Clinical trial • Phase III • Haematology
Emicizumab for Von Willebrand disease type 3
Phase III trial of Emicizumab for Von Willebrand disease type 3.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Von Willebrand disease type 3
- Trial Stage
- Phase III
- Drug Modality
- Bispecific antibody
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 16-01-2025
- First CTIS Authorization Date
- 05-05-2025
Trial design
Randomised, open-label, on-demand and/or prophylactic standard of care (soc) products used as comparators per local label. comparator products listed include wilate (500 iu and 1000 iu vwf/fviii formulations), fanhdi 500 iu, willfact 2000 iu (vwf), elocta (multiple iu strengths), novoseven (1 mg, 2 mg, 5 mg, 8 mg), voncento (various fviii/vwf combinations), haemate-p (various iu), veyvondi (650 iu, 1300 iu), feiba 50 u/ml (bypassing agent), advate (octocog alfa) as auxiliary—administered intravenously per local approved product labeling; arm b uses on-demand soc for 24 weeks then switch to emicizumab prophylaxis in extension.-controlled Phase III trial in Belgium, France, Germany and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- On-demand and/or prophylactic standard of care (SOC) products used as comparators per local label. Comparator products listed include Wilate (500 IU and 1000 IU VWF/FVIII formulations), Fanhdi 500 IU, Willfact 2000 IU (vWF), ELOCTA (multiple IU strengths), NovoSeven (1 mg, 2 mg, 5 mg, 8 mg), Voncento (various FVIII/VWF combinations), Haemate-P (various IU), Veyvondi (650 IU, 1300 IU), FEIBA 50 U/ml (bypassing agent), ADVATE (octocog alfa) as auxiliary—administered intravenously per local approved product labeling; Arm B uses on-demand SOC for 24 weeks then switch to emicizumab prophylaxis in extension.
- Real World Control
- Yes
- Target Sample Size
- 32
Eligibility
Recruits 32 paediatric patients.
- Vulnerable Population
- Vulnerable populations are included (isVulnerablePopulationSelected = true). The study includes infants and children (Age ≥ 1 month for Arms A and B; Age ≥ 2 years for Arm C). Informed consent is required from the participant or the participant's parent/legal guardian as applicable; age-appropriate assent and authorization documents are provided (assent and authorization forms for infants/children and parent/caregiver ICFs). Multiple age-specific ICF/assent documents are listed (e.g. Infant Authorization forms, Assent Forms ages 3-6, 7-11, 12-17, parent/caregiver ICFs), indicating assent/consent handling for minors and infant authorization procedures.
Inclusion criteria
- {"criterion_text":"- Age ≥ 2 years at the time of signing Informed Consent/Assent Form (Arm C)\n- Adequate hematological function\n- Documented exposure to on-demand and prophylactic SOC\n- For patient with childbearing potential; agreement to adhere to the contraception requirements\n- Confirmed diagnosis of Type 3 VWD, based on evaluation of VWF and FVIII levels\n- Age ≥ 1 month at the time of signing Informed Consent/Assent Form (Arms A and B)"}
Exclusion criteria
- {"criterion_text":"- Inherited or acquired bleeding disorder other than Congenital Type 3 VWD\n- History of intracranial hemorrhage\n- Previous or current treatment for thromboembolic disease or signs of thromboembolic disease\n- Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis\n- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number of treated bleeds over time","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Number of all bleeds over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Number of treated spontaneous bleeds over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Number of treated joint bleeds over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence and severity of adverse events","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence and severity of thromboembolic events","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence and severity of thrombotic microangiopathy events","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence and severity of injection-site reactions","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence of adverse events leading to drug discontinuation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions","definition_or_measurement_approach":""}
- {"endpoint_text":"- Changes from baseline in physical examination findings, vital signs, and ECG parameters","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence of laboratory abnormalities","definition_or_measurement_approach":""}
- {"endpoint_text":"- Trough plasma concentration of emicizumab","definition_or_measurement_approach":""}
- {"endpoint_text":"- Prevalence and incidence of ADAs to emicizumab","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change from baseline in Patient-Reported Outcomes Measurement Information System-29 (PROMIS-29)","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 32
- Recruitment Window Months
- 42
- Consent Approach
- Informed consent is obtained from the participant or the participant's parent/legal guardian as applicable. Age-specific assent and authorization documents are provided (assent forms for ages 3-6, 7-11, 12-17; Infant Authorization forms; parent/caregiver ICFs; PPA documents). Study documents and translations are available in multiple languages (documents and translations shown in English, French, Dutch, Spanish and Polish among others), indicating materials are provided in language-appropriate versions for participating countries.
Geography
- Total Number Of Sites
- 14
- Total Number Of Participants
- 47
Belgium
- Earliest CTIS Part Ii Submission Date
- 02-04-2025
- Latest Decision Or Authorization Date
- 03-02-2026
- Processing Time Days
- 307
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- UZ Leuven
- Department Name
- Thrombosis and Haemostasis (Cardiovascular diseases)
- Contact Person Name
- Quentin Van Thillo
- Contact Person Email
- quentin.vanthillo@uzleuven.be
- Number Of Participants
- 5
France
- Earliest CTIS Part Ii Submission Date
- 10-04-2025
- Latest Decision Or Authorization Date
- 20-02-2026
- Processing Time Days
- 316
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Service d'hématologie adulte
- Contact Person Name
- Clarisse CAZELLES
- Contact Person Email
- clarisse.cazelles@aphp.fr
- Number Of Participants
- 2
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Hémostase Clinique
- Contact Person Name
- Sophie SUSEN
- Contact Person Email
- Sophie.SUSEN@chu-lille.fr
- Number Of Participants
- 2
Germany
- Earliest CTIS Part Ii Submission Date
- 07-04-2025
- Latest Decision Or Authorization Date
- 04-02-2026
- Processing Time Days
- 303
- Number Of Sites
- 3
- Number Of Participants
- 13
Sites
- Site Name
- Gerinnungszentrum Rhein-Ruhr
- Department Name
- Gerinnungszentrum Rhein-Ruhr
- Contact Person Name
- Susan Halimeh
- Contact Person Email
- susan.halimeh@gzrr.de
- Number Of Participants
- ?
- Site Name
- Universitaetsklinikum Frankfurt AöR
- Department Name
- Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
- Contact Person Name
- Wolfgang Miesbach
- Contact Person Email
- miesbach@em.uni-frankfurt.de
- Number Of Participants
- ?
- Site Name
- Universitaetsklinikum Bonn AöR
- Department Name
- Institut für Experimentelle Hämatologie und Transfusionsmedizin Gebäude B 42
- Contact Person Name
- Johannes Oldenburg
- Contact Person Email
- Johannes.Oldenburg@ukbonn.de
- Number Of Participants
- ?
Italy
- Earliest CTIS Part Ii Submission Date
- 29-01-2025
- Latest Decision Or Authorization Date
- 04-02-2026
- Processing Time Days
- 371
- Number Of Sites
- 3
- Number Of Participants
- 6
Sites
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico Umberto I
- Department Name
- Divisione di Ematologia
- Contact Person Name
- Cristina Santoro
- Contact Person Email
- santoro@bce.uniromaI.it
- Number Of Participants
- 2
- Site Name
- Azienda Ospedaliero Universitaria Careggi
- Department Name
- Centro Malattie Emorragiche e della Coagulazione
- Contact Person Name
- Giancarlo Castaman
- Contact Person Email
- castaman@aou-careggi.toscana.it
- Number Of Participants
- 2
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- S.C. Medicina Emostasi e Trombosi
- Contact Person Name
- Flora Peyvandi
- Contact Person Email
- Flora.peyvandi@unimi.it
- Number Of Participants
- 2
Spain
- Earliest CTIS Part Ii Submission Date
- 14-02-2025
- Latest Decision Or Authorization Date
- 05-02-2026
- Processing Time Days
- 356
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Hematology
- Contact Person Name
- Ramiro José Núñez Vázquez
- Contact Person Email
- ramirojosenv@gmail.com
- Number Of Participants
- ?
- Site Name
- Hospital Universitario La Paz
- Department Name
- Hematology
- Contact Person Name
- Víctor Jiménez Yuste
- Contact Person Email
- vjyuste@gmail.com
- Number Of Participants
- ?
Poland
- Earliest CTIS Part Ii Submission Date
- 22-04-2025
- Latest Decision Or Authorization Date
- 04-02-2026
- Processing Time Days
- 288
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Instytut Hematologii I Transfuzjologii
- Department Name
- Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
- Contact Person Name
- Jerzy Windyga
- Contact Person Email
- sekretariatkzh@ihit.waw.pl
- Number Of Participants
- 3
Sweden
- Earliest CTIS Part Ii Submission Date
- 07-04-2025
- Latest Decision Or Authorization Date
- 04-02-2026
- Processing Time Days
- 303
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Sahlgrenska University Hospital-Vaestra Goetalandsregionen
- Department Name
- Koagulationscentrum Sahlgrenska
- Contact Person Name
- Anna Olsson
- Contact Person Email
- anna.el.olsson@vgregion.se
- Number Of Participants
- 4
Netherlands
- Earliest CTIS Part Ii Submission Date
- 25-04-2025
- Latest Decision Or Authorization Date
- 04-02-2026
- Processing Time Days
- 285
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Hematologie
- Contact Person Name
- Frank Leebeek
- Contact Person Email
- f.leebeek@erasmusmc.nl
- Number Of Participants
- 5
Sponsor
Primary sponsor
- Full Name
- F. Hoffmann-La Roche AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Almac Clinical Technologies LLC
- Responsibilities
- IxRS Provider
- Name
- Eresearchtechnology Inc.
- Responsibilities
- Clinical Outcome Assessment (eCOA, ePRO) Provider
- Name
- Medpace Reference Laboratories LLC
- Responsibilities
- Analysis lab for PD samples
- Name
- Labcorp Central Laboratory Services LP
- Responsibilities
- Central Laboratory Provider
- Name
- BioAgilytix Europe GmbH
- Responsibilities
- Analysis lab nAB
- Name
- QPS Netherlands B.V.
- Responsibilities
- PK and ADA analysis lab
Third parties
- {"country":"Germany","full_name":"BioAgilytix Europe GmbH","duties_or_roles":"Analysis lab nAB","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"IxRS Provider","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Clinical Outcome Assessment (eCOA, ePRO) Provider","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Analysis lab for PD samples","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Central Laboratory Provider","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"QPS Netherlands B.V.","duties_or_roles":"PK and ADA analysis lab","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Hemlibra 150 mg/mL solution for injection
- Active Substance
- Emicizumab
- Modality
- Bispecific antibody
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Authorisation Status
- Authorised
- Investigational Product Name
- Wilate 1000, 1000 IU VWF/1000 IUFVIII
- Active Substance
- Human coagulation factor VIII; Human von Willebrand factor
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- Wilate 500, 500 IU VWF/500 IUFVIII
- Active Substance
- Human coagulation factor VIII; Human von Willebrand factor
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- Fanhdi 500 IU
- Active Substance
- Human coagulation factor VIII
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- Willfact 2000 IU
- Active Substance
- Human von Willebrand factor
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- ELOCTA (multiple strengths)
- Active Substance
- Efmoroctocog alfa
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- NovoSeven (1 mg, 2 mg, 5 mg, 8 mg)
- Active Substance
- Eptacog alfa (activated)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- Voncento (various FVIII/VWF combinations)
- Active Substance
- Human coagulation factor VIII; Human von Willebrand factor
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- Haemate-P (various IU)
- Active Substance
- Human von Willebrand factor
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous/Infusion
- Route
- Intravenous/Infusion
- Authorisation Status
- Authorised
- Investigational Product Name
- VEYVONDI (650 IU, 1300 IU)
- Active Substance
- Vonicog alfa
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- FEIBA 50 U/ml
- Active Substance
- Factor VIII inhibitor bypassing fraction (mixture)
- Modality
- Other
- Routes Of Administration
- Intravenous/Infusion
- Route
- Intravenous/Infusion
- Authorisation Status
- Authorised
- Investigational Product Name
- ADVATE 1500 IU (and other ADVATE strengths)
- Active Substance
- Octocog alfa
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised
Related trials
Other published trials that may interest you.
- (S)-4,5-DIHYDRO-2-[2-HYDROXY-4-(3,6-DIOXAHEPTYLOXY)PHENYL]-4-METHYL-4-THIAZOLECARBOXYLIC ACID for Transfusion-dependent alpha thalassemia | Transfusion-dependent beta thalassemia | Low-risk myelodysplastic syndromes
- Luspatercept for Myelofibrosis | Anemia associated with myeloproliferative neoplasm-associated myelofibrosis
- GIVINOSTAT for Chronic myeloproliferative neoplasm
- GOLCADOMIDE for Follicular lymphoma (advanced stage)
- ISATUXIMAB for Acute lymphoblastic leukaemia | T-lymphoblastic lymphoma