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NCT05135559

PHASE3

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

Sponsor: Novo Nordisk A/S

View on ClinicalTrials.gov

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.

Official title: Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

Key Details

Gender

All

Age Range

Any - Any

Study Type

INTERVENTIONAL

Enrollment

153

Start Date

2022-03-24

Completion Date

2029-11-02

Last Updated

2025-12-30

Healthy Volunteers

Conditions

Haemophilia A and B With and Without Inhibitors

Interventions

DRUG

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.

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).

Locations (91)

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

Padova, 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

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (91)