Clinical trial • Phase III • Haematology|Rare Disease

efanesoctocog alfa for Haemophilia A

Phase III trial of efanesoctocog alfa for Haemophilia A. open-label, none/not specified-controlled. 124 participants.

Overview

Trial Therapeutic Area
Haematology|Rare Disease
Trial Disease
Haemophilia A
Trial Stage
Phase III
Drug Modality
Peptide/protein/enzyme
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
04-12-2023
First CTIS Authorization Date
11-04-2024

Trial design

open-label, none/not specified-controlled Phase III trial in Germany, Slovenia, Netherlands and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
124

Eligibility

Recruits 124 paediatric patients.

Vulnerable Population
Parents’ or legally designated representatives’ consent is required for patients who are <18 years of age or unable to give consent, or as applicable per local laws, before any study-related activities are undertaken. Patients who are <18 years of age should provide assent in addition to the parents’/legally designated representatives’ consent, if appropriate.

Inclusion criteria

  • {"criterion_text":"- Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and protocol. Parents’ or legally designated representatives’ consent is required for patients who are <18 years of age or unable to give consent, or as applicable per local laws, before any study-related activities are undertaken. Patients who are <18 years of age should provide assent in addition to the parents’/legally designated representatives’ consent, if appropriate.\n- Contraceptive use by patients should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Males: No contraceptive measures required for this study.\n- Must have completed one of the required parent studies: LTS16294, Sobi.BIVV001-001, Sobi.BIVV001-003, or Sobi.BIVV001-004, and be receiving a clinical benefit from the efanesoctocog alfa treatment, as judged by the Investigator. The interval between the patient’s last study dose in the parent study and Visit 1 of this study should preferably be within 7 days for the LTS16294, Sobi.BIVV001-001 and Sobi.BIVV001-004 studies to ensure there is no interruption in the prophylaxis dosing with efanesoctocog alfa. Patients coming from the Sobi.BIVV001-003 study, should preferably enter this study at the EoS visit of that study.\n- Willingness and ability of the patient or parent or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study."}

Exclusion criteria

  • {"criterion_text":"- Positive inhibitor result defined as ≥0.6 Bethesda units (BU)/mL, at the Baseline Visit.\n- Ongoing or planned participation in any interventional clinical study at Baseline Visit.\n- Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Safety Endpoint: The occurrence of adverse events (AEs), including serious adverse events (SAEs) and AESIs","definition_or_measurement_approach":"Collection and reporting of adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs) as measures of safety and tolerability."}
  • {"endpoint_text":"- Efficacy Endpoints: To collect further data on the efficacy of efanesoctocog alfa as a prophylaxis treatment; To collect further data on the efficacy of efanesoctocog alfa in the treatment of bleeding episodes","definition_or_measurement_approach":"Annualised Bleeding Rate (ABR) for treated bleeds and all bleeds (including untreated bleeds, excluding surgical bleeds) by type and location during prophylactic treatment; number of injections and total dose required to treat a bleeding episode."}

Recruitment

Planned Sample Size
124
Recruitment Window Months
35
Consent Approach
Capable of giving signed informed consent is required. Parents’ or legally designated representatives’ consent is required for patients <18 years or unable to give consent; patients <18 years should provide assent in addition to parents'/legally designated representatives' consent where appropriate. Age-specific information/assent and ICF documents are provided (examples in repository include assent forms for 6-11 years, 12-16 years, 14-17 years, parent/holder of parental authority forms and adult ICFs). Consent/ICF materials are available in multiple languages as per submitted documents (examples: English, German, Spanish, Italian, French, Greek, Slovenian, Bulgarian, Dutch, Norwegian, Swedish).

Geography

Total Number Of Sites
32
Total Number Of Participants
124

Germany

Earliest CTIS Part Ii Submission Date
13-03-2024
Latest Decision Or Authorization Date
11-04-2024
Processing Time Days
29
Number Of Sites
2
Number Of Participants
7

Sites

Site Name
Universitaetsklinikum Bonn AöR
Department Name
Institute of Experimental Hematology and Transfusion Medicine
Principal Investigator Name
Johannes Oldenburg
Principal Investigator Email
johannes.oldenburg@ukbonn.de
Contact Person Name
Johannes Oldenburg
Contact Person Email
johannes.oldenburg@ukbonn.de
Site Name
Universitaetsklinikum Frankfurt AöR
Department Name
Hematology
Principal Investigator Name
Wolfgang Miesbach
Principal Investigator Email
wolfgang.miesbach@kgu.de
Contact Person Name
Wolfgang Miesbach
Contact Person Email
wolfgang.miesbach@kgu.de

Slovenia

Earliest CTIS Part Ii Submission Date
27-11-2024
Latest Decision Or Authorization Date
26-02-2025
Processing Time Days
91
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
University Medical Center Ljubljana
Department Name
Clinical Department of Haematology
Principal Investigator Name
Irena Preloznik Zupan
Principal Investigator Email
irena.zupan@kclj.si
Contact Person Name
Irena Preloznik Zupan
Contact Person Email
irena.zupan@kclj.si

Netherlands

Earliest CTIS Part Ii Submission Date
11-02-2025
Latest Decision Or Authorization Date
17-03-2025
Processing Time Days
34
Number Of Sites
2
Number Of Participants
7

Sites

Site Name
Universitair Medisch Centrum Utrecht
Department Name
Internal medecine
Principal Investigator Name
Paul Van Der Valk
Principal Investigator Email
p.r.vandervalk@umcutrecht.nl
Contact Person Name
Paul Van Der Valk
Contact Person Email
p.r.vandervalk@umcutrecht.nl
Site Name
Amsterdam UMC Stichting
Department Name
Pediatric Hematology
Principal Investigator Name
Karin Fijnvandraat
Principal Investigator Email
c.j.fijnvandraat@amsterdamumc.nl
Contact Person Name
Karin Fijnvandraat

France

Earliest CTIS Part Ii Submission Date
31-03-2025
Latest Decision Or Authorization Date
07-04-2025
Processing Time Days
7
Number Of Sites
8
Number Of Participants
28

Sites

Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Institut Coeur Poumon, Dpartment of Hemostasis and Transfusion
Principal Investigator Name
Sophie Susen
Principal Investigator Email
sophie.susen@chru-lille.fr
Contact Person Name
Sophie Susen
Contact Person Email
sophie.susen@chru-lille.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Site Hôpital des Enfants, Competence and Resources Center for Constitutional Hemorrhagic Disorders
Principal Investigator Name
Yoann Huguenin
Principal Investigator Email
yoann.huguenin@chu-bordeaux.fr
Contact Person Name
Yoann Huguenin
Contact Person Email
yoann.huguenin@chu-bordeaux.fr
Site Name
Hospices Civils De Lyon
Department Name
Site Louis Pradel, Department of Biological Hematology and Clinical Hemostasis
Principal Investigator Name
Sandrine Meunier
Principal Investigator Email
sandrine.meunier@chu-lyon.fr
Contact Person Name
Sandrine Meunier
Contact Person Email
sandrine.meunier@chu-lyon.fr
Site Name
Centre Hospitalier Regional Et Universitaire De Brest
Department Name
Site de la Cavale Blanche, Department of Hematology
Principal Investigator Name
Brigitte Pan-Petesch
Principal Investigator Email
brigitte.pan-petesch@chu-brest.fr
Contact Person Name
Brigitte Pan-Petesch
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Site Hautepierre, Department of Hematology and Oncology
Principal Investigator Name
Dominique Desprez Flor
Principal Investigator Email
dominique.desprez@chru-strasbourg.fr
Contact Person Name
Dominique Desprez Flor
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Hôpital Bicêtre, Reference Centre for Haemophilia
Principal Investigator Name
Roseline d'Oiron
Principal Investigator Email
roseline.doiron@aphp.fr
Contact Person Name
Roseline d'Oiron
Contact Person Email
roseline.doiron@aphp.fr
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Department of Pediatric Hematology, Immunology and Oncology
Principal Investigator Name
Hervé Chambost
Principal Investigator Email
herve.chambost@hp-hm.fr
Contact Person Name
Hervé Chambost
Contact Person Email
herve.chambost@hp-hm.fr
Site Name
Centre Hospitalier Regional De Brest (duplicate listing not specified)
Department Name
Department of Hematology (example site listings aggregated)

Greece

Earliest CTIS Part Ii Submission Date
27-11-2024
Latest Decision Or Authorization Date
10-03-2025
Processing Time Days
103
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
Nosokomeio Paidon I Agia Sofia
Department Name
Haemophilia Centre, Haemostasis and Thrombosis Unit
Principal Investigator Name
Helen Pergantou
Principal Investigator Email
hpergantou@gmail.com
Contact Person Name
Helen Pergantou
Contact Person Email
hpergantou@gmail.com
Site Name
Laiko General Hospital Of Athens
Department Name
Blood Transfusion Service and National Reference Centre for Congenital Bleeding Disorders
Principal Investigator Name
Olga Katsarou-Fasouli
Principal Investigator Email
olgkats@hotmail.com
Contact Person Name
Olga Katsarou-Fasouli
Contact Person Email
olgkats@hotmail.com

Norway

Earliest CTIS Part Ii Submission Date
12-09-2025
Latest Decision Or Authorization Date
18-09-2025
Processing Time Days
6
Number Of Sites
1
Number Of Participants
20

Sites

Site Name
Oslo University Hospital HF
Department Name
Department of Haematology
Principal Investigator Name
Pål Andrè Holme
Principal Investigator Email
pholme@ous-hf.no
Contact Person Name
Pål Andrè Holme
Contact Person Email
pholme@ous-hf.no

Italy

Earliest CTIS Part Ii Submission Date
11-01-2024
Latest Decision Or Authorization Date
15-04-2024
Processing Time Days
95
Number Of Sites
8
Number Of Participants
21

Sites

Site Name
Azienda Unita Locale Socio Sanitaria N 8 Berica
Department Name
UOC Hematology
Principal Investigator Name
Ilaria Nichele
Principal Investigator Email
ilaria.nichele@aulss8.veneto.it
Contact Person Name
Ilaria Nichele
Site Name
Azienda Ospedaliero Universitaria Careggi
Department Name
Department of Oncology, SOD Hemorrhagic and Coagulation Diseases
Principal Investigator Name
Giancarlo Castaman
Principal Investigator Email
castaman@aou-careggi.toscana.it
Contact Person Name
Giancarlo Castaman
Site Name
Humanitas Research Hospital
Department Name
Center for Thrombosis and Hemorrhagic Diseases
Principal Investigator Name
Maria Elisa Mancuso
Principal Investigator Email
mariaelisa.mancuso@humanitas.it
Contact Person Name
Maria Elisa Mancuso
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
Department of Clinical Medicine and Surgery
Principal Investigator Name
Matteo Di Minno
Principal Investigator Email
matteo.diminno@unina.it
Contact Person Name
Matteo Di Minno
Contact Person Email
matteo.diminno@unina.it
Site Name
Azienda Ospedaliero Universitaria Parma
Department Name
SSD Hub Center Hemophilia and Congenital Hemorrhagic Diseases
Principal Investigator Name
Antonio Coppola
Principal Investigator Email
ancoppola@ao.pr.it
Contact Person Name
Antonio Coppola
Contact Person Email
ancoppola@ao.pr.it
Site Name
Azienda Ospedaliero Universitaria Renato Dulbecco
Department Name
Department Hemato-Oncology
Principal Investigator Name
Rita Carlotta Santoro
Principal Investigator Email
ritacarlottasantoro@gmail.com
Contact Person Name
Rita Carlotta Santoro
Contact Person Email
ritacarlottasantoro@gmail.com
Site Name
Azienda Ospedaliero-Universitaria Policlinico Umberto I
Department Name
Department of Translational and Precision Medicine - Division of Hematology
Principal Investigator Name
Cristina Santoro
Principal Investigator Email
santoro@bce.uniromal.it
Contact Person Name
Cristina Santoro
Contact Person Email
santoro@bce.uniromal.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Complex Structure of Medicine Hemostasis and Thrombosis
Principal Investigator Name
Flora Peyvandi
Principal Investigator Email
Flora.peyvandi@unimi.it
Contact Person Name
Flora Peyvandi
Contact Person Email
Flora.peyvandi@unimi.it

Spain

Earliest CTIS Part Ii Submission Date
18-03-2024
Latest Decision Or Authorization Date
11-04-2024
Processing Time Days
24
Number Of Sites
3
Number Of Participants
10

Sites

Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Hematology
Principal Investigator Name
Saturnino Haya Guaita
Principal Investigator Email
haya_sat@gva.es
Contact Person Name
Saturnino Haya Guaita
Contact Person Email
haya_sat@gva.es
Site Name
Hospital Unviersitario Miguel Servet
Department Name
Hematology
Principal Investigator Name
José Manuel Calvo Villas
Principal Investigator Email
informacion.sector2@salud.aragon.es
Contact Person Name
José Manuel Calvo Villas
Site Name
Complexo Hospitalario Universitario A Coruna
Department Name
Hematology
Principal Investigator Name
Marta Fernandez Docampo
Principal Investigator Email
Marta.Fernandez.Docampo@sergas.es
Contact Person Name
Marta Fernandez Docampo

Bulgaria

Earliest CTIS Part Ii Submission Date
07-03-2025
Latest Decision Or Authorization Date
24-03-2025
Processing Time Days
17
Number Of Sites
2
Number Of Participants
9

Sites

Site Name
Specialized Hospital For Active Treatment Of Hematological Diseases EAD
Department Name
Third Department of Clinical Hematology at Clinical Hematology Clinic
Principal Investigator Name
Toshko Lissitchkov
Principal Investigator Email
t_lissitchkov@yahoo.com
Contact Person Name
Toshko Lissitchkov
Contact Person Email
t_lissitchkov@yahoo.com
Site Name
University Multiprofile Hospital For Active Treatment Saint Georgi EAD
Department Name
Clinical Hematology Clinic
Principal Investigator Name
Emil Spasov
Principal Investigator Email
dr-espassov@yahoo.com
Contact Person Name
Emil Spasov
Contact Person Email
dr-espassov@yahoo.com

Sweden

Earliest CTIS Part Ii Submission Date
07-07-2025
Latest Decision Or Authorization Date
29-07-2025
Processing Time Days
22
Number Of Sites
3
Number Of Participants
14

Sites

Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Department of Haematology and Coagulation Disorders
Principal Investigator Name
Fariba Baghaei
Principal Investigator Email
fariba.baghaei@vgregion.se
Contact Person Name
Fariba Baghaei
Contact Person Email
fariba.baghaei@vgregion.se
Site Name
Region Skane Skanes Universitetssjukhus
Department Name
Hematology and Vascular diseases
Principal Investigator Name
Jan Astermark
Principal Investigator Email
Jan.Astermark@med.lu.se
Contact Person Name
Jan Astermark
Contact Person Email
Jan.Astermark@med.lu.se
Site Name
Karolinska University Hospital
Department Name
Department of Haematology
Principal Investigator Name
Maria Magnusson
Principal Investigator Email
maria.s.magnusson@regionstockholm.se
Contact Person Name
Maria Magnusson

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
laboratory support; pharmacovigilance; statistics; translations; vendor management (as per contract); additional sponsor duties coded 1,11,12,2,5,6
Name
Psi CRO Greece
Responsibilities
sponsor duties coded 1 and 12
Name
Almac Clinical Services Limited
Responsibilities
QP batch release for IMP test; sponsor duties coded 14 and 15
Name
Almac Clinical Technologies LLC
Responsibilities
sponsor duties coded 3
Name
Almac Clinical Services (Ireland) Limited
Responsibilities
sponsor duties coded 14

Third parties

  • {"country":"Switzerland","full_name":"Psi Cro AG","duties_or_roles":"codes:1,11,12,15 (as per contract (laboratory support; pharmacovigilance; statistics, translations; vendor management)),2,5,6","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"Psi CRO Greece","duties_or_roles":"codes:1,12","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"code:7","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"codes:14,15 (value: QP batch release for IMP test)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"code:15 (value: Global reimbursement arrangement)","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"code:15 (value: Logistics, storage of samples and future potential testing)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Esoterix Inc.","duties_or_roles":"code:4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"code:3","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"Labcorp","duties_or_roles":"codes:15 (Kit building and Logistics),4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Ireland","full_name":"Almac Clinical Services (Ireland) Limited","duties_or_roles":"code:14","organisation_type":"Pharmaceutical company"}

Investigational products

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 number EU/1/24/1824/005 present
Orphan Designation
Yes
Starting Dose
50 IU/kg
Dose Levels
50 IU/kg
Frequency
once-weekly
Maximum Dose
50 IU/kg (max daily dose amount listed as 50 IU/kg)
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 number EU/1/24/1824/006 present
Orphan Designation
Yes
Starting Dose
50 IU/kg
Dose Levels
50 IU/kg
Frequency
once-weekly
Maximum Dose
50 IU/kg (max daily dose amount listed as 50 IU/kg)
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 number EU/1/24/1824/007 present
Orphan Designation
Yes
Starting Dose
50 IU/kg
Dose Levels
50 IU/kg
Frequency
once-weekly
Maximum Dose
50 IU/kg (max daily dose amount listed as 50 IU/kg)
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 number EU/1/24/1824/001 present
Orphan Designation
Yes
Starting Dose
50 IU/kg
Dose Levels
50 IU/kg
Frequency
once-weekly
Maximum Dose
50 IU/kg (max daily dose amount listed as 50 IU/kg)
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 number EU/1/24/1824/002 present (authorization country code NO for one pack)
Orphan Designation
Yes
Starting Dose
50 IU/kg
Dose Levels
50 IU/kg
Frequency
once-weekly
Maximum Dose
50 IU/kg (max daily dose amount listed as 50 IU/kg)
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 number EU/1/24/1824/004 present
Orphan Designation
Yes
Starting Dose
50 IU/kg
Dose Levels
50 IU/kg
Frequency
once-weekly
Maximum Dose
50 IU/kg (max daily dose amount listed as 50 IU/kg)

Related trials

Other published trials that may interest you.