Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors (explorer7)

Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B With Inhibitors

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group, participants will get study medicine from the start of the study. In the other group, participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will get 1 injection with the study medicine every day under the skin. This participants will have to do themselves and can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for about six years. The length of time the participants will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (12 November 2025 at the latest). Participants will have to come to the clinic for up to 41 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months, depending on the group participants are in and approximately 8 weeks for the rest of the study. Participants will be asked to record information into an electronic diary during the study and may also be asked to wear an activity tracker.

Study Overview

• Status: Active, not recruiting
• Conditions: Haemophilia A With Inhibitors; Haemophilia B With Inhibitors
• Intervention / Treatment: Drug: Concizumab

• Study Type: Interventional
• Enrollment (Estimated): 136
• Phase: Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Algeria
  • Algiers, Algeria, 16000
    • Haematology and Blood Bank Department
  • Constantine, Algeria, 25000
    • CHU Constantine IBN BADIS/ Hematology department
• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • The Alfred
  • Western Australia
    • Murdoch, Western Australia, Australia, 6150
      • Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
• Austria
  • Wien, Austria, 1090
    • Klin. Abt. f. Hämatologie und Hämostaseologie, AKH Wien
• Bulgaria
  • Sofia, Bulgaria, 1527
    • UMHAT "Tsaritsa Yoanna-ISUL"
• Croatia
  • Zagreb, Croatia, 10 000
    • KBC Zagreb, Rebro, Hemofilija centar
  • Zagreb, Croatia, 10000
    • KBC Zagreb, Zavod za pedijatrijsku hematologiju
• Czechia
  • Praha, Czechia, 150 18
    • Fakultni Nemocnice V Motole
  • Praha 2, Czechia, 12000
    • Ustav Hematologie a krevni tranfuze
• Denmark
  • Aarhus N, Denmark, 8200
    • Skejby Blodsygdomme, blødercentret
• France
  • Bron, France, 69500
    • Hospices Civils de Lyon-Hopital Cardiologique Louis Pradel-1
  • Clermont-Ferrand, France, 63100
    • Centre Hospitalier de Clermont-Ferrand-Hopital Estaing
  • Le Kremlin-Bicetre, France, 94270
    • Ap-Hp-Hopital de Bicetre-1
  • Rennes, France, 35000
    • Centre Hospitalier Universitaire de Rennes - Hôpital Pontchaillou
• India
  • Karnataka
    • Bangalore, Karnataka, India, 560034
      • St. John's Medical college and Hospital
  • Maharashtra
    • Pune, Maharashtra, India, 411004
      • Sahyadri Super Speciality Hospital
    • Pune, Maharashtra, India, 411004
      • Sahyadri Speciality Hospital
  • New Delhi
    • New Dehli, New Delhi, India, 110029
      • All India Institute of Medical Sciences_New Dehli
  • Tamil Nadu
    • Ranipet, Tamil Nadu, India, 632 517
      • CMCV
• Italy
  • Castelfranco Veneto, Italy, 31033
    • Oncoematologia IOV
  • Milano, Italy, 20124
    • Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano
  • Udine, Italy, 33100
    • Azienda OU "S.Maria della Misericordia"
  • Verona, Italy, 37126
    • Ospedale Donna Bambino U.O.C. Oncoematologia Pediatrica
  • FI
    • Firenze, FI, Italy, 50134
      • Dipartimento di Ematologia Univ. Firenze
• Japan
  • Aichi, Japan, 466-8560
    • Nagoya University Hospital_Blood Transfusion
  • Kagoshima, Japan, 890-8760
    • Kagoshima City Hospital
  • Kanagawa, Japan, 216-8511
    • St. Marianna University School of Medicine Hospital_Pediatrics
  • Nara, Japan, 634-8522
    • Nara Medical University Hospital_Pediatrics
  • Saitama, Japan, 350-0495
    • Saitama Medical Univ. Hospital
  • Tokyo, Japan, 167-0035
    • Ogikubo Hospital_Pediatries & Blood
• Korea, Republic of
  • Daejeon, Korea, Republic of, 35233
    • Daejeon Eulji Medical Center, Eulji University
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital, Yonsei University Health System
• Malaysia
  • Ampang, Selangor, Malaysia, 68000
    • Hospital Ampang
  • Georgetown, Penang, Malaysia, 10450
    • Hospital Pulau Pinang_Georgetown, Penang
  • Sabah
    • Kota Kinabalu, Sabah, Malaysia, 88586
      • Hospital Queen Elizabeth 1
• Mexico
  • Nuevo León
    • Monterrey, Nuevo León, Mexico, 64460
      • Hospital Universitario Dr. Jose Eleuterio Gonzalez
• Norway
  • Oslo, Norway, 0027
    • Rikshospitalet - avdeling for blodsykdommer
• Poland
  • Lublin, Poland, 20-081
    • SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
  • Wroclaw, Poland, 50-367
    • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
  • Mazowieckie
    • Warszawa, Mazowieckie, Poland, 02-776
      • Intytut Hematologii i Transfuzjologii
  • Małopolskie
    • Kraków, Małopolskie, Poland, 30-688
      • Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
• Portugal
  • Porto, Portugal, 4200-319
    • Centro Hospitalar de São João_Porto
• Russian Federation
  • Krasnodar, Russian Federation, 350007
    • Children Regional Clinical Hospital
  • Moscow, Russian Federation, 119049
    • Morozovskaya municipal children hospital
  • Moscow, Russian Federation, 125167
    • National Medical Research institution of haemotology
  • Petrozavodsk, Russian Federation, 185019
    • Republican Hospital n.a. V. A. Baranov
  • Saint-Petersburg, Russian Federation, 191186
    • City out-patient clinic 37, City Hemophilia Centre
• Serbia
  • Kragujevac, Serbia, 34000
    • University Clinical Centre Kragujevac
• Slovakia
  • Bratislava, Slovakia, 851 07
    • Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz
• South Africa
  • Gauteng
    • Parktown, Johannesburg, Gauteng, South Africa, 2193
      • Charlotte Maxeke Johannesburg Academic Hospital
  • KwaZulu-Natal
    • Durban, KwaZulu-Natal, South Africa, 4013
      • Haematology Clinic
  • Limpopo
    • Polokwane, Limpopo, South Africa, 0699
      • Pietersburg Hospital
• Spain
  • Barcelona, Spain, 08035
    • Hospital Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Málaga, Spain, 29010
    • Hospital Universitario Regional de Malaga
  • Oviedo, Spain, 33011
    • Hospital Central de Asturias
  • Sevilla, Spain, 41013
    • Hospital Virgen del Rocío
• Sweden
  • Malmö, Sweden, 205 02
    • Koagulationsmottagning
  • Solna, Sweden, 171 64
    • Koagulationsmottagningen
• Thailand
  • Bangkok, Thailand, 10400
    • Ramathibodi Hospital_Bangkok_0
  • Chiang Mai, Thailand, 50200
    • Hematology and Oncology, Dept.of Pediatrics, CMU
  • Ubon Ratchathani, Thailand, 34000
    • Sunpasitthiprasong Hospital
• Turkey
  • Adana, Turkey, 01130
    • Acibadem Adana Hastanesi
  • Adana, Turkey, 01130
    • Cukurova Universitesi
  • Antalya, Turkey, 01010
    • Akdeniz Universitesi
  • Capa-ISTANBUL, Turkey, 34093
    • Istanbul University Oncology Institute
• Ukraine
  • Kyiv, Ukraine, 01135
    • National specialized children clinic "OHMATDYT"
  • Lviv, Ukraine, 79044
    • Institute of blood pathology and transfusion medicine
• United Kingdom
  • Birmingham, United Kingdom, B15 2TH
    • West Midlands Adult Comprehensive Care Haemophilia
  • London, United Kingdom, WC1N 3HR
    • Great Ormond Street Hospital for Children
  • Sheffield, United Kingdom, S10 2JF
    • Royal Hallamshire Hospital
• United States
  • California
    • Orange, California, United States, 92868
      • Center for Inherited Blood Dis
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Emory University_Atlanta_1
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia-Thromb Ctr
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine_St. Louis
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • St. Jude Affiliate Clinic at Novant Health Hemby Children's
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • TriStar Medical Group Children's Specialist
  • Texas
    • San Antonio, Texas, United States, 78229
      • University of Texas San Antonio

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
• Male aged 12 years or older at the time of signing informed consent.
• Congenital Haemophilia A or B of any severity with documented history of inhibitor (equal to or above 0.6 Bethesda Units (BU).
• Patient has been prescribed, or in need of, treatment with bypassing agents in the last 24 weeks prior to screening (for patients not previously enrolled in NN7415-4310 (explorer 4)).

Exclusion Criteria:

• Known or suspected hypersensitivity to any constituent of the trial product or related products.
• Known inherited or acquired coagulation disorder other than congenital haemophilia.
• Ongoing or planned Immune Tolerance Induction treatment.
• History of thromboembolic disease (includes 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.)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm 1: No prophylaxis; Haemophilia A with inhibitors (HAwI) and haemophilia B with inhibitors (HBwI) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension part, patients in arm 1 will receive daily concizumab subcutaneous (s.c., under the skin) injections.	Drug: Concizumab; Concizumab will be administered daily subcutaneously (s.c., under the skin). When patients are randomised to concizumab prophylaxis they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week 0: arm 2, 3 & 4) or visit 9a (week 24: arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (week 6: arm 2, 3 & 4) or 9a.3 (week 30: arm 1) and will be based on the concizumab exposure level measured at the previous visit 4a (week 4) or 9a.2 (week 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual bypassing product until visit 9a (week 24: end of main part for arm 1).
Experimental: Arm 2: Concizumab prophylaxis; HAwI and HBwI patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis.	Drug: Concizumab; Concizumab will be administered daily subcutaneously (s.c., under the skin). When patients are randomised to concizumab prophylaxis they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week 0: arm 2, 3 & 4) or visit 9a (week 24: arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (week 6: arm 2, 3 & 4) or 9a.3 (week 30: arm 1) and will be based on the concizumab exposure level measured at the previous visit 4a (week 4) or 9a.2 (week 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual bypassing product until visit 9a (week 24: end of main part for arm 1).
Experimental: Arm 3: Concizumab prophylaxis; The HAwI and HBwI patients enrolled into the concizumab phase 2 trial (NN7415-4310) at time of transfer will be offered enrolment into this trial. It is required that these patients are on concizumab prophylaxis up until enrolment into the trial. These patients will continue concizumab prophylaxis.	Drug: Concizumab; Concizumab will be administered daily subcutaneously (s.c., under the skin). When patients are randomised to concizumab prophylaxis they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week 0: arm 2, 3 & 4) or visit 9a (week 24: arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (week 6: arm 2, 3 & 4) or 9a.3 (week 30: arm 1) and will be based on the concizumab exposure level measured at the previous visit 4a (week 4) or 9a.2 (week 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual bypassing product until visit 9a (week 24: end of main part for arm 1).
Experimental: Arm 4: Concizumab prophylaxis; Patients previously on prophylaxis with by-passing agents and on-demand patients who are screened at a timepoint where the required number of patients in arms 1 and 2 have been randomised. These patients will, if eligible, be enrolled into the trial and will initiate concizumab prophylaxis at visit 2a (week 0).	Drug: Concizumab; Concizumab will be administered daily subcutaneously (s.c., under the skin). When patients are randomised to concizumab prophylaxis they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week 0: arm 2, 3 & 4) or visit 9a (week 24: arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (week 6: arm 2, 3 & 4) or 9a.3 (week 30: arm 1) and will be based on the concizumab exposure level measured at the previous visit 4a (week 4) or 9a.2 (week 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual bypassing product until visit 9a (week 24: end of main part for arm 1).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of treated spontaneous and traumatic bleeding episodes	This will be presented as 'count of episodes'.	On demand (arm 1): From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks). Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pre-dose (trough) concizumab plasma concentration (Ctrough)	This will be measured in 'ng/mL'.	Prior to the concizumab administration at week 24 (after restart)
Pre-dose thrombin peak	This will be measured in 'nmol/L'.	Prior to the concizumab administration at week 24 (after restart)
Pre-dose free tissue factor pathway inhibitor (TFPI) concentration	This will be measured in 'ng/mL'.	Prior to the concizumab administration at week 24 (after restart)
Maximum concizumab plasma concentration (Cmax)	This will be measured in 'ng/mL'.	From 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart)
Area under the concizumab plasma concentration-time curve (AUC)	This will be measured in 'ng*hr/mL'.	From 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart)
Change in 36 Item short form health survey version 2 (SF36v2) bodily pain	This will be presented as 'score on a scale'. The bodily pain subscale of the SF-36v2 questionnaire consists of 2 items. Subscale scores range from 0-100 and are transformed into population norm adjusted T-scores. A higher score indicates a better outcome on this subscale.	From start of treatment (week 0) until week 24. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
Change in SF36v2 physical functioning	This will be presented as 'score on a scale'. The physical function subscale of the SF-36v2 questionnaire consists of 1 item with 10 subitems. Subscale scores range from 0-100 and are transformed into population norm adjusted T-scores. A higher score indicates a better outcome on this subscale.	From start of treatment (week 0) until week 24. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
Number of treated spontaneous bleeding episodes	This will be presented as 'count of episodes'.	On demand (arm 1): From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of treated spontaneous and traumatic joint bleeds	This will be presented as 'count of episodes'.	On demand (arm 1): From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of treated spontaneous and traumatic target joint bleeds	This will be presented as 'count of episodes'.	On demand (arm 1): From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of thromboembolic events	This will be presented as 'count of thromboembolic events'. On demand (arm 1 main part): From randomisation to on demand treatment up until start of concizumab treatment. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of the new concizumab dosing regimen (week 25) up until the primary analysis cut-off. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day). Week 0, after the pause is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.	Timeframe is presented under 'outcome measure description'
Number of hypersensitivity type reactions	This will be presented as 'count of hypersensitivity type reactions'. On demand (arm 1 main part): From randomisation to on demand treatment up until start of concizumab treatment. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of the new concizumab dosing regimen (week 25) up until the primary analysis cut-off. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day). Week 0, after the pause is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.	Timeframe is presented under 'outcome measure description'
Number of injection site reactions	This will be presented as 'count of injection site reactions'. On demand (arm 1 main part): From randomisation to on demand treatment up until start of concizumab treatment. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of the new concizumab dosing regimen (week 25) up until the primary analysis cut-off. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day). Week 0, after the pause is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.	Timeframe is presented under 'outcome measure description'
Number of patients with antibodies to concizumab	This will be presented as 'count of patients'. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of the new concizumab dosing regimen (week 25) up until the primary analysis cut-off. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day). Week 0, after the pause is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.	Timeframe is presented under 'outcome measure description'
Number of thromboembolic events	This will be presented as 'count of thromboembolic events'. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day).	Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 280 weeks).
Number of hypersensitivity type reactions	This will be presented as 'count of hypersensitivity type reactions'. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day).	Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 280 weeks).
Number of injection site reactions	This will be presented as 'count of injection site reactions'. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day).	Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 280 weeks).
Number of patients with antibodies to concizumab	This will be presented as 'count of patients'. Week 0, before the pause is defined as the time of randomisation to on-demand administration or time of start of the initial concizumab dosing regimen (0.25 mg/kg/day).	Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 280 weeks).

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Reporting Anchor and Disclosure (1452), Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): October 21, 2019
• Primary Completion (Actual): December 27, 2021
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: September 6, 2019
• First Submitted That Met QC Criteria: September 6, 2019
• First Posted (Actual): September 10, 2019

Study Record Updates

• Last Update Posted (Actual): March 26, 2024
• Last Update Submitted That Met QC Criteria: March 25, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: NN7415-4311; U1111-1225-9670 (Other Identifier: World Health Organization (WHO)); 2018-004889-34 (Registry Identifier: European Medicines Agency (EudraCT))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No