A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
NCT ID: NCT05135559
Last Updated: 2025-12-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
153 participants
INTERVENTIONAL
2022-03-24
2029-11-02
Brief Summary
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Participants will have to inject the study medicine every day under the skin with a pen-injector.
The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.
Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Concizumab-naïve patients
Concizumab-naïve participants below 12 years of age at the time of consent/assent
Concizumab
Participants in Arm 1 will be assigned to concizumab prophylaxis starting with a loading dose on treatment day 0 followed by daily injections of an individual maintenance dose.
Participants in Arm 2 will be assigned to concizumab prophylaxis with daily injections of an individual maintenance dose.
Patients coming from compassionate use
Patients previously treated with concizumab via compassionate use, either on an individual patient basis or through the concizumab compassionate use programme NN7415-4807
Concizumab
Participants in Arm 1 will be assigned to concizumab prophylaxis starting with a loading dose on treatment day 0 followed by daily injections of an individual maintenance dose.
Participants in Arm 2 will be assigned to concizumab prophylaxis with daily injections of an individual maintenance dose.
Interventions
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Concizumab
Participants in Arm 1 will be assigned to concizumab prophylaxis starting with a loading dose on treatment day 0 followed by daily injections of an individual maintenance dose.
Participants in Arm 2 will be assigned to concizumab prophylaxis with daily injections of an individual maintenance dose.
Eligibility Criteria
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Inclusion Criteria
* Diagnosis of congenital severe haemophilia A (FVIII below 1%) or moderate/severe congenital haemophilia B (FIX (coagulation factor IX) below or equal to 2%), or congenital haemophilia with inhibitors.
* For arm 1 only: Male aged below 12 years of age at the time of signing informed consent.
* For arm 1 only: Patients with inhibitors (haemophilia A with inhibitors or haemophilia B with inhibitors)
1. Patients with HAwI (haemophilia A with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
2. Patients with HBwI (haemophilia B with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
3. Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products)
* For arm 1 only: Patients without inhibitors (haemophilia A or haemophilia B)
1. Patients with historical medical records of at least 52 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products; Surgery related PPX or short-term PPX (e.g., in relation to a severe bleed) is not allowed) during the last year prior to enrolment and with at least 3 documented treated bleeds (For participants less than (\<) 2 years of age there is no limitation for number of documented treated bleeds in the medical history) during this period
2. Patients with historical medical records of a total of at least 26 weeks of PPX (prophylaxis) treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available)
* For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.
Exclusion Criteria
* Known inherited or acquired coagulation disorder other than congenital haemophilia.
* Ongoing or planned Immune Tolerance Induction treatment.
* History of thromboembolic disease (aIncludes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events).
ALL
No
Sponsors
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Novo Nordisk A/S
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Transparency (dept. 2834)
Role: STUDY_DIRECTOR
Novo Nordisk A/S
Locations
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Rady Childrens Hosp San Diego
San Diego, California, United States
University of Colorado Hospital
Aurora, Colorado, United States
Arnold Palmer Children's Hospital
Orlando, Florida, United States
Nemours Child Orlando Hem/Onc.
Orlando, Florida, United States
Augusta Univ/Childrens Hosp-GA
Augusta, Georgia, United States
Memorial Health University Medical Center
Savannah, Georgia, United States
Childrens Hospital of Chicago
Chicago, Illinois, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, United States
Children's Hosp-New Orleans
New Orleans, Louisiana, United States
Ochsner Clinic Foundation
New Orleans, Louisiana, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Children's Nebraska
Omaha, Nebraska, United States
ECU Sickle Cell Comp Clinic
Greenville, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
St Christopher Hosp for Child
Philadelphia, Pennsylvania, United States
Vanderbilt Hemostasis Treatment Clinic
Nashville, Tennessee, United States
Cook Children's Hospital-Hematology-Oncology
Fort Worth, Texas, United States
Texas Children's Hospital_Houston
Houston, Texas, United States
Pediatrics Hematology/Oncology Clinic Battle Building
Charlottesville, Virginia, United States
Haematology and Blood Bank Department
Algiers, , Algeria
CHU Constantine BEN BADIS/ Hematology department
Constantine, , Algeria
University Clinical Center of Republic Srpska (545)
Banja Luka, , Bosnia and Herzegovina
University Clinical Centre Tuzla
Tuzla, , Bosnia and Herzegovina
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv, , Bulgaria
UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology
Sofia, , Bulgaria
UMHAT "Sveta Marina" EAD
Varna, , Bulgaria
BC Children's Hospital
Vancouver, British Columbia, Canada
McMaster Children's Hospital
Hamilton, Ontario, Canada
Tallinn Children's Hospital
Tallinn, , Estonia
Centre Hospitalier Metropole Savoie
Chambéry, , France
Ap-Hp-Hopital de Bicetre-1
Le Kremlin-Bicêtre, , France
AP-HP-HOPITAL NECKER_Service d'hématologie
Paris, , France
Aghia Sophia Childrens' Hospital
Athens, , Greece
'Ippokrateio' General Hospital of Thessaloniki
Thessaloniki, , Greece
'Ippokrateio' General Hospital of Thessaloniki
Thessaloniki, , Greece
Guwahati Medical College
Guwahati, Assam, India
Nirmal Hospital Pvt. Ltd.
Surat, Gujarat, India
SSSH_Dept. of Clinical Haematology & Haemato Oncology
Kolhāpur, Maharashtra, India
Seth GS Medical College & KEM Hospital
Mumbai, Maharashtra, India
K.J Somaiya Hospital and Research Centre
Mumbai, Maharashtra, India
MCGM - Comprehensive Thalassemia Care
Mumbai, Maharashtra, India
Sahyadri Super Speciality Hospital
Pune, Maharashtra, India
S.C.B. Medical College
Cuttack, Odisha, India
J K Lon Hospital
Jaipur, Rajasthan, India
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, India
SGPGI
Lucknow, Uttart Pradesh, India
A.O.U policlinico "G. Rodolico-San Marco"
Catania, , Italy
Dipartimento di Ematologia Univ. Firenze
Florence, , Italy
Azienda Ospedaliera di Padova
Padua, , Italy
Azienda Ospedaliera-Universitaria Parma
Parma, , Italy
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, , Japan
Saitama Children's Med Centre_Hematology-Oncology
Saitama, , Japan
Saint George Hospital University Medical Center
Beirut, , Lebanon
Hospital Nini
Tripoli, , Lebanon
Centre of Oncology and Hematology, Vilnius University
Vilnius, , Lithuania
Hospital Tunku Azizah
Kampung Baru, Kuala Lumpur, Malaysia
Hospital Pulau Pinang
George Town, Pulau Pinang, Malaysia
Sarawak General Hospital
Kuching, Sarawak, Malaysia
Hospital Sultanah Nur Zahirah
Kuala Terengganu, Terengganu, Malaysia
PHI University Clinic for Children's Diseases Skopje
Skopje, , North Macedonia
Klinisk forskningspost
Oslo, , Norway
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, Lower Silesian Voivodeship, Poland
Uniwersyteckie Centrum Kliniczne
Gdansk, , Poland
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, , Poland
Uniwersyteckie Centrum Kliniczne WUM
Warsaw, , Poland
Clinic of Haematology, Fundeni Clinical Institute
Bucharest, , Romania
Spitalul Clinic de Urgenta pentru Copii Cluj Napoca
Cluj-Napoca, , Romania
Spitalul Clinic Judetean De Urgenta Bihor
Oradea, , Romania
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, South Africa
Hospital Virgen de la Arrixaca - Hematología
El Palmar, Murcia, Spain
Hospital Vall d'Hebron
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Regional Universitario de Málaga
Málaga, , Spain
Koagulationscentrum
Gothenburg, , Sweden
Sunpasitthiprasong Hospital_Pediatrics Department
Ubon Ratchathani, Mueang Distirct,, Thailand
King Chulalongkorn Memorial Hospital_Bangkok_0
Bangkok, , Thailand
King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology
Bangkok, , Thailand
Ramathibodi Hospital
Bangkok, , Thailand
Siriraj Hospital - Hematology and Oncology
Bangkok, , Thailand
Gazi University
Ankara, Beşevler/Ankara, Turkey (Türkiye)
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara, Beşevler/Ankara, Turkey (Türkiye)
Acibadem Adana Hastanesi
Adana, , Turkey (Türkiye)
Acıbadem Adana Hastanesi-Hematoloji
Adana, , Turkey (Türkiye)
Ege Universitesi Tip Fakultesi
Izmir, , Turkey (Türkiye)
Ege Üniversitesi Hastanesi- Hematoloji
Izmir, , Turkey (Türkiye)
Ondokuz Mayis University Medical Faculty Ped. Haematology
Samsun, , Turkey (Türkiye)
Ondokuz Mayıs Üniversitesi Hastanesi - Hematoloji
Samsun, , Turkey (Türkiye)
Birmingham Children's Hospital
Birmingham, , United Kingdom
University Hospitals Bristol & Weston NHS Foundation Trust
Bristol, , United Kingdom
Evelina London Children's Hospital - Haemophilia
London, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Countries
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Other Identifiers
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U1111-1247-7330
Identifier Type: OTHER
Identifier Source: secondary_id
2020-000504-11
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
jRCT2031220097
Identifier Type: REGISTRY
Identifier Source: secondary_id
NN7415-4616
Identifier Type: -
Identifier Source: org_study_id