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

September 7, 2026 updated by: Novo Nordisk A/S

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 seven 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 (31 December 2026 at the latest). 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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
134

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Available

Available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Algeria
      • Algiers, Algeria, 16000
        • Haematology and Blood Bank Department
      • Constantine, Algeria, 25000
        • CHU Constantine BEN BADIS/ Hematology department
  • Australia
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • The Alfred
      • Parkville, Victoria, Australia, 3052
        • The Royal Children's Hospital
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
  • Austria
      • Vienna, Austria, 1090
        • Klin. Abt. f. Hämatologie und Hämostaseologie, AKH Wien
  • Bulgaria
      • Sofia, Bulgaria, 1527
        • UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology
  • Canada
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Hamltn Hth Sci/McMstr Child Hosp
  • Croatia
      • Zagreb, Croatia, 10 000
        • KBC Zagreb, Rebro, Hemofilija centar
      • Zagreb, Croatia, 10000
        • KBC Zagreb, Zavod za pedijatrijsku hematologiju
      • Zagreb, Croatia, 10 000
        • KBC Zagreb_Hematology
  • Czechia
      • Prague, Czechia, 12000
        • Ustav Hematologie a krevni tranfuze
      • Prague, Czechia, 150 18
        • Fakultní nemocnice Motol a Homolka
  • Denmark
      • Aarhus N, Denmark, 8200
        • Skejby Blodsygdomme, blødercentret
  • France
      • Bron, France, 69500
        • Hospices Civils de Lyon- Hopital Louis Pradel
      • Clermont-Ferrand, France, 63100
        • Centre Hospitalier de Clermont-Ferrand-Hopital Estaing
      • Le Kremlin-Bicêtre, France, 94270
        • Ap-Hp-Hopital de Bicetre
      • Rennes, France, 35000
        • Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou
  • India
      • New Delhi, India, 110029
        • All India Institute of Medical Sciences_New Dehli
    • 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
      • Ranipet, Tamil Nadu, India, 632517
        • CMCV
  • Italy
      • Castelfranco Veneto, Italy, 31033
        • Oncoematologia IOV
      • Castelfranco Veneto, Italy, 31033
        • Istituto Oncologico Veneto - Oncoematologia IOV
      • Milan, 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
      • Verona, Italy, 37126
        • Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Donna Bambino Borgo Trento - U.O.C. Oncoematologia Pediatrica
    • FI
      • Florence, 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
      • Kagoshima, Japan, 890-8760
        • Kagoshima City Hospital_Pediatrics
      • Kanagawa, Japan, 216-8511
        • St. Marianna University School of Medicine Hospital_Pediatrics
      • Mie, Japan, 514-8507
        • Mie University Hospital_Dept.of Blood Trans.Med & Cell
      • Nara, Japan, 634-8522
        • Nara Medical University Hospital_Pediatrics
      • Saitama, Japan, 350-0495
        • Saitama Medical Univ. Hospital_Dep of Int Med, Cent for Hemo
      • Tokyo, Japan, 167-0035
        • Ogikubo Hospital_Pediatries & Blood
  • Malaysia
      • Ampang, Selangor, Malaysia, 68000
        • Hospital Ampang
    • Pulau Pinang
      • George Town, Pulau Pinang, Malaysia, 10450
        • Hospital Pulau Pinang_Georgetown, Penang
    • Sabah
      • Kota Kinabalu, Sabah, Malaysia, 88586
        • Hospital Queen Elizabeth 1
    • Selangor
      • Ampang, Selangor, Malaysia, 68000
        • Hospital Ampang
  • Mexico
    • Nuevo León
      • Monterrey, Nuevo León, Mexico, 64460
        • Hospital Universitario Dr. Jose Eleuterio González
  • Norway
      • Oslo, Norway, 0027
        • Rikshospitalet - avdeling for blodsykdommer
  • Poland
      • Lublin, Poland, 20-081
        • SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
      • Lublin, Poland, 20-081
        • Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
      • Wroclaw, Poland, 50-367
        • Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
    • Greater Poland Voivodeship
      • Poznan, Greater Poland Voivodeship, Poland, 60-569
        • Uniwersytecki Szpital Kliniczny W Poznaniu
    • Lesser Poland Voivodeship
      • Krakow, Lesser Poland Voivodeship, Poland, 30-688
        • Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
    • Masovian Voivodeship
      • Warsaw, Masovian Voivodeship, Poland, 02-776
        • Instytut Hematologii i Transfuzjologii
  • Portugal
      • Porto, Portugal, 4200-319
        • ULS São João, E.P.E._H.São João_Imunohemoterapia
  • Russia
      • Krasnodar, Russia, 350007
        • Children Regional Clinical Hospital
      • Moscow, Russia, 119049
        • Morozovskaya municipal children hospital
      • Moscow, Russia, 125167
        • National Medical Research institution of haemotology
      • Petrozavodsk, Russia, 185019
        • Republican Hospital n.a. V. A. Baranov
      • Saint Petersburg, Russia, 191186
        • City out-patient clinic 37, City Hemophilia Centre
  • Serbia
      • Belgrade, Serbia, 11070
        • Institute for Mother and Child Health Care of Serbia
      • Belgrade, Serbia, 11000
        • Clinical Centre of Serbia, Institute for Haematology
      • Kragujevac, Serbia, 34000
        • University Clinical Centre Kragujevac
      • Novi Sad, Serbia, 21000
        • Clinical Centre of Vojvodina
      • Novi Sad, Serbia, 21000
        • Institute for Health Care of Children and Adolescents
  • 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
  • South Korea
      • Daejeon, South Korea, 35233
        • Daejeon Eulji Medical Center, Eulji University
      • Seoul, South Korea, 03722
        • Severance Hospital, Yonsei University Health System
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Vall d'hebrón
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz
      • Málaga, Spain, 29010
        • Hospital Regional Universitario de Málaga
      • Málaga, Spain, 29010
        • Hospital Universitario Regional de Málaga
      • Oviedo, Spain, 33011
        • Hospital Central de Asturias
      • Oviedo, Spain, 33011
        • Hospital Univ. Central de Asturias
      • Seville, Spain, 41013
        • Hospital Virgen Del Rocio
  • Sweden
      • Malmö, Sweden, 214 28
        • Skåne US - Koagulationsmottagning
      • Solna, Sweden, 171 64
        • KS Solna - Koagulationsmottagningen
  • Thailand
      • Bangkok, Thailand, 10400
        • Ramathibodi Hospital_Department of Haematology
      • Chiang Mai, Thailand, 50200
        • Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology
    • Mueang Distirct,
      • Ubon Ratchathani, Mueang Distirct,, Thailand, 34000
        • Sunpasitthiprasong Hospital_Pediatrics Department
  • Turkey (Türkiye)
      • Adana, Turkey (Türkiye), 01130
        • Acıbadem Adana Hastanesi-Hematoloji
      • Adana, Turkey (Türkiye), 01130
        • Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji
    • Capa-ISTANBUL
      • Capa-ISTANBUL, Capa-ISTANBUL, Turkey (Türkiye), 34093
        • İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
    • Konyaaltı/ Antalya
      • Antalya, Konyaaltı/ Antalya, Turkey (Türkiye), 01010
        • Akdeniz Üniversitesi Hastanesi- Hematoloji
  • Ukraine
      • Kyiv, Ukraine, 01135
        • National specialized children's hospital 'OHMATDYT' - Haemostasis centre
      • Lviv, Ukraine, 79044
        • Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
        • Queen Elizabeth Hospital, Birmingham - Haemophilia
      • London, United Kingdom, WC1N 3HR
        • Great Ormond Street Hospital for Children
      • Manchester, United Kingdom, M13 9WL
        • Manchester Royal Infirmary_Manchester
      • Nottingham, United Kingdom, NG7 2UH
        • Queen's Medical Centre - Haemophilia Comprehensive Care Centre
      • 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
        • Children's Healthcare Atlanta
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia-Thromb Ctr
    • Missouri
      • St 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Time Frame: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2): From week 0 up until the primary analysis cut-off (at least 32 weeks)
Rate of treated spontaneous and traumatic bleeding episodes is presented. The observation period used for reporting this endpoint is on-treatment without ancillary therapy excl. data on initial regimen for participants exposed to both regimens (OTwoATexIR). It is defined as the time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of annualised bleeding rate (ABR). Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2): From week 0 up until the primary analysis cut-off (at least 32 weeks)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Rate of Treated Spontaneous Bleeding Episodes
Time Frame: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of treated spontaneous bleeding episodes is presented. The observation period used for reporting this endpoint is OTwoATexIR. It is defined as the time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of Treated Spontaneous and Traumatic Joint Bleeds
Time Frame: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of treated spontaneous and traumatic joint bleeds is presented. Observation period used for reporting this endpoint is OTwoATexIR. It is defined as time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of Treated Spontaneous and Traumatic Target Joint Bleeds
Time Frame: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of treated spontaneous and traumatic target joint bleeds is presented. Observation period used for reporting the endpoint is OTwoATexIR. It is defined as time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in terms of ABR. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Change in 36-item Short Form Health Survey (SF-36v2) Bodily Pain
Time Frame: Baseline (week 0), Week 24
Change in 36-item SF-36v2 bodily pain from baseline (week 0) to week 24 is presented. SF-36 v2 Health Survey is 36-item generic patient-reported outcome (PRO) instrument measuring health-related quality of life and general health status across disease areas. SF-36 v2 scores are norm-based scores, i.e. transformed to a scale where the 2009 US general population has a mean of 50 and an SD of 10. Lowest and highest scores for bodily pain are 21.68 to 62.0. Higher values indicate better functional health and well-being. Observation period for reporting the data is on-treatment without data on initial regimen (OTexIR) which is defined as time period where participants are considered affected by on demand treatment or treatment with new concizumab dosing regimen. Week 0 is defined as time of randomisation to on-demand administration or start of new concizumab dosing regimen.
Baseline (week 0), Week 24
Change in SF36v2 Physical Functioning
Time Frame: Baseline (week 0), Week 24
Change in 36-item SF-36v2 physical functioning from baseline (week 0) to week 24 is presented. SF-36 v2 Health Survey is 36-item generic PRO instrument measuring health-related quality of life and general health status across disease areas. SF-36 v2 scores are norm-based scores, i.e. transformed to a scale where the 2009 US general population has a mean of 50 and an SD of 10. Lowest and highest scores for physical functioning are 19.26 to 57.54. Higher values indicate better functional health and well-being. Observation period used for reporting the data is OTexIR which is defined as time period where participants are considered affected by on demand treatment or treatment with new concizumab dosing regimen. Week 0 is defined as time of randomisation to on-demand administration or start of new concizumab dosing regimen.
Baseline (week 0), Week 24
Number of Thromboembolic Events
Time Frame: On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of thromboembolic events is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Thromboembolic Events
Time Frame: From week 0 to end of trial (week 167)
Number of thromboembolic events is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
From week 0 to end of trial (week 167)
Number of Hypersensitivity Type Reactions
Time Frame: On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of hypersensitivity type reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Hypersensitivity Type Reactions
Time Frame: From week 0 to end of trial (week 167)
Number of hypersensitivity type reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
From week 0 to end of trial (week 167)
Number of Injection Site Reactions
Time Frame: On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of injection site reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Injection Site Reactions
Time Frame: From week 0 to end of trial (week 167)
Number of injection site reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
From week 0 to end of trial (week 167)
Number of Participants With Antibodies to Concizumab
Time Frame: Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of participants with antibodies to concizumab is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Participants With Antibodies to Concizumab
Time Frame: From week 0 to end of trial (week 167)
Number of participants with antibodies to concizumab is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
From week 0 to end of trial (week 167)
Pre-dose (Trough) Concizumab Plasma Concentration (Ctrough)
Time Frame: Pre-dose (prior to concizumab administration at week 56)
Pre-dose (trough) concizumab plasma concentration is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Pre-dose (prior to concizumab administration at week 56)
Pre-dose Thrombin Peak
Time Frame: Pre-dose (prior to concizumab administration at week 56)
Pre-dose thrombin peak for concizumab is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Pre-dose (prior to concizumab administration at week 56)
Pre-dose Free Tissue Factor Pathway Inhibitor (TFPI) Concentration
Time Frame: Pre-dose (prior to concizumab administration at week 56)
Pre-dose free TFPI concentration for concizumab is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Pre-dose (prior to concizumab administration at week 56)
Maximum Concizumab Plasma Concentration (Cmax)
Time Frame: Week 24: Predose, 3 hours (h), 6h, 9h, 24h
Maximum concizumab plasma concentration is presented. The observation period used for reporting the endpoint is on OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Week 24: Predose, 3 hours (h), 6h, 9h, 24h
Area Under the Concizumab Plasma Concentration-time Curve (AUC)
Time Frame: Week 24: Predose, 3 hours (h), 6h, 9h, 24h
Area under the concizumab plasma concentration-time curve is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Week 24: Predose, 3 hours (h), 6h, 9h, 24h

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Reporting Anchor and Disclosure (1452), Novo Nordisk A/S

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 21, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 27, 2021

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 21, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 6, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 6, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 10, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 9, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 7, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NN7415-4311
  • 2018 (U.S. NIH Grant/Contract)
  • 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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