Clinical trial • Phase II • Haematology|Rare Disease

efanesoctocog alfa for Haemophilia A|Severe haemophilia A

Phase II trial of efanesoctocog alfa for Haemophilia A|Severe haemophilia A. 36 participants.

Overview

Trial Therapeutic Area
Haematology|Rare Disease
Trial Disease
Haemophilia A|Severe haemophilia A
Trial Stage
Phase II
Drug Modality
Peptide/protein/enzyme

Key dates

Initial CTIS Submission Date
23-11-2023
First CTIS Authorization Date
03-04-2024

Trial design

Phase II trial across 9 sites in Germany, Italy, Spain and others.

Target Sample Size
36

Eligibility

Recruits 36 No vulnerable populations selected..

Vulnerable Population
No vulnerable populations selected.

Recruitment

Planned Sample Size
36
Recruitment Window Months
24

Geography

Total Number Of Sites
9
Total Number Of Participants
36

Germany

Earliest CTIS Part Ii Submission Date
22-01-2024
Latest Decision Or Authorization Date
05-04-2024
Processing Time Days
74
Number Of Sites
2
Number Of Participants
7

Sites

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
Site Name
Universitaetsklinikum Bonn AöR
Department Name
Experimental Haematology 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

Italy

Earliest CTIS Part Ii Submission Date
13-12-2023
Latest Decision Or Authorization Date
04-04-2024
Processing Time Days
113
Number Of Sites
3
Number Of Participants
7

Sites

Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
Unit of Innovative Therapies for Genetic and Metabolic Diseases
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
Humanitas Research Hospital
Department Name
Phase I Unit
Principal Investigator Name
Maria Elisa Mancuso
Principal Investigator Email
mariaelisa.mancuso@humanitas.it
Contact Person Name
Maria Elisa Mancuso
Site Name
Azienda Ospedaliero Universitaria Careggi
Department Name
Center for Bleeding Clinical Trial Unit-1 (CTU-1)
Principal Investigator Name
Giancarlo Castaman
Principal Investigator Email
castaman@aou-careggi.toscana.it
Contact Person Name
Giancarlo Castaman

Spain

Earliest CTIS Part Ii Submission Date
05-03-2024
Latest Decision Or Authorization Date
03-04-2024
Processing Time Days
29
Number Of Sites
3
Number Of Participants
10

Sites

Site Name
Hospital Unviersitario Miguel Servet
Department Name
Hematology and Hemotherapy Department
Principal Investigator Name
Jose Manuel Calvo Villas
Principal Investigator Email
jmcvillas@hotmail.com
Contact Person Name
Jose Manuel Calvo Villas
Contact Person Email
jmcvillas@hotmail.com
Site Name
Complejo Hospitalario Universitario Juan Canalejo
Department Name
Hematology
Principal Investigator Name
Marta Fernandez Docampo
Principal Investigator Email
Marta.Fernandez.Docampo@sergas.es
Contact Person Name
Marta Fernandez Docampo
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Hemostasis and Thrombosis Unit
Principal Investigator Name
Haya Guaita Saturnino
Principal Investigator Email
hagusat@gmail.com
Contact Person Name
Haya Guaita Saturnino
Contact Person Email
hagusat@gmail.com

Bulgaria

Earliest CTIS Part Ii Submission Date
10-10-2025
Latest Decision Or Authorization Date
18-12-2025
Processing Time Days
69
Number Of Sites
1
Number Of Participants
12

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

Sponsor

Primary sponsor

Full Name
Swedish Orphan Biovitrum AB (publ)
Organisation Type
Pharmaceutical company
Country Of Registered Address
Sweden

Investigational products

Investigational Product Name
efanesoctocog alfa
Active Substance
efanesoctocog alfa
Modality
Peptide/protein/enzyme
Investigational Product Name
Other EHL-rFVIII products
Active Substance
recombinant factor VIII (rFVIII) - extended half-life products
Modality
Peptide/protein/enzyme

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