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New daily shot could help kids with hemophilia avoid bleeds

NCT ID NCT05135559

Disease control Sponsor: Novo Nordisk A/S Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 3 times

Summary

This study tests a daily injection called concizumab in children under 12 with hemophilia A or B, with or without inhibitors. The goal is to see if it safely prevents bleeding episodes. Participants receive daily shots under the skin for at least 2 years, and the study tracks how many bleeds occur.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 153 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2022

Expected to finish

Nov 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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).

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Conditions

The condition(s) this trial relates to.

hemophilia A hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Haemophilia A and B with and without inhibitors

Contacts and locations

Locations

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642, Greece

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, GR 54642, Greece

  • A.O.U policlinico "G. Rodolico-San Marco"

    Catania, 95123, Italy

  • AP-HP-HOPITAL NECKER_Service d'hématologie

    Paris, 75015, France

  • Acibadem Adana Hastanesi

    Adana, 01130, Turkey (Türkiye)

  • Acıbadem Adana Hastanesi-Hematoloji

    Adana, 01130, Turkey (Türkiye)

  • Aghia Sophia Childrens' Hospital

    Athens, GR-11527, Greece

  • Ap-Hp-Hopital de Bicetre-1

    Le Kremlin-Bicêtre, 94275, France

  • Arnold Palmer Children's Hospital

    Orlando, Florida, 32806, United States

  • Augusta Univ/Childrens Hosp-GA

    Augusta, Georgia, 30912, United States

  • Azienda Ospedaliera di Padova _

    Padova, 35128, Italy

  • Azienda Ospedaliera-Universitaria Parma

    Parma, 43126, Italy

  • BC Children's Hospital

    Vancouver, British Columbia, V6H 3V4, Canada

  • Birmingham Children's Hospital

    Birmingham, B4 6NH, United Kingdom

  • CHU Constantine BEN BADIS/ Hematology department

    Constantine, 25000, Algeria

  • Centre Hospitalier Metropole Savoie

    Chambéry, 73000, France

  • Centre of Oncology and Hematology, Vilnius University

    Vilnius, LT-08406, Lithuania

  • Charlotte Maxeke Johannesburg Academic Hospital

    Parktown, Johannesburg, Gauteng, 2193, South Africa

  • Children's Hosp-New Orleans

    New Orleans, Louisiana, 70118, United States

  • Children's Nebraska

    Omaha, Nebraska, 68114, United States

  • Childrens Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Clinic of Haematology, Fundeni Clinical Institute

    Bucharest, 022328, Romania

  • Cook Children's Hospital-Hematology-Oncology

    Fort Worth, Texas, 76104, United States

  • Dipartimento di Ematologia Univ. Firenze

    Florence, 50134, Italy

  • ECU Sickle Cell Comp Clinic

    Greenville, North Carolina, 27834, United States

  • Ege Universitesi Tip Fakultesi

    Izmir, 35100, Turkey (Türkiye)

  • Ege Üniversitesi Hastanesi- Hematoloji

    Izmir, 35100, Turkey (Türkiye)

  • Evelina London Children's Hospital - Haemophilia

    London, SE1 7EH, United Kingdom

  • Gazi University

    Ankara, Beşevler/Ankara, 06500, Turkey (Türkiye)

  • Gazi Üniversitesi Hastanesi- Hematoloji

    Ankara, Beşevler/Ankara, 06500, Turkey (Türkiye)

  • Great Ormond Street Hospital for Children

    London, WC1N 3HR, United Kingdom

  • Guwahati Medical College

    Guwahati, Assam, 781032, India

  • Haematology and Blood Bank Department

    Algiers, 16000, Algeria

  • Hospital Nini

    Tripoli, 1434, Lebanon

  • Hospital Pulau Pinang

    George Town, Pulau Pinang, 10450, Malaysia

  • Hospital Regional Universitario de Málaga

    Málaga, 29009, Spain

  • Hospital Sultanah Nur Zahirah

    Kuala Terengganu, Terengganu, 20400, Malaysia

  • Hospital Tunku Azizah

    Kampung Baru, Kuala Lumpur, 50300, Malaysia

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Vall d'Hebron

    Barcelona, 08035, Spain

  • Hospital Virgen de la Arrixaca - Hematología

    El Palmar, Murcia, 30120, Spain

  • Indiana Hemophilia-Thromb Ctr

    Indianapolis, Indiana, 46260, United States

  • J K Lon Hospital

    Jaipur, Rajasthan, 302004, India

  • K.J Somaiya Hospital and Research Centre

    Mumbai, Maharashtra, 400022, India

  • King Chulalongkorn Memorial Hospital_Bangkok_0

    Bangkok, 10330, Thailand

  • King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology

    Bangkok, 10330, Thailand

  • Klinisk forskningspost

    Oslo, 0372, Norway

  • MCGM - Comprehensive Thalassemia Care

    Mumbai, Maharashtra, 400066, India

  • McMaster Children's Hospital

    Hamilton, Ontario, L8N 3Z5, Canada

  • Memorial Health University Medical Center

    Savannah, Georgia, 31404, United States

  • Monroe Carell Jr. Children's Hospital at Vanderbilt

    Nashville, Tennessee, 37232, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nemours Child Orlando Hem/Onc.

    Orlando, Florida, 32827, United States

  • Nirmal Hospital Pvt. Ltd.

    Surat, Gujarat, 395002, India

  • Ochsner Clinic Foundation

    New Orleans, Louisiana, 70121, United States

  • Ondokuz Mayis University Medical Faculty Ped. Haematology

    Samsun, 55139, Turkey (Türkiye)

  • Ondokuz Mayıs Üniversitesi Hastanesi - Hematoloji

    Samsun, 55139, Turkey (Türkiye)

  • PHI University Clinic for Children's Diseases Skopje

    Skopje, 1000, North Macedonia

  • Pediatrics Hematology/Oncology Clinic Battle Building

    Charlottesville, Virginia, 22908, United States

  • Post Graduate Institute of Child Health

    Noida, Uttar Pradesh, 201303, India

  • Rady Childrens Hosp San Diego

    San Diego, California, 92123, United States

  • Ramathibodi Hospital

    Bangkok, 10400, Thailand

  • S.C.B. Medical College

    Cuttack, Odisha, 753007, India

  • SGPGI

    Lucknow, Uttart Pradesh, 226014, India

  • SSSH_Dept. of Clinical Haematology & Haemato Oncology

    Kolhāpur, Maharashtra, 416005, India

  • Sahlgrenska US - Koagulationscentrum

    Gothenburg, 413 46, Sweden

  • Sahyadri Super Speciality Hospital

    Pune, Maharashtra, 411004, India

  • Saint George Hospital University Medical Center

    Beirut, 961, Lebanon

  • Saitama Children's Med Centre_Hematology-Oncology

    Saitama, 330-8777, Japan

  • Sarawak General Hospital

    Kuching, Sarawak, 93586, Malaysia

  • Seth GS Medical College & KEM Hospital

    Mumbai, Maharashtra, 400012, India

  • Siriraj Hospital - Hematology and Oncology

    Bangkok, 10700, Thailand

  • Spitalul Clinic Judetean De Urgenta Bihor

    Oradea, 410469, Romania

  • Spitalul Clinic de Urgenta pentru Copii Cluj Napoca

    Cluj-Napoca, 400177, Romania

  • St Christopher Hosp for Child

    Philadelphia, Pennsylvania, 19134, United States

  • St. Marianna University School of Medicine Hospital_Pediatrics

    Kanagawa, 216-8511, Japan

  • Sunpasitthiprasong Hospital_Pediatrics Department

    Ubon Ratchathani, Mueang Distirct,, 34000, Thailand

  • Tallinn Children's Hospital

    Tallinn, 13419, Estonia

  • Texas Children's Hospital_Houston

    Houston, Texas, 77030, United States

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • UMHAT Sveta Marina EAD, Clinic of Pediatric Clinical Hematology and Oncology

    Varna, 9010, Bulgaria

  • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

    Plovdiv, 4002, Bulgaria

  • UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology

    Sofia, 1527, Bulgaria

  • University Clinical Center of Republic Srpska (545)

    Banja Luka, 78000, Bosnia and Herzegovina

  • University Clinical Centre Tuzla

    Tuzla, 75000, Bosnia and Herzegovina

  • University Hospitals Bristol & Weston NHS Foundation Trust

    Bristol, BS2 8BJ, United Kingdom

  • University of Colorado Hospital

    Aurora, Colorado, 80045, United States

  • Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

    Lublin, 20-093, Poland

  • Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

    Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-952, Poland

  • Uniwersyteckie Centrum Kliniczne WUM

    Warsaw, 02-091, Poland

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