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

NCT05135559 | Active Not Recruiting Phase 3 DMC FDA Drug

• 4 other identifiers: NN7415-4616U1111-1247-73302020-000504-11jRCT2031220097
• Study Type: interventional
• Target: 153
• Locations: 22 countries
• Sites: 89

Brief 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.

Conditions

Haemophilia A and B With and Without Inhibitors

Outcome Measures

Primary Outcomes (2)

• For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes

  Count of episode(s)

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

• For non-inhibitor patients treated on demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes

  Count of episode(s)

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

Secondary Outcomes (25)

• For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

• For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

• For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

• For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

• For non-inhibitor patients treated on-demand during at least the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)

  From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

Study Arms (2)

Concizumab-naïve patients

EXPERIMENTAL

Concizumab-naïve participants below 12 years of age at the time of consent/assent

Drug: Concizumab

Patients coming from compassionate use

EXPERIMENTAL

Patients previously treated with concizumab via compassionate use, either on an individual patient basis or through the concizumab compassionate use programme NN7415-4807

Drug: Concizumab

Interventions

Concizumab DRUG

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.

Concizumab-naïve patients; Patients coming from compassionate use

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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).
• 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).
• 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)
• 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
• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (91)

Rady Childrens Hosp San Diego

San Diego, California, 92123, United States

Location

University of Colorado Hospital

Aurora, Colorado, 80045, United States

Location

Arnold Palmer Children's Hospital

Orlando, Florida, 32806, United States

Location

Nemours Child Orlando Hem/Onc.

Orlando, Florida, 32827, United States

Location

Augusta Univ/Childrens Hosp-GA

Augusta, Georgia, 30912, United States

Location

Memorial Health University Medical Center

Savannah, Georgia, 31404, United States

Location

Childrens Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Indiana Hemophilia-Thromb Ctr

Indianapolis, Indiana, 46260, United States

Location

Children's Hosp-New Orleans

New Orleans, Louisiana, 70118, United States

Location

Ochsner Clinic Foundation

New Orleans, Louisiana, 70121, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Children's Nebraska

Omaha, Nebraska, 68114, United States

Location

ECU Sickle Cell Comp Clinic

Greenville, North Carolina, 27834, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

St Christopher Hosp for Child

Philadelphia, Pennsylvania, 19134, United States

Location

Vanderbilt Hemostasis Treatment Clinic

Nashville, Tennessee, 37212, United States

Location

Cook Children's Hospital-Hematology-Oncology

Fort Worth, Texas, 76104, United States

Location

Texas Children's Hospital_Houston

Houston, Texas, 77030, United States

Location

Pediatrics Hematology/Oncology Clinic Battle Building

Charlottesville, Virginia, 22908, United States

Location

Haematology and Blood Bank Department

Algiers, 16000, Algeria

Location

CHU Constantine BEN BADIS/ Hematology department

Constantine, 25000, Algeria

Location

University Clinical Center of Republic Srpska (545)

Banja Luka, 78000, Bosnia and Herzegovina

Location

University Clinical Centre Tuzla

Tuzla, 75000, Bosnia and Herzegovina

Location

UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

Plovdiv, 4002, Bulgaria

Location

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

Sofia, 1527, Bulgaria

Location

UMHAT "Sveta Marina" EAD

Varna, 9010, Bulgaria

Location

BC Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

McMaster Children's Hospital

Hamilton, Ontario, L8N 3Z5, Canada

Location

Tallinn Children's Hospital

Tallinn, 13419, Estonia

Location

Centre Hospitalier Metropole Savoie

Chambéry, 73000, France

Location

Ap-Hp-Hopital de Bicetre-1

Le Kremlin-Bicêtre, 94275, France

Location

AP-HP-HOPITAL NECKER_Service d'hématologie

Paris, 75015, France

Location

Aghia Sophia Childrens' Hospital

Athens, GR-11527, Greece

Location

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

Location

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, GR 54642, Greece

Location

Guwahati Medical College

Guwahati, Assam, 781032, India

Location

Nirmal Hospital Pvt. Ltd.

Surat, Gujarat, 395002, India

Location

SSSH_Dept. of Clinical Haematology & Haemato Oncology

Kolhāpur, Maharashtra, 416005, India

Location

Seth GS Medical College & KEM Hospital

Mumbai, Maharashtra, 400012, India

Location

K.J Somaiya Hospital and Research Centre

Mumbai, Maharashtra, 400022, India

Location

MCGM - Comprehensive Thalassemia Care

Mumbai, Maharashtra, 400066, India

Location

Sahyadri Super Speciality Hospital

Pune, Maharashtra, 411004, India

Location

S.C.B. Medical College

Cuttack, Odisha, 753007, India

Location

J K Lon Hospital

Jaipur, Rajasthan, 302004, India

Location

Post Graduate Institute of Child Health

Noida, Uttar Pradesh, 201303, India

Location

SGPGI

Lucknow, Uttart Pradesh, 226014, India

Location

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

Catania, 95123, Italy

Location

Dipartimento di Ematologia Univ. Firenze

Florence, 50134, Italy

Location

Azienda Ospedaliera di Padova

Padua, 35128, Italy

Location

Azienda Ospedaliera-Universitaria Parma

Parma, 43126, Italy

Location

St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, 216-8511, Japan

Location

Saitama Children's Med Centre_Hematology-Oncology

Saitama, 330-8777, Japan

Location

Saint George Hospital University Medical Center

Beirut, 961, Lebanon

Location

Hospital Nini

Tripoli, 1434, Lebanon

Location

Centre of Oncology and Hematology, Vilnius University

Vilnius, LT-08406, Lithuania

Location

Hospital Tunku Azizah

Kampung Baru, Kuala Lumpur, 50300, Malaysia

Location

Hospital Pulau Pinang

George Town, Pulau Pinang, 10450, Malaysia

Location

Sarawak General Hospital

Kuching, Sarawak, 93586, Malaysia

Location

Hospital Sultanah Nur Zahirah

Kuala Terengganu, Terengganu, 20400, Malaysia

Location

PHI University Clinic for Children's Diseases Skopje

Skopje, 1000, North Macedonia

Location

Klinisk forskningspost

Oslo, 0372, Norway

Location

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

Lublin, 20-093, Poland

Location

Uniwersyteckie Centrum Kliniczne WUM

Warsaw, 02-091, Poland

Location

Clinic of Haematology, Fundeni Clinical Institute

Bucharest, 022328, Romania

Location

Spitalul Clinic de Urgenta pentru Copii Cluj Napoca

Cluj-Napoca, 400177, Romania

Location

Spitalul Clinic Judetean De Urgenta Bihor

Oradea, 410469, Romania

Location

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng, 2193, South Africa

Location

Hospital Virgen de la Arrixaca - Hematología

El Palmar, Murcia, 30120, Spain

Location

Hospital Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Regional Universitario de Málaga

Málaga, 29009, Spain

Location

Koagulationscentrum

Gothenburg, 413 46, Sweden

Location

Sunpasitthiprasong Hospital_Pediatrics Department

Ubon Ratchathani, Mueang Distirct,, 34000, Thailand

Location

King Chulalongkorn Memorial Hospital_Bangkok_0

Bangkok, 10330, Thailand

Location

King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology

Bangkok, 10330, Thailand

Location

Ramathibodi Hospital

Bangkok, 10400, Thailand

Location

Siriraj Hospital - Hematology and Oncology

Bangkok, 10700, Thailand

Location

Gazi University

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

Location

Gazi Üniversitesi Hastanesi- Hematoloji

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

Location

Acibadem Adana Hastanesi

Adana, 01130, Turkey (Türkiye)

Location

Acıbadem Adana Hastanesi-Hematoloji

Adana, 01130, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi

Izmir, 35100, Turkey (Türkiye)

Location

Ege Üniversitesi Hastanesi- Hematoloji

Izmir, 35100, Turkey (Türkiye)

Location

Ondokuz Mayis University Medical Faculty Ped. Haematology

Samsun, 55139, Turkey (Türkiye)

Location

Ondokuz Mayıs Üniversitesi Hastanesi - Hematoloji

Samsun, 55139, Turkey (Türkiye)

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

University Hospitals Bristol & Weston NHS Foundation Trust

Bristol, BS2 8BJ, United Kingdom

Location

Evelina London Children's Hospital - Haemophilia

London, SE1 7EH, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N 3HR, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

concizumab

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: November 15, 2021
• First Posted: November 26, 2021
• Study Start: March 24, 2022
• Primary Completion: April 21, 2026
• Study Completion (Estimated): November 2, 2029
• Last Updated: December 30, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

JP (2); ES (4); IT (4); LE (2); TH (5); SE (1); TU (8); GB (4); PL (4); FR (3); IN (11); ZA (1); MY (4); RO (3); US (19); LI (1); NO (1); GR (3); AL (2); CA (2); BU (3); BO (2)