Clinical trial • Not applicable • Haematology|Rare Disease
EFANESOCTOCOG ALFA for Haemophilia A
Not applicable trial of EFANESOCTOCOG ALFA for Haemophilia A. open-label, none/not specified-controlled. 175 participants.
Overview
- Trial Therapeutic Area
- Haematology|Rare Disease
- Trial Disease
- Haemophilia A
- Trial Stage
- Not applicable
- Drug Modality
- Peptide/protein/enzyme
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 20-12-2024
- First CTIS Authorization Date
- 28-04-2025
Trial design
open-label, none/not specified-controlled Not applicable trial across 33 sites in Czechia, Germany, Ireland and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 175
- Trial Duration For Participant
- 1095
Eligibility
Recruits 175 paediatric patients.
- Vulnerable Population
- The trial includes vulnerable populations (minors). Assent and age-specific information/consent materials are provided: assent forms for ages 6-11, 12-14, and 15-17 years and parental/legal representative informed consent forms. The protocol requires capability to give signed informed consent for adults; for minors parental/legal representative consent and age-appropriate assent documents are used (multiple ICF/assent templates exist).
Inclusion criteria
- {"criterion_text":"- 1. Male or female patient with a diagnosis of haemophilia A."}
- {"criterion_text":"- 2. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol."}
- {"criterion_text":"- 3. Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations."}
- {"criterion_text":"- 4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment."}
- {"criterion_text":"- 5. Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study."}
- {"criterion_text":"- 6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs)."}
- {"criterion_text":"- 7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes."}
- {"criterion_text":"- 8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees)."}
Exclusion criteria
- {"criterion_text":"- 1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A"}
- {"criterion_text":"- 2. Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit [BU]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit."}
- {"criterion_text":"- 3. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study."}
- {"criterion_text":"- 4. Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Annualised joint bleeding rate (AjBR) over the prospective period","definition_or_measurement_approach":"Annualised joint bleeding rate (AjBR) measured over the prospective observation period (rate of joint bleeding events per year during prospective follow-up)."}
Secondary endpoints
- {"endpoint_text":"- 1. Annual Bleeding Rate over the prospective observation period","definition_or_measurement_approach":"Annual bleeding rate calculated as the number of bleeding events per year during the prospective observation period."}
- {"endpoint_text":"- 2. Number of injections and total dose to control a bleeding event over the prospective observation period","definition_or_measurement_approach":"Count of injections administered and the cumulative dose used to control each bleeding event during the prospective observation period (number and total IU/dose per event)."}
- {"endpoint_text":"- 3. Change in HJHS from enrolment to Months 12, 24 and 36","definition_or_measurement_approach":"Change in Hemophilia Joint Health Score (HJHS) from baseline (enrolment) to months 12, 24 and 36, using the standard HJHS assessment."}
- {"endpoint_text":"- 4. Target joint development, resolution, and recurrency on a patient and joint level during the period on efanesoctocog alfa","definition_or_measurement_approach":"Assessment of development, resolution and recurrence of target joints at both patient and joint level during treatment with efanesoctocog alfa (tracking target joint status over follow-up)."}
Recruitment
- Planned Sample Size
- 175
- Recruitment Window Months
- 42
- Consent Approach
- Adults provide written informed consent (ICF). Minors provide age-appropriate assent with parental/legal representative consent; specific assent/ICF documents exist for ages 6-11, 12-14, and 15-17, plus parental information/consent forms and pregnancy follow-up forms. Multiple language versions of participant information/assent/ICF are available (examples in file list include EN, DE, CZ, ES, FR, SV, HR, IT).
Geography
- Total Number Of Sites
- 33
- Total Number Of Participants
- 175
Czechia
- Earliest CTIS Part Ii Submission Date
- 04-03-2025
- Latest Decision Or Authorization Date
- 29-04-2025
- Processing Time Days
- 56
- Number Of Sites
- 3
- Number Of Participants
- 18
Sites
- Site Name
- Fakultni Nemocnice V Motole
- Department Name
- Klinika dětské hematologie a onkologie
- Contact Person Name
- Ester Zápotocká
- Contact Person Email
- ester.zapotocka@fnmotol.cz
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Oddělení klinické hematologie
- Contact Person Name
- Petr Smejkal
- Contact Person Email
- smejkal.petr@fnbrno.cz
- Site Name
- Fakultni Nemocnice Ostrava
- Department Name
- Ambulance krevního centra
- Contact Person Name
- Radomíra Hrdličková
- Contact Person Email
- radomira.hrdlickova@fno.cz
Germany
- Earliest CTIS Part Ii Submission Date
- 31-03-2025
- Latest Decision Or Authorization Date
- 30-04-2025
- Processing Time Days
- 30
- Number Of Sites
- 5
- Number Of Participants
- 35
Sites
- Site Name
- HZRM Haemophilie-Zentrum Rhein Main GmbH
- Department Name
- Director of Haemophilia Centre Rhein Main – HZRM, Frankfurt
- Contact Person Name
- Carmen Escuriola-Ettingshausen
- Contact Person Email
- Carmen.escuriola@hzrm.de
- Site Name
- Universitaetsklinikum Bonn AöR
- Department Name
- Director of Institute of Experimental Haematology and Transfusions Medicine
- Contact Person Name
- Johannes Oldenburg
- Contact Person Email
- johannes.oldenburg@ukbonn.de
- Site Name
- Klinikum der Universitaet Muenchen AöR
- Department Name
- Head of Paediatric Haemostaseology and Haemophilia Centre
- Contact Person Name
- Martin Olivieri
- Contact Person Email
- martin.olivieri@med.uni-muenchen.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Head Outpatient Clinics Pediatric Hematology and Oncology
- Contact Person Name
- Susanne Holzhauer
- Contact Person Email
- Susanne.Holzhauer@charite.de
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- Head of the haemostaseology department
- Contact Person Name
- Florian Langer
- Contact Person Email
- langer@uke.de
Ireland
- Earliest CTIS Part Ii Submission Date
- 26-03-2025
- Latest Decision Or Authorization Date
- 02-05-2025
- Processing Time Days
- 37
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- Children's Health Ireland
- Department Name
- Haematology Department
- Contact Person Name
- Elizabeth Mary (Beatrice) Nolan
- Contact Person Email
- Beatrice.nolan@olchc.ie
Spain
- Earliest CTIS Part Ii Submission Date
- 17-02-2025
- Latest Decision Or Authorization Date
- 29-04-2025
- Processing Time Days
- 71
- Number Of Sites
- 7
- Number Of Participants
- 35
Sites
- Site Name
- University Hospital Son Espases
- Department Name
- Hematology
- Contact Person Name
- Mariana Canaro Hirnyk
- Contact Person Email
- huse.comunicacio@ssib.es
- Site Name
- Hospital Universitario Miguel Servet
- Department Name
- Hematology
- Contact Person Name
- José Manuel Calvo Villas
- Contact Person Email
- informacion.sector2@salud.aragon.es
- Site Name
- Complexo Hospitalario Universitario A Coruna
- Department Name
- Hematology
- Contact Person Name
- Marta Fernandez Docampo
- Contact Person Email
- Marta.Fernandez.Docampo@sergas.es
- Site Name
- Hospital De La Santa Creu I Sant Pau
- Department Name
- Hematology
- Contact Person Name
- José Mateo Arranz
- Contact Person Email
- jmateo@santpau.cat
- Site Name
- Hospital Universitario De Salamanca
- Department Name
- Hematology
- Contact Person Name
- José Maria Bastida Bermejo
- Contact Person Email
- jmbastida@saludcastillayleon.es
- Site Name
- Hospital Universitario Regional De Malaga
- Department Name
- Hematology
- Contact Person Name
- Francisco José Lopez Jaime
- Contact Person Email
- estudios.clinicos@ibima.eu
- Site Name
- Hospital Universitario De Salamanca (duplicate entry not expected but listed above)
- Department Name
- Hematology
Sweden
- Earliest CTIS Part Ii Submission Date
- 31-03-2025
- Latest Decision Or Authorization Date
- 29-04-2025
- Processing Time Days
- 29
- Number Of Sites
- 2
- Number Of Participants
- 12
Sites
- Site Name
- Karolinska University Hospital
- Department Name
- Department of Hematology/Coagulation Unit
- Contact Person Name
- Maria Magnusson
- Contact Person Email
- maria.s.magnusson@regionstockholm.se
- Site Name
- Sahlgrenska University Hospital-Vaestra Goetalandsregionen
- Department Name
- Department of Hematology and Coagulation Disorders
- Contact Person Name
- Anna Olsson
- Contact Person Email
- anna.el.olsson@vgregion.se
Croatia
- Earliest CTIS Part Ii Submission Date
- 30-07-2025
- Latest Decision Or Authorization Date
- 29-08-2025
- Processing Time Days
- 30
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- University Hospital Centre Zagreb
- Department Name
- Department of internal medicine
- Contact Person Name
- Ana Boban
- Contact Person Email
- ana.boban@kbc-zagreb.hr
France
- Earliest CTIS Part Ii Submission Date
- 25-02-2025
- Latest Decision Or Authorization Date
- 28-04-2025
- Processing Time Days
- 62
- Number Of Sites
- 7
- Number Of Participants
- 35
Sites
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Paediatric Haematology, Immunology and Oncology Department
- Contact Person Name
- Hervé Chambost
- Contact Person Email
- herve.chambost@ap-hm.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Adult Hematology Department, Necker-Enfants Malades Hospital
- Contact Person Name
- Laurent Frenzel
- Contact Person Email
- laurent.frenzel@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- Site Hôtel Dieu, Haemostasis Center
- Contact Person Name
- Nicolas Drillaud
- Contact Person Email
- nicolas.drillaud@chu-nantes.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Site Tripode, Bordeaux skills and resources center for constitutional hemorrhagic disorders
- Contact Person Name
- Sabine-Marie Castet
- Contact Person Email
- sabine-marie.castet@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Universitaire De Rennes
- Department Name
- Site Pontchaillou, Treatment Centre for Haemorrhagic Diseases
- Contact Person Name
- Sophie Bayart
- Contact Person Email
- sophie.bayart@chu-rennes.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Arnaud de Villeneuve Hospital, Biological Hematology Department
- Contact Person Name
- Christine Biron-Andreani
- Contact Person Email
- c-biron@chu-montpellier.fr
- Site Name
- CHU de Rouen - Hôpital Charles Nicolle
- Department Name
- Haemophilia Centre
- Contact Person Name
- Virginie Barbay
- Contact Person Email
- virginie.barbay@chu-rouen.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 05-05-2025
- Latest Decision Or Authorization Date
- 02-07-2025
- Processing Time Days
- 58
- Number Of Sites
- 7
- Number Of Participants
- 29
Sites
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
- Department Name
- Department of Diagnostics SSD Maternal, Infant and Traumatology Transfusion Medicine
- Contact Person Name
- Berardino Pollio
- Contact Person Email
- bpollio@cittadellasalute.to.it
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- Medical Clinic II, Hemophilia Center
- Contact Person Name
- Ezio Zanon
- Contact Person Email
- ezio.zanon@unipd.it
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino (second site listing)
- Department Name
- Homeostasis and Thrombosis Center/Haematology Unit
- Contact Person Name
- Alessandra Borchiellini
- Contact Person Email
- aborchiellini@cittadellasalute.to.it
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
- Department Name
- Hematology Unit with BMT
- Contact Person Name
- Gaetano Giuffrida
- Contact Person Email
- gaegiuffrida@gmail.com
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Centre for Haemorrhagic and Thrombotic Diseases
- Contact Person Name
- Raimondo De Cristofaro
- Contact Person Email
- Raimondo.decristofaro@policlinicogemelli.it
- Site Name
- Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d'Aragona
- Department Name
- Center for Congenital Hemorrhagic Diseases and Thrombotic Diseases
- Contact Person Name
- Anna Guida
- Contact Person Email
- ambulatoriomecsalerno@gmail.com
- Site Name
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
- Department Name
- SSD Congenital Hemorragic Diseases
- Contact Person Name
- Leila Valdrè
- Contact Person Email
- LELIA.VALDRE@AOSP.80.IT
Sponsor
Primary sponsor
- Full Name
- Swedish Orphan Biovitrum AB (publ)
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Sweden
Contract research organisations
- Name
- Psi Cro AG
- Responsibilities
- [1,11,12,2,5,6,8,9]
Third parties
- {"country":"Switzerland","full_name":"Psi Cro AG","duties_or_roles":"[1,11,12,2,5,6,8,9]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"[{\"code\":\"15\",\"value\":\"eTMF\"},{\"code\":\"7\"}]","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ALTUVOCT 4 000 IU powder and solvent for solution for injection
- Active Substance
- EFANESOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS INJECTION
- Route
- INTRAVENOUS INJECTION
- Authorisation Status
- Marketing authorisation (MA number EU/1/24/1824/007) indicated
- Orphan Designation
- Yes
- Investigational Product Name
- ALTUVOCT 2 000 IU powder and solvent for solution for injection
- Active Substance
- EFANESOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS INJECTION
- Route
- INTRAVENOUS INJECTION
- Authorisation Status
- Marketing authorisation (MA number EU/1/24/1824/005) indicated
- Orphan Designation
- Yes
- Investigational Product Name
- ALTUVOCT 500 IU powder and solvent for solution for injection
- Active Substance
- EFANESOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS INJECTION
- Route
- INTRAVENOUS INJECTION
- Authorisation Status
- Marketing authorisation (MA number EU/1/24/1824/002) indicated
- Orphan Designation
- Yes
- Investigational Product Name
- ALTUVOCT 1 000 IU powder and solvent for solution for injection
- Active Substance
- EFANESOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS INJECTION
- Route
- INTRAVENOUS INJECTION
- Authorisation Status
- Marketing authorisation (MA number EU/1/24/1824/004) indicated
- Orphan Designation
- Yes
- Investigational Product Name
- ALTUVOCT 3 000 IU powder and solvent for solution for injection
- Active Substance
- EFANESOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS INJECTION
- Route
- INTRAVENOUS INJECTION
- Authorisation Status
- Marketing authorisation (MA number EU/1/24/1824/006) indicated
- Orphan Designation
- Yes
- Investigational Product Name
- ALTUVOCT 250 IU powder and solvent for solution for injection
- Active Substance
- EFANESOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS INJECTION
- Route
- INTRAVENOUS INJECTION
- Authorisation Status
- Marketing authorisation (MA number EU/1/24/1824/001) indicated
- Orphan Designation
- Yes
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