Clinical trial • Phase IV • Haematology
SIMOCTOCOG ALFA for Haemophilia A
Phase IV trial of SIMOCTOCOG ALFA for Haemophilia A. None/Not specified-controlled. 101 participants.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Haemophilia A
- Trial Stage
- Phase IV
- Drug Modality
- Bispecific antibody|Peptide/protein/enzyme
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 30-07-2024
- First CTIS Authorization Date
- 27-08-2024
Trial design
None/Not specified-controlled Phase IV trial across 14 sites in Germany, Norway, Sweden and others.
- Comparator
- None/Not specified
- Target Sample Size
- 101
Eligibility
Recruits 101 paediatric patients.
- Vulnerable Population
- Participants may be of any age (including children). The protocol requires that participants or the participants’ parent(s)/legal guardian(s) be capable of giving signed informed consent and understanding trial documents. Age-specific assent and consent documents are provided (children assent and teenager assent forms and parent/guardian information/ICF documents are included in the submitted documentation), indicating that minors will provide assent appropriate to age bands and parents/guardians provide consent.
Inclusion criteria
- {"criterion_text":"- 1.\tParticipants can be of any age at the time of enrolment into the trial."}
- {"criterion_text":"- 2.\tMale persons with hemophilia A (HA), of any severity, who have a historical inhibitor titre ≥ 0.6 BU/mL, including those who have failed previous ITI attempt(s)"}
- {"criterion_text":"- 3.\tPersons undergoing ITI with Nuwiq®, octanate® or wilate® or undergoing ITI with Nuwiq®, octanate® or wilate® and receiving prophylactic therapy with emicizumab, aPCC or rFVIIa"}
- {"criterion_text":"- 4.\tParticipants or participants’ parent(s)/legal guardian(s) must be capable of giving signed informed consent and be able to understand the trial documents"}
Exclusion criteria
- {"criterion_text":"- 1.\tParticipants are excluded from the trial if any coagulation disorder other than HA is diagnosed"}
- {"criterion_text":"- 2.\tPartly retrospective patients will be excluded if detailed documentation on treatment, all BEs, inhibitor titres and FVIII levels is not available for the retrospective period"}
Endpoints
Primary endpoints
- {"endpoint_text":"- The primary endpoint in Group 1 and Group 2 is to evaluate ITI outcome as determined by achievement of the following ITI criteria: 1.\tInhibitor titre < 0.6 BU/mL for at least 2 consecutive measurements 2.\tFVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg BW 3.\tFVIII half-life ≥ 6 h","definition_or_measurement_approach":"Measured laboratory criteria: serial inhibitor titre measurements (Bethesda units) showing <0.6 BU/mL on at least two consecutive measurements; FVIII recovery assessed relative to predefined reference value (≥66% of 1.5% IU/kg BW); FVIII half-life measured and confirmed ≥ 6 hours."}
- {"endpoint_text":"- The primary endpoint for Group 3 is to evaluate the ABR compared with the ABR in Group 1 and Group 2.","definition_or_measurement_approach":"Annualised bleeding rate (ABR) calculated for participants in Group 3 and compared against ABR in Groups 1 and 2 (standard ABR calculation over observation period)."}
Secondary endpoints
- {"endpoint_text":"- For participants in Group 1 and Group 2: 1.\tTime to achieve ITI outcome 2.\tUse of emicizumab, aPCC, rFVIIa during ITI 3.\tRate of FVIII inhibitor relapses during a follow-up period in participants who have achieved complete ITI success","definition_or_measurement_approach":"Time-to-event analysis for time to achieve ITI outcome; recording and summarising use of emicizumab, aPCC and rFVIIa during ITI; tracking and reporting relapses of FVIII inhibitors during follow-up in participants who achieved complete ITI success."}
- {"endpoint_text":"- For participants in all 3 groups: 1.\tFrequency and severity of all bleeding episodes (BEs), all treated BEs, all spontaneous BEs, all joint BEs, and target joint BEs over time (≥ 3 bleeds in the same joint within 24 weeks) 2.\tNumber of infusions required to control BEs 3.\tFrequency and severity of bleeding during and after surgical procedures","definition_or_measurement_approach":"Bleeding episodes captured and categorised (treated, spontaneous, joint, target joint); frequency and severity summarised over time; count of infusions required to control BEs and collection of bleeding events related to surgical procedures."}
- {"endpoint_text":"- For participants in all 3 groups: 4.\tProportion of participants experiencing ADRs 5.\tThrombotic events (location, treatment, outcome) 6.\tTreatment costs","definition_or_measurement_approach":"Safety endpoints: proportion with adverse drug reactions (ADRs), documentation of thrombotic events with location/treatment/outcome; health-economic data collection for treatment costs."}
Recruitment
- Planned Sample Size
- 101
- Recruitment Window Months
- 110
- Consent Approach
- Informed consent is required from participants or, for minors, from parent(s)/legal guardian(s) (inclusion criterion: participants or participants’ parent(s)/legal guardian(s) must be capable of giving signed informed consent and understanding trial documents). Age-appropriate assent forms and parent/guardian information/ICF documents are provided (children assent and teenager assent forms and parent/guardian ICFs are included among submitted documents), indicating age-specific consent/assent handling across multiple paediatric age bands.
Geography
- Total Number Of Sites
- 14
- Total Number Of Participants
- 22
Germany
- Earliest CTIS Part Ii Submission Date
- 20-08-2024
- Latest Decision Or Authorization Date
- 11-07-2025
- Processing Time Days
- 325
- Number Of Sites
- 5
- Number Of Participants
- 6
Sites
- Site Name
- Gerinnungszentrum Rhein-Ruhr Aerztepartnerschaft Dr. med. Hannelore Rott Fachaerztin fuer Transfusionsmedizin Haemostaseologie Dr. med. Susan Halimeh Fachaerztin fuer Transfusionsmedizin Haemostaseologie Dr. med. Guenther Kappert Facharzt fuer Laboratoriumsmedizin Haemostaseologie
- Principal Investigator Name
- Susan Halimeh
- Principal Investigator Email
- Susan.halimeh@gzrr.de
- Contact Person Name
- Susan Halimeh
- Contact Person Email
- Susan.halimeh@gzrr.de
- Site Name
- Universitaetsklinikum Muenster AöR
- Department Name
- Klinik für Kinder- und Jugendmedizin
- Principal Investigator Name
- Meike Nowacki
- Principal Investigator Email
- meike.nowacki@ukmuenster.de
- Contact Person Name
- Meike Nowacki
- Contact Person Email
- meike.nowacki@ukmuenster.de
- Site Name
- HZRM Haemophilie-Zentrum Rhein Main GmbH
- Principal Investigator Name
- Carmen Escuriola
- Principal Investigator Email
- carmen.escuriola@hzrm.de
- Contact Person Name
- Carmen Escuriola
- Contact Person Email
- carmen.escuriola@hzrm.de
- Site Name
- Justus-Liebig-Universitaet Giessen
- Department Name
- Sektion Haemostaseologie
- Principal Investigator Name
- Ulrich Sachs
- Principal Investigator Email
- ulrich.sachs@med.uni-giessen.de
- Contact Person Name
- Ulrich Sachs
- Contact Person Email
- ulrich.sachs@med.uni-giessen.de
- Site Name
- Universitaetsklinikum Bonn AöR
- Department Name
- Insititut für Experimentelle Haematologie und Transfusionsmedizin (IHT)
- Principal Investigator Name
- Johannes Oldenburg
- Principal Investigator Email
- johannes.oldenburg@ukbonn.de
- Contact Person Name
- Johannes Oldenburg
- Contact Person Email
- johannes.oldenburg@ukbonn.de
Norway
- Earliest CTIS Part Ii Submission Date
- 20-08-2024
- Latest Decision Or Authorization Date
- 09-07-2025
- Processing Time Days
- 323
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Oslo University Hospital HF
- Department Name
- Center for rare diagnostics
- Principal Investigator Name
- Heidi Glosli
- Principal Investigator Email
- hglosli@ous-hf.no
- Contact Person Name
- Heidi Glosli
- Contact Person Email
- hglosli@ous-hf.no
Sweden
- Earliest CTIS Part Ii Submission Date
- 20-08-2024
- Latest Decision Or Authorization Date
- 08-07-2025
- Processing Time Days
- 322
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Region Skane Skanes Universitetssjukhus
- Department Name
- Department of hematology
- 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
Finland
- Earliest CTIS Part Ii Submission Date
- 20-08-2024
- Latest Decision Or Authorization Date
- 08-07-2025
- Processing Time Days
- 322
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- HUS-Yhtymae
- Department Name
- New Children’s Hospital
- Principal Investigator Name
- Satu Långström
- Principal Investigator Email
- satu.langstrom@hus.fi
- Contact Person Name
- Satu Långström
- Contact Person Email
- satu.langstrom@hus.fi
Croatia
- Earliest CTIS Part Ii Submission Date
- 20-08-2024
- Latest Decision Or Authorization Date
- 21-07-2025
- Processing Time Days
- 335
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- University Hospital Centre Zagreb
- Department Name
- Internal medicine
- Principal Investigator Name
- Ana Boban
- Principal Investigator Email
- ana.boban@kbc-zagreb.hr
- Contact Person Name
- Ana Boban
- Contact Person Email
- ana.boban@kbc-zagreb.hr
Spain
- Earliest CTIS Part Ii Submission Date
- 20-08-2024
- Latest Decision Or Authorization Date
- 29-08-2025
- Processing Time Days
- 374
- Number Of Sites
- 4
- Number Of Participants
- 2
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Dept of Hematology and Hemotherapy
- Principal Investigator Name
- Olga Benitez Hidalgo
- Principal Investigator Email
- obenitez@vhebron.net
- Contact Person Name
- Olga Benitez Hidalgo
- Contact Person Email
- obenitez@vhebron.net
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Hematology Department
- Principal Investigator Name
- Ramiro Nuñez Vazquez
- Principal Investigator Email
- ramirojosenv@gmail.com
- Contact Person Name
- Ramiro Nuñez Vazquez
- Contact Person Email
- ramirojosenv@gmail.com
- Site Name
- Hospital General Universitario Dr. Balmis
- Department Name
- Dept of Haemostasis and Thrombosis
- Principal Investigator Name
- Ana Marco Rico
- Principal Investigator Email
- marco_anaric@gva.es
- Contact Person Name
- Ana Marco Rico
- Contact Person Email
- marco_anaric@gva.es
- Site Name
- Hospital Universitario San Juan De Alicante
- Department Name
- Hematology Department
- Principal Investigator Name
- Ana Marco Rico
- Principal Investigator Email
- marco_anaric@gva.es
- Contact Person Name
- Ana Marco Rico
- Contact Person Email
- marco_anaric@gva.es
Bulgaria
- Earliest CTIS Part Ii Submission Date
- 14-10-2025
- Latest Decision Or Authorization Date
- 28-10-2025
- Processing Time Days
- 14
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Specialized Hospital For Active Treatment Of Hematological Diseases EAD
- Department Name
- Clinic of Pediatric Hematology for treatment of children with benign hematological diseases 0-18y
- Principal Investigator Name
- Atanas Banchev
- Principal Investigator Email
- bantschev@gmail.com
- Contact Person Name
- Atanas Banchev
- Contact Person Email
- bantschev@gmail.com
Sponsor
Primary sponsor
- Full Name
- HZRM Haemophilie-Zentrum Rhein Main GmbH
- Organisation Type
- Hospital/Clinic/Other health care facility
- Country Of Registered Address
- Germany
Contract research organisations
- Name
- Allucent (UK) Limited
- Responsibilities
- CRO responsibilities
Third parties
- {"country":"United Kingdom","full_name":"Allucent (UK) Limited","duties_or_roles":"CRO responsibilities","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Universitaetsklinik Balgrist","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Germany","full_name":"Universitaetsklinikum Bonn AöR","duties_or_roles":"F8 mutations, FVIII inhibitor epitope mapping, Thrombotic risk markers, antiemicizumab antib detection","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Germany","full_name":"LKF Laboratorium fuer Klinische Forschung GmbH","duties_or_roles":"Coordinates laboratory samples collection and distribution","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Switzerland","full_name":"nspm AG","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Emory Children Center","duties_or_roles":"epitope mapping","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Germany","full_name":"HZRM Haemophilie-Zentrum Rhein Main GmbH","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"France","full_name":"Hospices Civils De Lyon","duties_or_roles":"TGA analysis","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
Co-sponsors
- Emory University
Investigational products
- Investigational Product Name
- Nuwiq 250 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 500 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 1000 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 1500 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 2000 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 2500 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 3000 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Nuwiq 4000 IU powder and solvent for solution for injection
- Active Substance
- SIMOCTOCOG ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Octanate 250 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
- Active Substance
- HUMAN COAGULATION FACTOR VIII
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Octanate 500 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
- Active Substance
- HUMAN COAGULATION FACTOR VIII
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Octanate 1000 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
- Active Substance
- HUMAN COAGULATION FACTOR VIII
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Wilate 500, 500 I.E. VWF/500 I.E. FVIII, Pulver und Lösungsmittel zur Herstellung einer intravenösen Injektionslösung.
- 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 1000, 1000 I.E. VWF/1000 I.E. FVIII, Pulver und Lösungsmittel zur Herstellung einer intravenösen Injektionslösung.
- 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
- FEIBA 2500 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung.
- Active Substance
- FACTOR VIII INHIBITOR BYPASSING FRACTION
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- FEIBA 500 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung.
- Active Substance
- FACTOR VIII INHIBITOR BYPASSING FRACTION
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- FEIBA 1000 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung
- Active Substance
- FACTOR VIII INHIBITOR BYPASSING FRACTION
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Hemlibra 150 mg/mL solution for injection
- Active Substance
- EMICIZUMAB
- Modality
- Bispecific antibody
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- SUBCUTANEOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- Hemlibra 30 mg/mL solution for injection
- Active Substance
- EMICIZUMAB
- Modality
- Bispecific antibody
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- SUBCUTANEOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- NovoSeven 1 mg (50 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG ALFA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- NovoSeven 2 mg (100 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG ALFA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- NovoSeven 5 mg (250 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG ALFA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- NovoSeven 8 mg (400 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG ALFA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- CEVENFACTA 5 mg (225 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG BETA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- CEVENFACTA 2 mg (90 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG BETA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Investigational Product Name
- CEVENFACTA 1 mg (45 KIU) powder and solvent for solution for injection
- Active Substance
- EPTACOG BETA (ACTIVATED)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- Authorised
- Combination Treatment
- Yes
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