- Department Name
- Oncology and Hematology
- Contact Person Name
- Aina Ulvmoen
- Contact Person Email
- ainulv@ous-hf.no
- Site Name
- Universitetssykehuset Nord-Norge HF
- Department Name
- Oncology and Hematology
- Contact Person Name
- Ole Mikal Wormdal
- Contact Person Email
- ole.mikal.wormdal@unn.no
Ireland
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 30-10-2024
- Processing Time Days
- 19
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Children's Health Ireland
- Department Name
- Division of Haematology/Oncology
- Contact Person Name
- Sarah Curry
- Contact Person Email
- sarah.curry@olchc.ie
Greece
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 09-12-2024
- Processing Time Days
- 59
- Number Of Sites
- 2
- Number Of Participants
- 13
Sites
- Site Name
- Nosokomeio Paidon I Agia Sofia
- Department Name
- Unit of Pediatric Hematology and Oncology, First Department of Pediatrics.
- Contact Person Name
- Antonis Kattamis
- Contact Person Email
- ankatt@med.uoa.gr
- Site Name
- Athens General Children's Hospital Panagioti And Aglaia Kyriakou
- Department Name
- Oncology Department
- Contact Person Name
- Dimitrios Doganis
- Contact Person Email
- doganisd@gmail.com
Belgium
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 30-10-2024
- Processing Time Days
- 19
- Number Of Sites
- 3
- Number Of Participants
- 9
Sites
- Site Name
- UZ Leuven
- Department Name
- Pediatric Hematology and Oncology
- Contact Person Name
- Sandra Jacobs
- Contact Person Email
- tessa.deroo@uzleuven.be
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Pediatric Hemato-Oncology
- Contact Person Name
- Leen Willems
- Contact Person Email
- gudrun.lerno@uzgent.be
- Site Name
- Cliniques Universitaires Saint-Luc
- Department Name
- Paediatric hemato-oncology
- Contact Person Name
- An van Damme
- Contact Person Email
- georgia.demarteau@saintluc.uclouvain.be
France
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 13-11-2024
- Processing Time Days
- 33
- Number Of Sites
- 5
- Number Of Participants
- 25
Sites
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- La Timone Children Hospital, Department of Pediatric Hematology Oncology
- Contact Person Name
- Nicolas Andre
- Contact Person Email
- Nicolas.andre@ap-hm.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Institute of Pediatric Hematology and Oncology (iHOPe) GCS Lyon University Oncology
- Contact Person Name
- Pierre Leblond
- Contact Person Email
- pierre.leblond@ihope.fr
- Site Name
- Institut Gustave Roussy
- Department Name
- Pediatric Unit
- Contact Person Name
- Samuel Abbou
- Contact Person Email
- Samuel.abbou@gustaveroussy.fr
- Site Name
- Centre Oscar Lambret
- Department Name
- Pediatric Unit. Oncology department
- Contact Person Name
- Sandra Raimbault
- Contact Person Email
- s-raimbault@o-lambret.fr
- Site Name
- Institut Curie
- Department Name
- Pediatric department
- Contact Person Name
- François Doz
- Contact Person Email
- Francois.doz@curie.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 04-11-2024
- Processing Time Days
- 24
- Number Of Sites
- 9
- Number Of Participants
- 20
Sites
- Site Name
- Azienda Sanitaria Universitaria Friuli Centrale
- Department Name
- P.O. Santa Maria della Misericordia SOC Clinica Pediatrica
- Contact Person Name
- Chiara Pilotto
- Contact Person Email
- chiara.pilotto@asufc.sanita.fvg.it
- Site Name
- Azienda Ospedaliera Universitaria Meyer IRCCS
- Department Name
- SOSD NeuroOncology
- Contact Person Name
- Carla Fonte
- Contact Person Email
- carla.fonte@meyer.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- Dipartimento di Oncoematologia, Terapia Cellulare, Terapie Geniche e trapianto Emopoietico
- Contact Person Name
- Angela Mastronuzzi
- Contact Person Email
- angela.mastronuzzi@opbg.net
- Site Name
- Fondazione IRCCS Istituto Nazionale Dei Tumori
- Department Name
- Dipartimento di Oncologia ed Ematologia
- Contact Person Name
- Elisabetta Schiavello
- Contact Person Email
- elisabetta.schiavello@istitutotumori.mi.it
- Site Name
- University Hospital Consorziale Policlinico
- Department Name
- Unità Operativa Complessa di Pediatria ad Indirizzo Oncoematologico
- Contact Person Name
- Teresa Perillo
- Contact Person Email
- terryperillo@hotmail.com
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- Oncoematologia Pediatrica
- Contact Person Name
- Elisabetta Viscardi
- Contact Person Email
- elisabetta.viscardi@unipd.it
- Site Name
- Azienda Ospedaliera Santobono Pausilipon
- Department Name
- Dipartimento di Oncologia Pediatrica
- Contact Person Name
- Lucia Quaglietta
- Contact Person Email
- l.quaglietta@santobonopausilipon.it
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- Emato-Oncologia Pediatrica
- Contact Person Name
- Claudia Milanaccio
- Contact Person Email
- claudiamilanaccio@gaslini.org
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
- Department Name
- SC Oncoematologia Pediatrica
- Contact Person Name
- Franca Fagioli
- Contact Person Email
- franca.fagioli@unito.it
Finland
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 30-10-2024
- Processing Time Days
- 19
- Number Of Sites
- 2
- Number Of Participants
- 9
Sites
- Site Name
- HUS-Yhtymae
- Department Name
- Hematology and oncology
- Contact Person Name
- Satu Langström
- Contact Person Email
- satu.langstrom@hus.fi
- Site Name
- Tampere University Hospital
- Department Name
- Hematology and oncology
- Contact Person Name
- Kristiina Nordfors
- Contact Person Email
- kristiina.nordfors@pirha.fi
Sweden
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 30-10-2024
- Processing Time Days
- 19
- Number Of Sites
- 3
- Number Of Participants
- 13
Sites
- Site Name
- Region Skane Skanes Universitetssjukhus
- Department Name
- Pediatric Cancer center, Skånes Universitetssjukhus Lund
- Contact Person Name
- Helena Morse
- Contact Person Email
- helena.morse@skane.se
- Site Name
- Queen Silvia Childrens Hospital - Sahlgrenska University Hospital
- Department Name
- Pediatric Clinical Research Center, Drottning Silivias barnsjukhus
- Contact Person Name
- Aron Onerup
- Contact Person Email
- aron.onerup@vgregion.se
- Site Name
- Karolinska University Hospital
- Department Name
- Childhood cancer center, Astrid Lindgrens Barnsjukhus
- Contact Person Name
- Tomas Sjöberg Bexelius
- Contact Person Email
- tomas.sjoberg-bexelius@regionstockholm.se
Germany
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 05-11-2024
- Processing Time Days
- 25
- Number Of Sites
- 14
- Number Of Participants
- 35
Sites
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- Pediatrics III
- Contact Person Name
- Stephan Tippelt
- Contact Person Email
- Stephan.tippelt@uk-essen.de
- Site Name
- Universitaetsklinikum Heidelberg AöR
- Department Name
- Klinische Kooperationseinheit Pädiatrische Onkologie
- Contact Person Name
- Olaf Witt
- Contact Person Email
- o.witt@kitz-heidelberg.de
- Site Name
- Universitaetsklinikum Erlangen AöR
- Department Name
- Kinder und Jugendklinik
- Contact Person Name
- Markus Metzler
- Contact Person Email
- markus.metzler@uk-erlangen.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Pädiatrische Klinik mit Schwerpunkt Onkologie und Hämatologie
- Contact Person Name
- Pablo Hernáiz Driever
- Contact Person Email
- pablo.hernaiz@charite.de
- Site Name
- Universitaet Leipzig
- Department Name
- Kinderklinik, selbst. Abteilung für Pädiatrische Onkologie, Hämatologie und Hämostaseologie
- Contact Person Name
- Susanne Wendt
- Contact Person Email
- susanne.wendt@medizin.uni-leipzig.de
- Site Name
- Universitaetsklinikum Frankfurt AöR
- Department Name
- Clinic for Pediatrics and Adolescent Medicine
- Contact Person Name
- Jan-Henning Klusmann
- Contact Person Email
- kkjm-direktor@ukffm.de
- Site Name
- Evangelisches Klinikum Bethel gGmbH
- Department Name
- Campus Bielefeld-Bethel, Universitätsklinik für Kinder- und Jugendmedizin
- Contact Person Name
- Norbert Jorch
- Contact Person Email
- norbert.jorch@evkb.de
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- Paediatric Haematology and Oncology
- Contact Person Name
- Uwe Kordes
- Contact Person Email
- kordes@uke.de
- Site Name
- Universitaetsklinikum Augsburg
- Department Name
- Schwäbisches Kinderkrebszentrum
- Contact Person Name
- Michael Frühwald
- Contact Person Email
- michael.fruehwald@uk-augsburg.de
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Universitäts klinik für Kinder- und Jugendmedizin
- Contact Person Name
- Ursula Holzer
- Contact Person Email
- ursula.holzer@med.uni-tuebingen.de
- Site Name
- Medical Center - University Of Freiburg
- Department Name
- Zentrum für Kinder-und Jugendmedizin
- Contact Person Name
- Miriam van Buiren
- Contact Person Email
- miriam.buiren@uniklinik-freiburg.de
- Site Name
- Medizinische Hochschule Hannover
- Department Name
- Department of Pediatric Hematology and Oncology
- Contact Person Name
- Martin Stanulla
- Contact Person Email
- Stanulla.martin@mh-hannover.de
- Site Name
- Universitaetsklinikum Wuerzburg AöR
- Department Name
- Kinderklinik und Poliklinik
- Contact Person Name
- Matthias Eyrich
- Contact Person Email
- Eyrich_M@ukw.de
- Site Name
- Universitaetsmedizin Goettingen
- Department Name
- Division of Pediatric Oncology and Hematology
- Contact Person Name
- Ingrid Kühnle
- Contact Person Email
- Ingrid.Kuehnle@med.uni-goettingen.de
Czechia
- Earliest CTIS Part Ii Submission Date
- 11-10-2024
- Latest Decision Or Authorization Date
- 31-10-2024
- Processing Time Days
- 20
- Number Of Sites
- 2
- Number Of Participants
- 15
Sites
- Site Name
- Fakultni Nemocnice Motol A Homolka
- Department Name
- Klinika dětské hematologie a onkologie
- Contact Person Name
- Michal Zápotocký
- Contact Person Email
- michal.zapotocky@fnmotol.cz
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Dětská Nemocnice, Klinika dětské onkologie
- Contact Person Name
- Zdeněk Pavelka
- Contact Person Email
- pavelka.zdenek@fnbrno.cz
Sponsor
Primary sponsor
- Full Name
- Day One Biopharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Icon Clinical Research Limited
- Responsibilities
- - Clinical operations and site support; Ex-US RA/MoH, EC/IRB, investigator expedited and periodic safety reporting; training portal and document exchange (Firecrest); translation services via ICON Language Solutions; multiple operational duties (codes 1,2,5,6,8,11,12,15 as listed).
- Name
- PRA Hellas CRO A.E.
- Responsibilities
- - Regional CRO responsibilities including clinical operations, regulatory support and other contracted activities (codes include 1,12,2,6).
- Name
- PPD Development LP
- Responsibilities
- - Release testing and stability testing of study IMPs (tablets, powder for reconstitution).
- Name
- IQVIA Inc.
- Responsibilities
- - Safety database (Argus) hosting and management.
- Name
- ClinChoice
- Responsibilities
- - Regulatory support/QP certification and local regulatory activities (includes QP Certification – EU for SoC chemotherapy).
Third parties
- {"country":"United States","full_name":"ClinChoice","duties_or_roles":"Codes: 10; 15 (QP Certification – EU (SoC chemotherapy))","organisation_type":"Industry"}
- {"country":"United Kingdom","full_name":"Myonex Limited","duties_or_roles":"15 (QP Certification – UK (SoC chemotherapy) - Investigational Labeling (SoC chemotherapy))","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"15 (Tumor specimen/slides samples storage)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"15 (Release Testing (tablets, powder for reconstitution) - Stability Testing (tablets, powder for reconstitution))","organisation_type":"Pharmaceutical company"}
- {"country":"China","full_name":"Shanghai Syntheall Pharmaceutical Co. Ltd.","duties_or_roles":"15 (Manufacture, testing, and stability of Regulatory Starting Materials (ACTA))","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"15 (EDC Medidata Rave, contracted via ICON)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Inseption Group LLC","duties_or_roles":"11; 15 (Clinical Operations Support Resource - Trial Master File Management and Support)","organisation_type":"Pharmaceutical company"}
- {"country":"Canada","full_name":"Clinical Logistics Inc.","duties_or_roles":"15 (Central lab kits management)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Imaging Endpoints II LLC","duties_or_roles":"15 (Medical image analysis; review - X-ray, MRI, ultrasound, etc.)","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"15 (Tumor specimen/slides samples storage)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"15 (Patient travel reimbursement and travel coordinator)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United Kingdom","full_name":"Imperial Clinical Research Services International Limited","duties_or_roles":"15 (Printing services, contracted via ICON)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"14; 15 (QP release - EU (tablets, powder for reconstitution))","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Fisher Clinical Services Inc.","duties_or_roles":"14","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Experic LLC","duties_or_roles":"15 (Packaging and Labeling (tablets, powder for reconstitution))","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Q International Courier LLC","duties_or_roles":"15 (Shipping courier for laboratory samples from investigational sites to short and long term storage and testing laboratories)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"15 (IMP label translation; SmPC translations)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"3","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Quotient Sciences Philadelphia LLC","duties_or_roles":"15 (Manufacturing (tablets, powder for reconstitution) -Release testing (powder for reconstitution) -Stability testing (powder for reconstitution))","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"QPS LLC","duties_or_roles":"15 (Plasma Samples analysis and storage)","organisation_type":"Pharmaceutical company"}
- {"country":"Denmark","full_name":"Klifo A/S","duties_or_roles":"14; 15 (QP Certification – EU (SoC chemotherapy))","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"1; 11; 12; 15; 2; 5; 6; 8 (Extensive clinical operations, regulatory and safety reporting, training and document exchange, translation services via ICON Language Solutions, Ex‑US RA/MoH, EC/IRB support; Investigator expedited and periodic safety reporting)","organisation_type":"Pharmaceutical company"}
- {"country":"Luxembourg","full_name":"TecEx","duties_or_roles":"15 (Importer of records, contracted via ICON)","organisation_type":"Industry"}
- {"country":"United States","full_name":"Iqvia Inc.","duties_or_roles":"15 (EDC / Safety Database hosting and management)","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"1; 12; 2; 6 (Clinical operations support functions)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Tovorafenib
- Active Substance
- TOVORAFENIB
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Authorisation Status
- Investigational (prodAuthStatus=1)
- Orphan Designation
- Yes
- Maximum Dose
- 600 mg (maxDailyDoseAmount 600.00 mg)
- Investigational Product Name
- VINCRISIN 1 mg/ml solution injectable, 2 mg (vincristine sulfate)
- Active Substance
- VINCRISTINE SULFATE
- Modality
- Small molecule
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS
- Authorisation Status
- Marketing authorisation (prodAuthStatus=2)
- Maximum Dose
- 2 mg (maxDailyDoseAmount 2.00 mg)
- Investigational Product Name
- Carboplatin Hikma 10 mg/ml Konzentrat zur Herstellung einer Infusionslösung
- Active Substance
- CARBOPLATIN
- Modality
- Small molecule
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS
- Authorisation Status
- Marketing authorisation (prodAuthStatus=2)
- Maximum Dose
- 1050 mg (maxDailyDoseAmount 1050.00 mg)
- Investigational Product Name
- VELBE 10 mg Trockensubstanz zur Injektionsbereitung / Vinblastin STADA 10 mg Pulver zur Herstellung einer Injektionslösung (vinblastine sulfate)
- Active Substance
- VINBLASTINE SULFATE
- Modality
- Small molecule
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS
- Authorisation Status
- Marketing authorisation (prodAuthStatus=2)
- Maximum Dose
- 10 mg (maxDailyDoseAmount 10.00 mg)
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