Haemophilia A and B With and Without InhibitorsPhase 3Explorer10NCT05135559
A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
A Phase 3 study testing Concizumab in people with Haemophilia A and B With and Without Inhibitors, run by Novo Nordisk A/S at 91 sites in 24 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A and B With and Without Inhibitors
- What is being tested?
- Concizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 153 participants
- Where
- 91 sitesin 24 countries
- Study dates
- 24 Mar 2022to 2 Nov 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
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- 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.
Who cannot join
- 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).
Trial locations and contacts
91 sites in 24 countries.
91 sites in 24 countries
United States19 sites
Augusta Univ/Childrens Hosp-GA
Augusta, Georgia, 30912
University of Colorado Hospital
Aurora, Colorado, 80045
Pediatrics Hematology/Oncology Clinic Battle Building
Charlottesville, Virginia, 22908
Childrens Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Cook Children's Hospital-Hematology-Oncology
Fort Worth, Texas, 76104
ECU Sickle Cell Comp Clinic
Greenville, North Carolina, 27834
Texas Children's Hospital_Houston
Houston, Texas, 77030
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260
The Children's Mercy Hospital
Kansas City, Missouri, 64108
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
Children's Hosp-New Orleans
New Orleans, Louisiana, 70118
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
Children's Nebraska
Omaha, Nebraska, 68114
Arnold Palmer Children's Hospital
Orlando, Florida, 32806
Nemours Child Orlando Hem/Onc.
Orlando, Florida, 32827
St Christopher Hosp for Child
Philadelphia, Pennsylvania, 19134
Rady Childrens Hosp San Diego
San Diego, California, 92123
Memorial Health University Medical Center
Savannah, Georgia, 31404
India11 sites
S.C.B. Medical College
Cuttack, Odisha, 753007
Guwahati Medical College
Guwahati, Assam, 781032
J K Lon Hospital
Jaipur, Rajasthan, 302004
SSSH_Dept. of Clinical Haematology & Haemato Oncology
Kolhāpur, Maharashtra, 416005
SGPGI
Lucknow, Uttart Pradesh, 226014
Seth GS Medical College & KEM Hospital
Mumbai, Maharashtra, 400012
K.J Somaiya Hospital and Research Centre
Mumbai, Maharashtra, 400022
MCGM - Comprehensive Thalassemia Care
Mumbai, Maharashtra, 400066
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, 201303
Sahyadri Super Speciality Hospital
Pune, Maharashtra, 411004
Nirmal Hospital Pvt. Ltd.
Surat, Gujarat, 395002
Turkey (Türkiye)8 sites
Acibadem Adana Hastanesi
Adana, 01130
Acıbadem Adana Hastanesi-Hematoloji
Adana, 01130
Gazi University
Ankara, Beşevler/Ankara, 06500
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara, Beşevler/Ankara, 06500
Ege Universitesi Tip Fakultesi
Izmir, 35100
Ege Üniversitesi Hastanesi- Hematoloji
Izmir, 35100
Ondokuz Mayis University Medical Faculty Ped. Haematology
Samsun, 55139
Ondokuz Mayıs Üniversitesi Hastanesi - Hematoloji
Samsun, 55139
Thailand5 sites
King Chulalongkorn Memorial Hospital_Bangkok_0
Bangkok, 10330
King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology
Bangkok, 10330
Ramathibodi Hospital
Bangkok, 10400
Siriraj Hospital - Hematology and Oncology
Bangkok, 10700
Sunpasitthiprasong Hospital_Pediatrics Department
Ubon Ratchathani, Mueang Distirct,, 34000
Italy4 sites
A.O.U policlinico "G. Rodolico-San Marco"
Catania, 95123
Dipartimento di Ematologia Univ. Firenze
Florence, 50134
Azienda Ospedaliera di Padova _
Padova, 35128
Azienda Ospedaliera-Universitaria Parma
Parma, 43126
Malaysia4 sites
Hospital Pulau Pinang
George Town, Pulau Pinang, 10450
Hospital Tunku Azizah
Kampung Baru, Kuala Lumpur, 50300
Hospital Sultanah Nur Zahirah
Kuala Terengganu, Terengganu, 20400
Sarawak General Hospital
Kuching, Sarawak, 93586
Poland4 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, 20-093
Uniwersyteckie Centrum Kliniczne WUM
Warsaw, 02-091
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, Lower Silesian Voivodeship, 50-556
Spain4 sites
Hospital Vall d'Hebron
Barcelona, 08035
Hospital Virgen de la Arrixaca - Hematología
El Palmar, Murcia, 30120
Hospital Universitario La Paz
Madrid, 28046
Hospital Regional Universitario de Málaga
Málaga, 29009
United Kingdom4 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
University Hospitals Bristol & Weston NHS Foundation Trust
Bristol, BS2 8BJ
Evelina London Children's Hospital - Haemophilia
London, SE1 7EH
Great Ormond Street Hospital for Children
London, WC1N 3HR
Bulgaria3 sites
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv, 4002
UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology
Sofia, 1527
UMHAT Sveta Marina EAD, Clinic of Pediatric Clinical Hematology and Oncology
Varna, 9010
France3 sites
Centre Hospitalier Metropole Savoie
Chambéry, 73000
Ap-Hp-Hopital de Bicetre-1
Le Kremlin-Bicêtre, 94275
AP-HP-HOPITAL NECKER_Service d'hématologie
Paris, 75015
Greece3 sites
Aghia Sophia Childrens' Hospital
Athens, GR-11527
'Ippokrateio' General Hospital of Thessaloniki
Thessaloniki, 54642
'Ippokrateio' General Hospital of Thessaloniki
Thessaloniki, GR 54642
Romania3 sites
Clinic of Haematology, Fundeni Clinical Institute
Bucharest, 022328
Spitalul Clinic de Urgenta pentru Copii Cluj Napoca
Cluj-Napoca, 400177
Spitalul Clinic Judetean De Urgenta Bihor
Oradea, 410469
Algeria2 sites
Haematology and Blood Bank Department
Algiers, 16000
CHU Constantine BEN BADIS/ Hematology department
Constantine, 25000
Bosnia and Herzegovina2 sites
University Clinical Center of Republic Srpska (545)
Banja Luka, 78000
University Clinical Centre Tuzla
Tuzla, 75000
Canada2 sites
McMaster Children's Hospital
Hamilton, Ontario, L8N 3Z5
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4
Japan2 sites
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, 216-8511
Saitama Children's Med Centre_Hematology-Oncology
Saitama, 330-8777
Lebanon2 sites
Saint George Hospital University Medical Center
Beirut, 961
Hospital Nini
Tripoli, 1434
Lithuania1 site
Centre of Oncology and Hematology, Vilnius University
Vilnius, LT-08406
North Macedonia1 site
PHI University Clinic for Children's Diseases Skopje
Skopje, 1000
South Africa1 site
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, 2193
About this study
Official title: Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors
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.
- Being tested
- Concizumab
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ConcizumabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Concizumab-naïve patients | Experimental | Concizumab-naïve participants below 12 years of age at the time of consent/assent Receives: 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 Receives: Drug: Concizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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)
Measured: 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)
Count of episode(s)
Measured: 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
Count of episode(s)
Measured: 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
Count of episode(s)
Measured: 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
Count of episode(s)
Measured: 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)
Count of episode(s)
Measured: 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 bleeding episodes
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated joint bleeding episodes
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints
Count of episode(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Concizumab-naïve pateints - Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined
Count of event(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of thromboembolic events, reported both separately for inhibitor and non-inhibitor patients and combined
Count of event(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of hypersensitivity type reactions, reported both separately for inhibitor and non-inhibitor patients and combined
Count of event(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of injection site reactions, reported both separately for inhibitor and non-inhibitor patients and combined
Count of event(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of patients who develop antibodies to concizumab - yes/no, reported both separately for inhibitor and non-inhibitor patients and combined
Count of patient(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined
Count of event(s)
Measured: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Concizumab plasma concentrations prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined
Measured in ng/mL
Measured: Week 32
Peak thrombin generation prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined
Measured in nM
Measured: Week 32
Free TFPI concentration prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined
Measured in ng/mL
Measured: Week 32
Pre-dose (trough) concizumab plasma concentration (Ctrough), reported both separately for inhibitor and non-inhibitor patients and combined
Measured in ng/mL
Measured: Prior to the concizumab administration at week 20
Maximum concizumab plasma concentration (Cmax), reported both separately for inhibitor and non-inhibitor patients and combined
Measured in ng/mL
Measured: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20
Area under the concizumab plasma concentration-time curve (AUC), reported both separately for inhibitor and non-inhibitor patients and combined
Measured in ng*hr/mL
Measured: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2021
- Study start24 Mar 2022
- Primary completion21 Apr 2026
- Study completion2 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia A and B With and Without InhibitorsStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 153 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency (dept. 2834), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05135559
- Sponsor's protocol ID
- NN7415-4616
- Other IDs
- U1111-1247-7330, 2020-000504-11, jRCT2031220097
- Acronym
- Explorer10
- Status last verified
- Sep 2026
- First submitted
- 15 Nov 2021
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors
- ClinicalTrials.gov ID
- NCT05135559
- Acronym / sponsor ID
- Explorer10 / NN7415-4616
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 153 participants (estimated)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 24 Mar 2022 (actual)
- Primary completion
- 21 Apr 2026 (actual)
- Study completion
- 2 Nov 2029 (estimated)
- First posted
- 26 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Concizumab-naïve patients | Experimental | Drug: ConcizumabConcizumab-naïve participants below 12 years of age at the time of consent/assent |
| Patients coming from compassionate use | Experimental | Drug: ConcizumabPatients previously treated with concizumab via compassionate use, either on an individual patient basis or through the concizumab compassionate use programme NN7415-4807 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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)
- For arm 1 only: Patients without inhibitors (haemophilia A or haemophilia B)
Key exclusion criteria (4 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ConcizumabParticipants 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. | Drug | Concizumab-naïve patients, Patients coming from compassionate use |
Primary outcome
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
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Count of episode(s)
For non-inhibitor patients treated on demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Count of episode(s)
25 secondary outcomes
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)
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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)
Time frame: 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 bleeding episodes
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated joint bleeding episodes
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Concizumab-naïve pateints - Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of thromboembolic events, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of hypersensitivity type reactions, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of injection site reactions, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of patients who develop antibodies to concizumab - yes/no, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
Concizumab plasma concentrations prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: Week 32
Peak thrombin generation prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: Week 32
Free TFPI concentration prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: Week 32
Pre-dose (trough) concizumab plasma concentration (Ctrough), reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: Prior to the concizumab administration at week 20
Maximum concizumab plasma concentration (Cmax), reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20
Area under the concizumab plasma concentration-time curve (AUC), reported both separately for inhibitor and non-inhibitor patients and combined
Time frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Transparency (dept. 2834) study director
Novo Nordisk A/S
91 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Concizumab in people with Haemophilia A and B With and Without Inhibitors, run by Novo Nordisk A/S at 91 sites in 24 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 91 sites in United States, India, Turkey (Türkiye), Thailand, Italy and 19 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.