Clinical trial • Phase III • Haematology|Rare Disease
LUSPATERCEPT for Myelodysplastic syndrome|Lower-risk myelodysplastic syndrome
Phase III trial of LUSPATERCEPT for Myelodysplastic syndrome|Lower-risk myelodysplastic syndrome. open-label, none/not specified-controlled.
Overview
- Trial Therapeutic Area
- Haematology|Rare Disease
- Trial Disease
- Myelodysplastic syndrome|Lower-risk myelodysplastic syndrome
- Trial Stage
- Phase III
- Drug Modality
- Peptide/protein/enzyme
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 06-10-2023
- First CTIS Authorization Date
- 13-02-2024
Trial design
open-label, none/not specified-controlled Phase III trial in Belgium, Czechia, France and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 72
- Trial Duration For Participant
- 730
Eligibility
Recruits 72 The record indicates isVulnerablePopulationSelected = true. All participants must be 18 years or older (consent provided by participant). Country-specific subject information and informed consent forms (ICFs) are provided (multiple languages and versions). There are specific ICF templates for pregnant partner and newborn (e.g. PP_Newborn ICF in Belgium) and optional / future research/sample collection ICFs, indicating tailored consent documents for specific situations. No assent for minors is applicable because minimum age is 18..
- Pregnancy Exclusion
- pregnant, plan to get pregnant or breastfeeding
- Vulnerable Population
- The record indicates isVulnerablePopulationSelected = true. All participants must be 18 years or older (consent provided by participant). Country-specific subject information and informed consent forms (ICFs) are provided (multiple languages and versions). There are specific ICF templates for pregnant partner and newborn (e.g. PP_Newborn ICF in Belgium) and optional / future research/sample collection ICFs, indicating tailored consent documents for specific situations. No assent for minors is applicable because minimum age is 18.
Inclusion criteria
- {"criterion_text":"- Participants are eligible to be included in the study if all following criteria apply: A) Participant must be 18 years or older, have documented diagnosis of MDS, have an Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2. For Cohort 1 only: participant must have an endogenous serum erythropoietin (EPO) level of < 500 U/L and no prior ESA treatment. For Cohort 2 only: participant must be refractory or intolerant to prior ESA treatment as defined by any of the following: i) Refractory to prior ESA treatment: Documentation of nonresponse or response that was no longer maintained to prior ESA-containing regimen, either as a single agent or in combination (eg, with G-CSF). The ESA regimen must have been either: (1) Recombinant human erythropoietin ≥ 40,000 IU/week for at least 8 doses or equivalent; or 1050 mg/kg/week for at least 8 doses or equivalent OR (2) Darbepoetin- darbepoetin alpha ≥ 240 μg every week for at least 12-weeks or equivalent ii) Intolerant to prior ESA treatment: Documentation of discontinuation of prior ESA-containing regimen, either as a single agent or in combination (eg, with G-CSF), at any time after introduction due to intolerance or an AE. B) And must require RBC transfusion documented by the following criteria: i) Average transfusion requirement of ≥ 1 units of packed RBCs (pRBCs) confirmed for a minimum of 8-weeks immediately prior to first treatment of luspatercept ii) Hb levels at the time of or within 7 days prior to administration of an RBC transfusion must be ≤ 10.0 g/dL in order for the transfusion to be counted towards meeting eligibility criteria. RBC transfusions administered when Hb levels are > 10 g/dL and/or RBC transfusions administered for elective surgery do not qualify as a required transfusion for the purpose of meeting eligibility criteria"}
Exclusion criteria
- {"criterion_text":"- A participant will be excluded from the study if they have a secondary MDS, known history of diagnosis of AML, prior allogenic or autologous stem cell transplant, history of allergy or hypersensitivity to study drug components, pregnant, plan to get pregnant or breastfeeding, prior history of malignancies, known significant anemia due to iron, vitamin B12 or folate deficiencies."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Primary endpoint: composite of achievement of RBC-TI for 8-weeks with a concurrent mean Hb increase of ≥ 1 g/dL from Week 1 to Week 24.","definition_or_measurement_approach":"Composite endpoint defined as achievement of red blood cell transfusion independence (RBC-TI) for 8 weeks with an associated concurrent mean hemoglobin (Hb) increase of ≥ 1 g/dL measured from Week 1 to Week 24."}
Secondary endpoints
- {"endpoint_text":"- Main secondary efficacy endpoints: mean change in total RBC units transfused over a fixed 16 week period from Week 9 to Week 24 and from Week 33 to Week 48. Time from first dose to first onset of RBC TI ≥ 8, 12, and 16 weeks, Maximum duration of RBC-TI for participants who achieve RBC TI ≥ 8- and 16-week period","definition_or_measurement_approach":"Efficacy endpoints include mean change in total RBC units transfused over fixed 16-week intervals (Week 9–24 and Week 33–48); time from first dose to first onset of RBC-TI lasting ≥8, 12, and 16 weeks; and maximum duration of RBC-TI for participants achieving RBC-TI for ≥8 and ≥16 weeks."}
- {"endpoint_text":"- Main secondary safety endpoint is the type, frequency, severity of AEs, and relationship of adverse events (AEs) to luspatercept from screening to 6 weeks (42-days) post last dose and from Week 1 to Week 48","definition_or_measurement_approach":"Safety endpoints include characterization of adverse events (type, frequency, severity) and assessment of relationship to luspatercept from screening through 6 weeks (42 days) after last dose and over Weeks 1–48."}
Recruitment
- Planned Sample Size
- 72
- Recruitment Window Months
- 38
- Consent Approach
- Informed consent is obtained from each adult participant (minimum age 18). Country-specific subject information and informed consent forms (ICFs) are provided in local languages and include main ICFs plus optional modules (optional future research, optional sample collection, pregnant-partner ICF). Specific ICF templates are available per Member State (documents listed for ES, CZ, PL, BE, FR, IT, DE).
Methods
- Investigator Recruitment by Endpoint Clinical Inc. (responsibility listed as Investigator Recruitment in sponsor third-party duties).
- Country-specific recruitment arrangements documented (recruitment arrangement (K1) documents exist for ES, CZ, PL, BE, FR, IT, DE).
- Lost-to-follow-up tracing by Omnitrace Corp. (duty: Lost to Follow up Tracing).
Geography
- Total Number Of Sites
- 37
- Total Number Of Participants
- 72
Belgium
- Earliest CTIS Part Ii Submission Date
- 10-01-2024
- Latest Decision Or Authorization Date
- 19-02-2024
- Processing Time Days
- 40
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- UZ Leuven
- Department Name
- Hematology Dept.
- Principal Investigator Name
- Mariëlle Beckers
- Principal Investigator Email
- marielle.beckers@uzleuven.be
- Contact Person Name
- Mariëlle Beckers
- Contact Person Email
- marielle.beckers@uzleuven.be
- Site Name
- Algemeen Ziekenhuis Delta
- Department Name
- Hematology Dept
- Principal Investigator Name
- Dries Deeren
- Principal Investigator Email
- dries.deeren@azdelta.be
- Contact Person Name
- Dries Deeren
- Contact Person Email
- dries.deeren@azdelta.be
Czechia
- Earliest CTIS Part Ii Submission Date
- 16-01-2024
- Latest Decision Or Authorization Date
- 13-02-2024
- Processing Time Days
- 28
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Interní hematoonkologická klinika
- Principal Investigator Name
- Jiří Mayer
- Principal Investigator Email
- mayer.jiri@fnbrno.cz
- Contact Person Name
- Jiří Mayer
- Contact Person Email
- mayer.jiri@fnbrno.cz
- Site Name
- Institute Of Hematology And Blood Transfusion
- Department Name
- Ústav hematologie a krevni transfuze
- Principal Investigator Name
- Jaroslav Čermák
- Principal Investigator Email
- jaroslav.cermak@uhkt.cz
- Contact Person Name
- Jaroslav Čermák
- Contact Person Email
- jaroslav.cermak@uhkt.cz
- Site Name
- Vseobecna Fakultni Nemocnice V Praze
- Department Name
- I.interní klinika - klinika hematologie
- Principal Investigator Name
- Anna Jonášová
- Principal Investigator Email
- anna.jonasova@vfn.cz
- Contact Person Name
- Anna Jonášová
- Contact Person Email
- anna.jonasova@vfn.cz
France
- Earliest CTIS Part Ii Submission Date
- 15-01-2024
- Latest Decision Or Authorization Date
- 14-02-2024
- Processing Time Days
- 30
- Number Of Sites
- 7
- Number Of Participants
- 15
Sites
- Site Name
- Centre Hospitalier Universitaire Grenoble Alpes
- Department Name
- Hematology Department
- Principal Investigator Name
- Sophie Park
- Principal Investigator Email
- spark@chu-grenoble.fr
- Contact Person Name
- Sophie Park
- Contact Person Email
- spark@chu-grenoble.fr
- Site Name
- Centre Hospitalier Regional Universitaire De Tours
- Department Name
- Hematology cell therapy
- Principal Investigator Name
- Emmanuel Gyan
- Principal Investigator Email
- Emmanuel.gyan@univ-tours.fr
- Contact Person Name
- Emmanuel Gyan
- Contact Person Email
- Emmanuel.gyan@univ-tours.fr
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- Hematology Department
- Principal Investigator Name
- Thomas Cluzeau
- Principal Investigator Email
- cluzeau.t@chu-nice.fr
- Contact Person Name
- Thomas Cluzeau
- Contact Person Email
- cluzeau.t@chu-nice.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Hematology Seniors
- Principal Investigator Name
- Lionel Ades
- Principal Investigator Email
- Lionel.ades@aphp.fr
- Contact Person Name
- Lionel Ades
- Contact Person Email
- Lionel.ades@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Poitiers
- Department Name
- Onco-Hematology and Cell Therapy Department
- Principal Investigator Name
- Jose Miguel Torregrosa-Diaz
- Principal Investigator Email
- jose-miguel.torregrosa-diaz@chu-poitiers.fr
- Contact Person Name
- Jose Miguel Torregrosa-Diaz
- Contact Person Email
- jose-miguel.torregrosa-diaz@chu-poitiers.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Hematology Department
- Principal Investigator Name
- Maël Heiblig
- Principal Investigator Email
- Mael.heiblig@chu-lyon.fr
- Contact Person Name
- Maël Heiblig
- Contact Person Email
- Mael.heiblig@chu-lyon.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- Blood diseases service
- Principal Investigator Name
- Sylvain Thepot
- Principal Investigator Email
- Sylvain.Thepot@chu-angers.fr
- Contact Person Name
- Sylvain Thepot
- Contact Person Email
- Sylvain.Thepot@chu-angers.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 12-01-2024
- Latest Decision Or Authorization Date
- 13-02-2024
- Processing Time Days
- 32
- Number Of Sites
- 8
- Number Of Participants
- 16
Sites
- Site Name
- Azienda Ospedaliera Ordine Mauriziano Di Torino
- Department Name
- SCDU Ematologia e Terapie cellulari
- Principal Investigator Name
- Daniela Cilloni
- Principal Investigator Email
- daniela.cilloni@unito.it
- Contact Person Name
- Daniela Cilloni
- Contact Person Email
- daniela.cilloni@unito.it
- Site Name
- Azienda Ospedaliero-Universitaria Maggiore Della Carita
- Department Name
- SCDU Ematologia
- Principal Investigator Name
- Andrea Patriarca
- Principal Investigator Email
- andrea.patriarca@uniupo.it
- Contact Person Name
- Andrea Patriarca
- Contact Person Email
- andrea.patriarca@uniupo.it
- Site Name
- Humanitas Research Hospital
- Department Name
- Oncologia ed Ematologia
- Principal Investigator Name
- Matteo Della Porta
- Principal Investigator Email
- matteo.della_porta@hunimed.eu
- Contact Person Name
- Matteo Della Porta
- Contact Person Email
- matteo.della_porta@hunimed.eu
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- U.O.C. Ematologia e trapianti di midollo
- Principal Investigator Name
- Fabrizio Pane
- Principal Investigator Email
- fabpane@unina.it
- Contact Person Name
- Fabrizio Pane
- Contact Person Email
- fabpane@unina.it
- Site Name
- Fondazione IRCCS Policlinico San Matteo
- Department Name
- SC Ematologia I
- Principal Investigator Name
- Luca Malcovati
- Principal Investigator Email
- luca.malcovati@unipv.it
- Contact Person Name
- Luca Malcovati
- Contact Person Email
- luca.malcovati@unipv.it
- Site Name
- Careggi University Hospital
- Department Name
- SOD Ematologia
- Principal Investigator Name
- Valeria Santini
- Principal Investigator Email
- dmsc@pec.unifi.it
- Contact Person Name
- Valeria Santini
- Contact Person Email
- dmsc@pec.unifi.it
- Site Name
- Azienda Ospedaliera Policlinico Universitario Tor Vergata
- Department Name
- Biomedicina e Prevenzione
- Principal Investigator Name
- Maria Teresa Voso
- Principal Investigator Email
- voso@med.uniroma2.it
- Contact Person Name
- Maria Teresa Voso
- Contact Person Email
- voso@med.uniroma2.it
- Site Name
- Grande Ospedale Metropolitano Bianchi Melacrino Morelli
- Department Name
- UOC Ematologia
- Principal Investigator Name
- Caterina Alati
- Principal Investigator Email
- caterina.alati@gmail.com
- Contact Person Name
- Caterina Alati
- Contact Person Email
- caterina.alati@gmail.com
Germany
- Earliest CTIS Part Ii Submission Date
- 17-01-2024
- Latest Decision Or Authorization Date
- 01-03-2024
- Processing Time Days
- 44
- Number Of Sites
- 3
- Number Of Participants
- 7
Sites
- Site Name
- Universitaet Leipzig
- Department Name
- Klinik und Poliklinik für Hämatologie
- Principal Investigator Name
- Dominic Brauer
- Principal Investigator Email
- dominic.brauer@medizin.uni
- Contact Person Name
- Dominic Brauer
- Contact Person Email
- dominic.brauer@medizin.uni
- Site Name
- Studienzentrum am Raschplatz GbR
- Principal Investigator Name
- Eyck von der Heyde
- Principal Investigator Email
- vdheyde@onkologie-am-raschplatz.de
- Contact Person Name
- Eyck von der Heyde
- Contact Person Email
- vdheyde@onkologie-am-raschplatz.de
- Site Name
- Klinikum rechts der Isar der TU Muenchen AöR
- Department Name
- Klinik und Poliklinik für Innere Medizin III: Hämatologie und Internistische Onkologie
- Principal Investigator Name
- Katharina Götze
- Principal Investigator Email
- katharina.goetze@tum.de
- Contact Person Name
- Katharina Götze
- Contact Person Email
- katharina.goetze@tum.de
Spain
- Earliest CTIS Part Ii Submission Date
- 22-12-2023
- Latest Decision Or Authorization Date
- 14-02-2024
- Processing Time Days
- 54
- Number Of Sites
- 9
- Number Of Participants
- 18
Sites
- Site Name
- Complejo Hospitalario Universitario De Ourense
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Jose Luis Sastre Moral
- Principal Investigator Email
- joseluis.sastre.moral@sergas.es
- Contact Person Name
- Jose Luis Sastre Moral
- Contact Person Email
- joseluis.sastre.moral@sergas.es
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Haematology department
- Principal Investigator Name
- Elvira Mora Castera
- Principal Investigator Email
- mora_elv@gva.es
- Contact Person Name
- Elvira Mora Castera
- Contact Person Email
- mora_elv@gva.es
- Site Name
- Institut Catala D'oncologia
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Blanca Xicoy Cirici
- Principal Investigator Email
- bxicoy@iconcologia.net
- Contact Person Name
- Blanca Xicoy Cirici
- Contact Person Email
- bxicoy@iconcologia.net
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- David Valcárcel Ferreira
- Principal Investigator Email
- dvalcarcel@vhio.net
- Contact Person Name
- David Valcárcel Ferreira
- Contact Person Email
- dvalcarcel@vhio.net
- Site Name
- Institut Catala D'oncologia
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Montserrat Arnan Sangerman
- Principal Investigator Email
- marnan@iconcologia.net
- Contact Person Name
- Montserrat Arnan Sangerman
- Contact Person Email
- marnan@iconcologia.net
- Site Name
- Hospital Universitario De Salamanca
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Maria Diez Campelo
- Principal Investigator Email
- mdiezcampelo@usal.es
- Contact Person Name
- Maria Diez Campelo
- Contact Person Email
- mdiezcampelo@usal.es
- Site Name
- Hospital Universitario Virgen De Las Nieves
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Francisca Hernandez Mohedo
- Principal Investigator Email
- paquihernandez@yahoo.es
- Contact Person Name
- Francisca Hernandez Mohedo
- Contact Person Email
- paquihernandez@yahoo.es
- Site Name
- Hospital Clinico Universitario De Valencia
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Maria del Mar Tormo Diaz
- Principal Investigator Email
- tormo_mar@gva.es
- Contact Person Name
- Maria del Mar Tormo Diaz
- Contact Person Email
- tormo_mar@gva.es
- Site Name
- Hospital Universitario De La Princesa
- Department Name
- Servicio de Hematologia
- Principal Investigator Name
- Valle Gomez Garcia de Soria
- Principal Investigator Email
- vallegomez@yahoo.com
- Contact Person Name
- Valle Gomez Garcia de Soria
- Contact Person Email
- vallegomez@yahoo.com
Poland
- Earliest CTIS Part Ii Submission Date
- 10-01-2024
- Latest Decision Or Authorization Date
- 16-02-2024
- Processing Time Days
- 37
- Number Of Sites
- 5
- Number Of Participants
- 7
Sites
- Site Name
- Uniwersytecki Szpital Kliniczny Nr 1 W Lublinie
- Department Name
- Klinika Hematoonkologii i Transplantacj
- Principal Investigator Name
- Maria Cioch
- Principal Investigator Email
- mariacioch@wp.pl
- Contact Person Name
- Maria Cioch
- Contact Person Email
- mariacioch@wp.pl
- Site Name
- Mtz Clinical Research Powered By Pratia
- Principal Investigator Name
- Krzysztof Mądry
- Principal Investigator Email
- krzysztof.madry@wum.edu.pl
- Contact Person Name
- Krzysztof Mądry
- Contact Person Email
- krzysztof.madry@wum.edu.pl
- Site Name
- Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego
- Department Name
- Oddział Hematologii
- Principal Investigator Name
- Aleksandra Butrym
- Principal Investigator Email
- aleksandra.butrym@gmail.com
- Contact Person Name
- Aleksandra Butrym
- Contact Person Email
- aleksandra.butrym@gmail.com
- Site Name
- Pratia Onkologia Katowice
- Principal Investigator Name
- Sebastian Grosicki
- Principal Investigator Email
- sgrosicki@wp.pl
- Contact Person Name
- Sebastian Grosicki
- Contact Person Email
- sgrosicki@wp.pl
- Site Name
- Wojewodzkie Wielospecjalistyczne Centrum Onkologii I Traumatologii Im M.Kopernika W Lodzi
- Department Name
- Oddział Hematologii i Transplantologii - Klinika Hematologii
- Principal Investigator Name
- Agnieszka Wierzbowska
- Principal Investigator Email
- agawierzbowska@wp.pl
- Contact Person Name
- Agnieszka Wierzbowska
- Contact Person Email
- agawierzbowska@wp.pl
Sponsor
Primary sponsor
- Full Name
- Celgene Corp.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Pharmaceutical Product Development LLC
- Responsibilities
- Monitoring, SUSAR Reporting, Feasibility, Vendor Management, Clinical, Management & Monitoring, Site Start up, Services, Site Monitoring & Site, Management, Pharmacovigilance, Data Analytics
- Name
- Q2 Solutions, 2 Squared Solutions LLC
- Responsibilities
- Routine clinical pathology testing, Clinical Chemistry, Clinical haematology, Clinical microbiology, Central Lab Services, IHC Biomarker, expression analysis (PD-L1), other APH
- Name
- Endpoint Clinical Inc.
- Responsibilities
- Investigator Recruitment, IVRS - tratment randomization, Subject Number Assignment, Treatment/Arm Assignment, Drug (re) Supplies Assignment
- Name
- QPS LLC
- Responsibilities
- bioanalytical analysis, Nab
Third parties
- {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"Provide licensing services for use of the Quality of Life Questionnaire-Core 30 and translations","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Q2 Solutions, 2 Squared Solutions LLC","duties_or_roles":"Routine clinical pathology testing, Clinical Chemistry, Clinical haematology, Clinical microbilogy, Central Lab Services, IHC Biomarker, expression analysis (PD-L1), other APH","organisation_type":"Industry"}
- {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Monitoring, SUSAR Reporting, Feasibility, Vendor Management, Clinical, Management & Monitoring, Site Start up, Services, Site Monitoring & Site, Management, Pharmacovigilance, Data Analytics","organisation_type":"Pharmaceutical company"}
- {"country":"India","full_name":"Accenture Solutions Private Limited","duties_or_roles":"Pharmacovigilance: Medical review & Cases, Data Entry.; Pharmacovigilance: Medical review & Cases, Data Entry.; Embarc operations; (also listed with code 6 duties)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"Investigator Recruitment, IVRS - tratment randomization, Subject Number Assignment, Treatment/Arm Assignment, Drug (re) Supplies Assignment","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"MLL Dx GmbH","duties_or_roles":"Biomarkers - genetics/genomics, FISH analyses","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Omnitrace Corp.","duties_or_roles":"Lost to Follow up Tracing","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"QPS LLC","duties_or_roles":"bioanalytical analysis, Nab","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Reblozyl 75 mg powder for solution for injection
- Active Substance
- LUSPATERCEPT
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- Subcutaneous
- Authorisation Status
- Authorised (marketing authorisation EU/1/20/1452/002)
- Orphan Designation
- Yes
- Starting Dose
- 1.75 mg/kg
- Dose Levels
- 1.75 mg/kg (highest starting dose stated)
- Maximum Dose
- 60.8 mg/kg (max total amount as listed)
- Investigational Product Name
- Reblozyl 25 mg powder for solution for injection
- Active Substance
- LUSPATERCEPT
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- Subcutaneous
- Authorisation Status
- Authorised (marketing authorisation EU/1/20/1452/001)
- Orphan Designation
- Yes
- Starting Dose
- 1.75 mg/kg
- Dose Levels
- 1.75 mg/kg (highest starting dose stated)
- Maximum Dose
- 60.8 mg/kg (max total amount as listed)
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