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Clinical Trials in the UK / NCT05135559
Active, not recruiting Phase 3

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

NCT05135559 · tracked via the Priya Life Science UK tracker
Sponsor
Novo Nordisk A/S
Phase
Phase 3
Started
2022-03-24
Last updated
2026-09-18

Condition(s) studied

Haemophilia A and B With and Without Inhibitors

Investigational drug(s) / intervention(s)

Concizumab

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.

Study summary

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.

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.

Eligibility

Sex
ALL
Min age
Max age
Healthy volunteers
No
Inclusion Criteria: * Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study. * 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 or suspected hypersensitivity to study intervention or related products. * 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).

Primary outcome measure(s)

Trial sites (91)

FacilityCityRegionStatus
Rady Childrens Hosp San Diego San Diego California
University of Colorado Hospital Aurora Colorado
Arnold Palmer Children's Hospital Orlando Florida
Nemours Child Orlando Hem/Onc. Orlando Florida
Augusta Univ/Childrens Hosp-GA Augusta Georgia
Memorial Health University Medical Center Savannah Georgia
Childrens Hospital of Chicago Chicago Illinois
Indiana Hemophilia-Thromb Ctr Indianapolis Indiana
Children's Hosp-New Orleans New Orleans Louisiana
Ochsner Clinic Foundation New Orleans Louisiana
The Children's Mercy Hospital Kansas City Missouri
Children's Nebraska Omaha Nebraska
ECU Sickle Cell Comp Clinic Greenville North Carolina
Nationwide Children's Hospital Columbus Ohio
St Christopher Hosp for Child Philadelphia Pennsylvania
Monroe Carell Jr. Children's Hospital at Vanderbilt Nashville Tennessee
Cook Children's Hospital-Hematology-Oncology Fort Worth Texas
Texas Children's Hospital_Houston Houston Texas
Pediatrics Hematology/Oncology Clinic Battle Building Charlottesville Virginia
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, Clinic of Pediatric Clinical Hematology and Oncology Varna Bulgaria
BC Children's Hospital Vancouver British Columbia
McMaster Children's Hospital Hamilton Ontario
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
Nirmal Hospital Pvt. Ltd. Surat Gujarat
SSSH_Dept. of Clinical Haematology & Haemato Oncology Kolhāpur Maharashtra
Seth GS Medical College & KEM Hospital Mumbai Maharashtra
K.J Somaiya Hospital and Research Centre Mumbai Maharashtra

+ 51 more sites — see the full list on the official registry below.

More Novo Nordisk A/S trials in the UK

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05135559 on ClinicalTrials.gov ↗ ← All trials in the UK