Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruiting
Participants planned
131
Countries
10
Sites
31
Haemophilia A
Safety Objective To collect safety and tolerability data of efanesoctocog alfa in previously treated patients with haemophilia A. Efficacy Objective 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…
Key facts
- Sponsor
- Swedish Orphan Biovitrum AB (publ)
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 24 Feb 2025 → ongoing
- Decision date (initial)
- 2025-03-24
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Swedish Orphan Biovitrum AB (Sobi)
External identifiers
- EU CT number
- 2023-506537-29-00
- WHO UTN
- U1111-1296-0765
- ClinicalTrials.gov
- NCT06716814
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Efficacy, Safety
Safety Objective
To collect safety and tolerability data of efanesoctocog alfa in previously treated patients with haemophilia A.
Efficacy Objective
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
Conditions and MedDRA coding
Haemophilia A
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10018937 | Haemophilia A | 10010331 |
Study design 3 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Visit 1 / Baseline Visit (on-site) Should be done in connection to the End of Treatment (EoT)/EoS visit (or equivalent) in the respective parent study.
|
Not Applicable | None | ||
| 2 | Treatment Patients will be treated with a once-weekly prophylactic dose of efanesoctocog alfa at 50 IU/kg and also trained on how to complete the patient diary. Assessments will be conducted during the on-site visits, which will occur every 6 months, and during phone call visits, which will occur half-way between each on-site visit.
|
Not Applicable | None | ||
| 3 | EoT Visit Patients will be scheduled for an EoT Visit once efanesoctocog alfa is commercially available in each patient’s
respective country or in March 2027, whichever comes first. An EoS safety phone call will be performed 14 (+7 days) after the EoT Visit.
|
Not Applicable | None |
Regulatory references
- Plan to share IPD
- Yes
- IPD plan description
- Qualified researchers may request access to patient level data and related study documents. Patient level data will be anonymized and study documents, if applicable will be redacted to protect the privacy of trial participants. Further details on Sponsor's data sharing criteria, and process for requesting access can be found at: https://www.sobi.com/en/policies.
| EU CT number | Title | Sponsor |
|---|---|---|
| 2020-000769-18 | A Phase 3 open-label, multicenter study of the safety, efficacy, and pharmacokinetics of intravenous recombinant coagulation Factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated pediatric patients <12 years of age with severe hemophilia A, Az intravénás, rekombináns VIII-as véralvadási faktor Fc - von Willebrand faktor - XTEN fúziós fehérje (rFVIIIFc-VWF-XTEN; BIVV001) biztonságosságának, hatásosságának és farmakokinetikájának III. fázisú, nyílt, többközpontú vizsgálata korábban már kezelt, súlyos A-típusú hemofíliában szenvedő, 12 évesnél fiatalabb betegek körében, Az intravénás, rekombináns VIII-as véralvadási faktor Fc - von Willebrand faktor - XTEN fúziós fehérje (rFVIIIFc-VWF-XTEN; BIVV001) biztonságosságának, hatásosságának és farmakokinetikájának III. fázisú, nyílt, többközpontú vizsgálata korábban már kezelt, súlyos A-típusú hemofíliában szenvedő, 12 évesnél fiatalabb betegek körében, Estudio abierto multicéntrico de fase III sobre la seguridad, eficacia y farmacocinética del factor de coagulación recombinante intravenoso VIII Fc-Factor von Willebrand-Proteína de fusión XTEN (rFVIIIFc-VWF-XTEN; BIVV001) en pacientes pediátricos previamente tratados menores de 12 años con hemofilia A grave, Studio di fase 3, multicentrico, in aperto per valutare la sicurezza, l’efficacia e la farmacocinetica del Fattore VIII della coagulazione ricombinante Fc - fattore di von Willebrand - proteina di fusione XTEN (rFVIIIFc-VWF-XTEN; BIVV001) somministrato per via endovenosa in pazienti pediatrici di < 12 anni di età affetti da emofilia A severa precedentemente trattati | |
| 2019-002023-15 | Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients ≥12 Years of Age With Severe Hemophilia A, Étude de phase 3, en ouvert, multicentrique visant à évaluer l'innocuité, l'efficacité et la pharmacocinétique de l'association intraveineuse du facteur VIII de coagulation recombinant fusionné au fragment Fc-facteur de von Willebrand-protéine de fusion XTEN (rFVIIIFc-VWF-XTEN ; BIVV001) chez des patients ayant déjà été traités, âgés de ≥ 12 ans atteints d'hémophilie A sévère, Een open, multicentrisch fase 3-onderzoek naar de veiligheid, werkzaamheid en farmacokinetiek van intraveneus toegediend fusieproteïne van recombinant stollingsfactor VIII-Fc, von-willebrandfactor en XTEN (rFVIIIFc-VWF-XTEN; BIVV001) bij eerder behandelde patiënten ≥ 12 jaar met ernstige hemofilie A, Étude de phase 3, en ouvert, multicentrique visant à évaluer l'innocuité, l'efficacité et la pharmacocinétique de l’association intraveineuse du facteur VIII de coagulation recombinant fusionné au fragment Fc-facteur de von Willebrand-protéine de fusion XTEN (rFVIIIFc-VWF-XTEN ; BIVV001) chez des patients ayant déjà été traités, âgés de ≥ 12 ans atteints d’hémophilie A sévère, Étude de phase 3, en ouvert, multicentrique visant à évaluer l'innocuité, l'efficacité et la pharmacocinétique de l'association intraveineuse du facteur VIII de coagulation recombinant fusionné au fragment Fc-facteur de von Willebrand-protéine de fusion XTEN (rFVIIIFc-VWF-XTEN ; BIVV001) chez des patients ayant déjà été traités, âgés de ≥ 12 ans atteints d'hémophilie A sévère, Een open, multicentrisch fase 3-onderzoek naar de veiligheid, werkzaamheid en farmacokinetiek van intraveneus toegediend fusieproteïne van recombinant stollingsfactor VIII-Fc, vonwillebrandfactor en XTEN (rFVIIIFc-VWF-XTEN; BIVV001) bij eerder behandelde patiënten ≥ 12 jaar met ernstige hemofilie A, Étude de phase 3, en ouvert, multicentrique visant à évaluer l'innocuité, l'efficacité et la pharmacocinétique de l'association intraveineuse du facteur VIII de coagulation recombinant fusionné au fragment Fc-facteur de von Willebrand-protéine de fusion XTEN (rFVIIIFc-VWF-XTEN ; BIVV001) chez des patients ayant déjà été traités, âgés de ≥ 12 ans atteints d'hémophilie A sévère, Een open, multicentrisch fase 3-onderzoek naar de veiligheid, werkzaamheid en farmacokinetiek van intraveneus toegediend fusieproteïne van recombinant stollingsfactor VIII-Fc, vonwillebrandfactor en XTEN (rFVIIIFc-VWF-XTEN; BIVV001) bij eerder behandelde patiënten ≥ 12 jaar met ernstige hemofilie A, Az intravénás, rekombináns VIII-as véralvadási faktor Fc - von Willebrand faktor - XTEN fúziós fehérje (rFVIIIFc-VWF-XTEN; BIVV001) biztonságosságának, hatékonyságának és farmakokinetikájának III. fázisú, nyílt, többközpontú vizsgálata a korábban már kezelt, 12 éves vagy idősebb, súlyos A-típusú hemofíliás betegeknél, Estudio abierto multicéntrico de fase 3 sobre la seguridad, eficacia y farmacocinética del factor de coagulación recombinante intravenoso VIII Fc-Factor von Willebrand-Proteína de fusión XTEN (rFVIIIFc-VWF-XTEN; BIVV001) en pacientes tratados previamente con >=12 años con hemofilia A severa, Studio di fase III, multicentrico e in aperto sulla sicurezza, sull'efficacia e sulla farmacocinetica della proteina di fusione costituita dal fattore VIII della coagulazione ricombinante legato al dominio Fc, dal fattore di von Willebrand e da XTEN (rFVIIIFc-VWF-XTEN; BIVV001) somministrata per via endovenosa in pazienti pretrattati di eta' = o > a 12 anni con emofilia A severa | |
| 2020-002215-22 | A Phase 3 open-label, multicenter study of the long-term safety and efficacy of intravenous recombinant coagulation factor VIII Fc-von willebrand factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated patients with severe hemophilia A , Μια ανοιχτής επισήμανσης, πολυκεντρική μελέτη Φάσης 3 για τη μακροπρόθεσμη ασφάλεια και αποτελεσματικότητα της ενδοφλέβιας ανασυνδυασμένης πρωτεΐνης σύντηξης ανθρώπινου παράγοντα πήξης VIII Fc-von Willebrand factor-XTEN (rFVIIIFc-VWF-XTEN, BIVV001) σε ασθενείς που έχουν υποβληθεί σε προηγούμενη θεραπεία με σοβαρή αιμορροφιλία Α., III. fázisú, nyílt, multicentrikus vizsgálat, amely az intravénás, rekombináns VIII-as véralvadási faktor Fc-von Willebrand faktor - XTEN fúziós fehérje (rFVIIIFc-VWF-XTEN; BIVV001) hosszú távú biztonságosságát és hatásosságát vizsgálja korábban már kezelt, súlyos A-típusú hemofíliában szenvedő betegek körében, Estudio abierto multicéntrico de fase III sobre la seguridad y eficacia a largo plazo del factor de coagulación recombinante intravenoso VIII·Fc-Factor von Willebrand-Proteína de fusión XTEN (rFVIIIFc-VWF-XTEN; BIVV001) en pacientes previamente tratados con hemofilia A grave, Studio di fase 3, multicentrico, in aperto per la valutazione della sicurezza a lungo termine e dell’efficacia del Fattore VIII della coagulazione ricombinante Fc - fattore di von Willebrand - proteina di fusione XTEN (rFVIIIFc-VWF-XTEN; BIVV001) endovenoso in pazienti affetti da emofilia A grave precedentemente trattati |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- 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.
- 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.
- 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.
- 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 3
- Positive inhibitor result defined as ≥0.6 Bethesda units (BU)/mL, at the Baseline Visit.
- Ongoing or planned participation in any interventional clinical study at Baseline Visit.
- 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 and secondary outcome measures (English text)
Primary endpoints 2
- Safety Endpoint: The occurrence of adverse events (AEs), including serious adverse events (SAEs) and AESIs
- 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
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 6
ALTUVOCT 2 000 IU powder and solvent for solution for injection
PRD11432036 · Product
- Active substance
- Efanesoctocog Alfa
- Substance synonyms
- Recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein, rFVIIIFc-VWF-XTEN, BIVV001, BIVV-001
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 50 IU/kg international unit(s)/kilogram
- Max total dose
- 5850 IU/kg international unit(s)/kilogram
- Max treatment duration
- 117 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/24/1824/005
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2176
- Modified vs. Marketing Authorisation
- No
ALTUVOCT 3 000 IU powder and solvent for solution for injection
PRD11432043 · Product
- Active substance
- Efanesoctocog Alfa
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 50 IU/kg international unit(s)/kilogram
- Max total dose
- 5850 IU/Kg iu/kilogram
- Max treatment duration
- 117 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/24/1824/006
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2176
- Modified vs. Marketing Authorisation
- No
ALTUVOCT 4 000 IU powder and solvent for solution for injection
PRD11432046 · Product
- Active substance
- Efanesoctocog Alfa
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 50 IU/kg international unit(s)/kilogram
- Max total dose
- 5850 IU/kg international unit(s)/kilogram
- Max treatment duration
- 117 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/24/1824/007
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2176
- Modified vs. Marketing Authorisation
- No
ALTUVOCT 250 IU powder and solvent for solution for injection
PRD11427583 · Product
- Active substance
- Efanesoctocog Alfa
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 50 IU/kg international unit(s)/kilogram
- Max total dose
- 5850 IU/kg international unit(s)/kilogram
- Max treatment duration
- 117 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/24/1824/001
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2176
- Modified vs. Marketing Authorisation
- No
ALTUVOCT 1 000 IU powder and solvent for solution for injection
PRD11431539 · Product
- Active substance
- Efanesoctocog Alfa
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 50 IU/kg international unit(s)/kilogram
- Max total dose
- 5850 IU/kg international unit(s)/kilogram
- Max treatment duration
- 117 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/24/1824/004
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2176
- Modified vs. Marketing Authorisation
- No
ALTUVOCT 500 IU powder and solvent for solution for injection
PRD11431456 · Product
- Active substance
- Efanesoctocog Alfa
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 50 IU/Kg iu/kilogram
- Max total dose
- 5850 IU/kg international unit(s)/kilogram
- Max treatment duration
- 117 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/24/1824/002
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2176
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Swedish Orphan Biovitrum AB (publ)
- Sponsor organisation
- Swedish Orphan Biovitrum AB (publ)
- Address
- Tvistevagen 48, Alidhem Alidhem
- City
- Umea
- Postcode
- 907 36
- Country
- Sweden
Scientific contact point
- Organisation
- Swedish Orphan Biovitrum AB (publ)
- Contact name
- Elena Santagostino
Public contact point
- Organisation
- Swedish Orphan Biovitrum AB (publ)
- Contact name
- Contact point
Third parties 10
| Organisation | City, country | Duties |
|---|---|---|
| Almac Clinical Services Limited ORG-100017464
|
Craigavon, United Kingdom (Northern Ireland) | Code 14, Other |
| Psi Cro AG ORG-100034251
|
Zug, Switzerland | On site monitoring, Code 11, Code 12, Other, Code 2, Code 5, Data management |
| Veeva Systems Inc. ORG-100006053
|
Pleasanton, United States | E-data capture |
| Labcorp ORG-100042736
|
Mechelen, Belgium | Other, Laboratory analysis |
| Scout Clinical ORG-100042228
|
Dallas, United States | Other |
| Labcorp Central Laboratory Services S.a.r.l. ORG-100011524
|
Meyrin, Switzerland | Other |
| Almac Clinical Services (Ireland) Limited ORG-100033336
|
Dundalk, Ireland | Code 14 |
| Esoterix Inc. ORG-100042046
|
Englewood, United States | Laboratory analysis |
| Psi CRO Greece ORG-100047165
|
Athens, Greece | On site monitoring, Code 12 |
| Almac Clinical Technologies LLC ORG-100043036
|
Souderton, United States | Interactive response technologies (IRT) |
Locations
10 EU/EEA countries · 31 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Bulgaria | Ongoing, recruiting | 16 | 2 |
| France | Ongoing, recruiting | 28 | 7 |
| Germany | Ended | 7 | 2 |
| Greece | Ongoing, recruiting | 6 | 2 |
| Italy | Ongoing, recruiting | 21 | 8 |
| Netherlands | Ended | 7 | 2 |
| Norway | Ongoing, recruiting | 20 | 1 |
| Slovenia | Ended | 2 | 1 |
| Spain | Ongoing, recruiting | 10 | 3 |
| Sweden | Ongoing, recruiting | 14 | 3 |
| Rest of world | — | 0 | — |
Investigational sites
University Multiprofile Hospital For Active Treatment Saint Georgi EAD
Clinical Hematology Clinic, Bulevard Vasil Aprilov 15a, 4002, Plovdiv
Specialized Hospital For Active Treatment Of Hematological Diseases EAD
Third Department of Clinical Hematology at Clinical Hematology Clinic, Bulevard Kliment Ohridski 1a, 1797, Sofiya
Centre Hospitalier Regional De Marseille
Department of Pediatric Hematology, Immunology and Oncology, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Universitaire De Bordeaux
Site Hôpital des Enfants, Competence and Resources Center for Constitutional Hemorrhagic Disorders, Place Amelie Raba Leon, 33000, Bordeaux
Assistance Publique Hopitaux De Paris
Hôpital Bicêtre, Reference Centre for Haemophilia, 78 Rue Du General Leclerc, 94270, Le Kremlin-Bicetre
Centre Hospitalier Regional Et Universitaire De Brest
Site de la Cavale Blanche, Department of Hematology, Boulevard Tanguy Prigent, 29200, Brest
Les Hopitaux Universitaires De Strasbourg
Site Hautepierre, Department of Hematology and Oncology, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
Hospices Civils De Lyon
Site Louis Pradel, Department of Biological Hematology and Clinical Hemostasis, 59 Boulevard Pinel, 69500, Bron
Centre Hospitalier Universitaire De Lille
Institut Coeur Poumon, Dpartment of Hemostasis and Transfusion, Boulevard Du Professeur Jules Leclercq, 59000, Lille
Universitaetsklinikum Bonn AöR
Institute of Experimental Hematology and Transfusion Medicine, Venusberg-Campus 1, Venusberg, Bonn
Universitaetsklinikum Frankfurt AöR
Hematology, Theodor-Stern-Kai 7, 60590, Frankfurt Am Main
Nosokomeio Paidon I Agia Sofia
Haemophilia Centre, Haemostasis and Thrombosis Unit, Thivon Papadiamantopoulou, 115 27, Athens
Laiko General Hospital Of Athens
Blood Transfusion Service and National Reference Centre for Congenital Bleeding Disorders, Agiou Thoma (goudi) 17, 115 27, Athens
Azienda Ospedaliero Universitaria Careggi
Department of Oncology, SOD Hemorrhagic and Coagulation Diseases, Largo Giovanni Alessandro Brambilla 3, 50134, Florence
Azienda Unita Locale Socio Sanitaria N 8 Berica
UOC Hematology, Viale Ferdinando Rodolfi 37, 36100, Vicenza
Azienda Ospedaliero Universitaria Parma
SSD Hub Center Hemophilia and Congenital Hemorrhagic Diseases, Viale Antonio Gramsci 14, 43126, Parma
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department of Clinical Medicine and Surgery, Via Sergio Pansini 5, 80131, Naples
Humanitas Research Hospital
Center for Thrombosis and Hemorrhagic Diseases, Via Alessandro Manzoni 56, 20089, Rozzano
Azienda Ospedaliero-Universitaria Policlinico Umberto I
Department of Translational and Precision Medicine - Division of Hematology, Viale Del Policlinico 155, 00161, Rome
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Complex Structure of Medicine Hemostasis and Thrombosis, Via Francesco Sforza 28, 20122, Milan
Azienda Ospedaliero Universitaria Renato Dulbecco
Department Hemato-Oncology, Viale Pio X 83, 88100, Catanzaro
Universitair Medisch Centrum Utrecht
Internal medecine, Heidelberglaan 100, 3584 CX, Utrecht
Amsterdam UMC Stichting
Pediatric Hematology, Meibergdreef 9, 1105 AZ, Amsterdam
University Medical Center Ljubljana
Clinical Department of Haematology, Zaloska Cesta 7, 1000, Ljubljana
Hospital Unviersitario Miguel Servet
Hematology, Paseo De Isabel La Catolica 1-3, 50009, Zaragoza
Hospital Universitario Y Politecnico La Fe
Hematology, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Complexo Hospitalario Universitario A Coruna
Hematology, Lugar Jubias De Arriba 84, 15006, A Coruna
Karolinska University Hospital
Department of Haematology, Eugeniavagen 3, 171 64, Solna
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department of Haematology and Coagulation Disorders, Bla Straket 5, Goteborgs Annedal, Goteborg
Region Skane Skanes Universitetssjukhus
Hematology and Vascular diseases, Jan Waldenstroms Gata 16 Plan 5, Malmo St Johannes, Malmo
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Bulgaria | 2025-09-10 | 2025-09-30 | |||
| France | 2025-05-28 | 2025-06-10 | |||
| Greece | 2025-06-17 | 2025-06-18 | |||
| Italy | 2025-03-05 | 2025-03-06 | |||
| Norway | 2025-12-03 | 2026-01-06 | |||
| Spain | 2025-02-24 | 2025-03-10 | |||
| Sweden | 2025-08-27 | 2025-09-16 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 71 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Letter from sponsor_study_population_included_Redacted | 1 |
| Protocol (for publication) | D1_Protocol_2023-506537-29-00_Redacted | 4.0 |
| Protocol (for publication) | D1_Protocol_EL_2023-506537-29-00_Redacted | 4.0 |
| Protocol (for publication) | D4_Patient Diary_DE_2023-506537-29-00 | 1 |
| Protocol (for publication) | D4_Patient Diary_EN_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient Diary_ES_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient Diary_IT_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient Diary_NO_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient Diary_SE_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient_Diary_BG_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient_Diary_EL_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient_Diary_FR_2023-506537-29-00 | 3 |
| Protocol (for publication) | D4_Patient_Diary_NL_2023-506537-29-00 | 2.0 |
| Protocol (for publication) | D4_Patient_Diary_SI_2023-506537-29-00 | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment and Informed Consent Procedure | NA |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 3.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | NA |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Recruitment and Informed Consent Procedure | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_redline | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_REDLINE | N/A |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Main_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Main_SLO_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Assent 12-16 years | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Assent 6-11 years | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Assent_12_17 years | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_Redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_Redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent_Redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parents_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Partner Pregnancy FU_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy Data Collection_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy Follow Up | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy FU_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_FU_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy_FU_SLO_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Privacy_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_14-17 yrs_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adult_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Babys Health Data Collection_Redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Holder of the Parental Authority_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Data Collection_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Follow Up_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy Follow Up_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner Follow-Up_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner Follow-Up_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Reimbursement Form_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information_GP Letter | 2.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_Altuvoct | N/A |
| Synopsis of the protocol (for publication) | D1_Protocol Laysynopsis_DE_2023-506537-29-00 | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Laysynopsis_ES_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol Laysynopsis_FR_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol Laysynopsis_IT_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol Laysynopsis_NO_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol Laysynopsis_SE_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Synopsis_for_laypersons_BG_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Synopsis_for_laypersons_EL_2023-506537-29-00 | 3 |
| Synopsis of the protocol (for publication) | D1_Synopsis_for_laypersons_NL_2023-506537-29-00 | 2.0 |
| Synopsis of the protocol (for publication) | D1_Synopsis_for_laypersons_SI_2023-506537-29-00 | 2.0 |
Application history
13 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-12-04 | Germany | Acceptable 2024-04-10
|
2024-04-11 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-09-16 | Acceptable 2024-11-04
|
2024-11-04 | |
| 3 | SUBSEQUENT ADDITION OF MSC | APP-3 | 2024-12-30 | Acceptable 2024-11-04
|
2025-04-07 | |
| 4 | SUBSEQUENT ADDITION OF MSC | APP-4 | 2024-12-30 | Acceptable 2024-11-04
|
2025-03-10 | |
| 5 | SUBSEQUENT ADDITION OF MSC | APP-5 | 2024-12-30 | Acceptable 2024-11-04
|
2025-03-17 | |
| 6 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-12-30 | Acceptable | 2025-01-27 | |
| 7 | SUBSEQUENT ADDITION OF MSC | APP-7 | 2024-12-31 | Acceptable 2024-11-04
|
2025-02-26 | |
| 8 | SUBSEQUENT ADDITION OF MSC | APP-8 | 2025-01-22 | 2025-03-24 | ||
| 9 | SUBSEQUENT ADDITION OF MSC | APP-9 | 2025-05-21 | Acceptable 2024-11-04
|
2025-07-29 | |
| 10 | SUBSEQUENT ADDITION OF MSC | APP-10 | 2025-07-09 | Acceptable 2024-11-04
|
2025-09-18 | |
| 11 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-07-22 | Acceptable | 2025-08-26 | |
| 12 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-11-14 | Acceptable 2026-02-06
|
2026-02-06 | |
| 13 | SUBSTANTIAL MODIFICATION | SM-6 | 2026-03-11 | Acceptable | 2026-06-01 |