Haemophilia A Without InhibitorsPhase 3explorer8NCT04082429
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
A Phase 3 study testing Concizumab in people with Haemophilia A Without Inhibitors and Haemophilia B Without Inhibitors, run by Novo Nordisk A/S at 113 sites in 33 countries.
Trial summary
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Haemophilia A Without Inhibitors, Haemophilia B 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
- 156 participants
- Where
- 113 sitesin 33 countries
- Study dates
- 13 Nov 2019to 21 Feb 2028
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%).
Who cannot join
- Known or suspected hypersensitivity to any constituent of the trial product or related products.
- Known inherited or acquired coagulation disorder other than congenital haemophilia.
- Presence of confirmed inhibitors 0.6 BU or greater at screening.
- 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.)
Trial locations and contacts
113 sites in 33 countries.
113 sites in 33 countries
Japan14 sites
Nagoya University Hospital_Blood Transfusion
Aichi, 466-8560
Hiroshima University Hospital, Hematology
Hiroshima, 734-8551
Hiroshima University Hospital_Hematology
Hiroshima, 734-8551
Hyogo prefectural kobe children's hospital Dept. of Haem and Onclogy
Hyōgo, 654-0047
Hyogo prefectural kobe children's hospital
Hyōgo, 654-0047
Itoigawa sogo Hospital_Department of Pediatrics
Niigata, 941-8502
Osaka National Hospital_Dept. of Infectious diseases
Osaka, 546-0006
Saitama Children's Med Centre_Hematology-Oncology
Saitama, 330-8777
Saitama Medical Univ. Hospital_Dep of Int Med, Cent for Hemo
Saitama, 350-0495
Saitama Medical Univ. Hospital
Saitama, 350-0495
Shizuoka Children's Hospital, Hematology-Oncology
Shizuoka, 420-8660
Shizuoka Children's Hospital
Shizuoka, 420-8660
National Center for Child Health and Development_Hematology
Tokyo, 157-8535
Ogikubo Hospital_Pediatries & Blood
Tokyo, 167-0035
United States12 sites
Center for Blood Disorders Augusta University
Augusta, Georgia, 30912
Novant Hlth Vasc Ins Charlotte
Charlotte, North Carolina, 28204
Children's Hospital of Michigan
Detroit, Michigan, 48201
M.S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260
University of Iowa_Iowa City
Iowa City, Iowa, 52242
Michigan State University
Lansing, Michigan, 48911
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
Versiti, CCBD
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center_Nashville_0
Nashville, Tennessee, 37212
Center for Inherited Blood Disorders
Orange, California, 92868
University of Texas San Antonio
San Antonio, Texas, 78229
Turkey (Türkiye)9 sites
Acıbadem Adana Hastanesi-Hematoloji
Adana, 01130
Gazi University
Ankara, Beşevler/Ankara, 06500
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara, Beşevler/Ankara, 06500
Hacettepe University Medical Faculty
Ankara, 06230
Hacettepe Üniversitesi Hastanesi- Endokrinoloji
Ankara, 06230
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-ISTANBUL, Capa-ISTANBUL, 34093
Trakya Üniversitesi Tıp Fakültesi Hastanesi-Hematoloji
Edirne, 22030
Ege Üniversitesi Hastanesi- Hematoloji
Izmir, 35100
Ondokuz Mayıs Üniversitesi Hastanesi - Hematoloji
Samsun, 55139
France6 sites
Centre Hospitalier Regional Et Universitaire de Brest-Hopital de La Cavale Blanche
Brest, 29200
CHU de Caen - Côte de Nacre
Caen, 14033
Hopital de Bicetre
Le Kremlin-Bicêtre, 94270
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, 44093
Hôpital Pontchaillou
Rennes, 35033
Centre Hospitalier Universitaire de Saint Etienne-Hopital Nord
Saint-Priest-en-Jarez, 42270
Poland6 sites
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
Krakow, Lesser Poland Voivodeship, 30-688
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
Lublin, 20-081
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, 20-093
Uniwersytecki Szpital Kliniczny W Poznaniu
Poznan, Greater Poland Voivodeship, 60-569
Instytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, 02-776
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Wroclaw, 50-367
India5 sites
St. John's Medical college and Hospital
Bangalore, Karnataka, 560034
J K Lon Hospital
Jaipur, Rajasthan, 302004
Sahyadri Speciality Hospital
Pune, Maharashtra, 411004
Sahyadri Super Speciality Hospital
Pune, Maharashtra, 411004
CMCV
Ranipet, Tamil Nadu, 632517
Italy5 sites
Dipartimento di Ematologia Univ. Firenze
Florence, FI, 50134
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano
Milan, MI, 20124
Policlinico Umberto I Sezione Ematologia
Roma, 00161
A.O.U Città Salute Scienza Torino - Ospedale Molinette
Torino, 10126
A.O.U Città Salute Scienza Torino
Torino, 10126
Russia5 sites
Children Regional Clinical Hospital
Krasnodar, 350007
Morozovskaya municipal children hospital
Moscow, 119049
National Medical Research institution of haemotology
Moscow, 125167
Republican Hospital n.a. V. A. Baranov
Petrozavodsk, 185019
City out-patient clinic 37, City Hemophilia Centre
Saint Petersburg, 191186
South Korea5 sites
Daejeon Eulji Medical Center, Eulji University
Daejeon, 35233
Daejeon Eulji University Hospital
Daejeon, 35233
Jeju National University Hospital
Jeju City, 63241
Jeju National University Hospital
Jeju-do, 63241
Severance Hospital, Yonsei University Health System
Seoul, 03722
Spain5 sites
Hospital Universitario La Paz
Madrid, 28046
Hospital Regional Universitario de Málaga
Málaga, 29010
Hospital Univ. Central de Asturias
Oviedo, 33011
Hospital Virgen del Rocío
Seville, 41013
Hospital Universitario y Politécnico La Fe
Valencia, 46026
Australia4 sites
The Alfred
Melbourne, Victoria, 3004
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
Murdoch, Western Australia, 6150
Royal Children's Hospital
Parkville, Victoria, 3052
The Royal Children's Hospital
Parkville, Victoria, 3052
Serbia4 sites
Clinical Centre of Serbia, Institute for Haematology
Belgrade, 11000
Institute for Mother and Child Health Care of Serbia
Belgrade, 11070
University Clinical Centre Kragujevac
Kragujevac, 34000
Clinical Centre of Vojvodina
Novi Sad, 21000
United Kingdom4 sites
Belfast City Hospital
Belfast, BT9 7AB
Royal Free Haemophilia Comprehensive Care Center
London, NW3 2QG
Royal Free Hospital - Haemophilia
London, NW3 2QG
Royal Hallamshire Hospital
Sheffield, S10 2JF
Algeria2 sites
Haematology and Blood Bank Department
Algiers, 16000
CHU Constantine BEN BADIS/ Hematology department
Constantine, 25000
Bulgaria2 sites
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
Canada2 sites
Hamltn Hth Sci/McMstr Child Hosp
Hamilton, Ontario, L8N 3Z5
Eastern Health Authority
St. John's, Newfoundland and Labrador, A1B 3V6
Germany2 sites
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn, 53127
Universität des Saarlandes - Hämostaseologie und Transfusionsmedizin
Homburg, 66424
Lithuania2 sites
Children Oncohaematology department Children's Hospital,
Vilnius, 08406
Vilnius University hospital Santaros klinikos
Vilnius, LT-08661
Malaysia2 sites
Hospital Ampang
Ampang, Selangor, 68000
Hospital Ampang
Ampang, Selangor, 68000
South Africa2 sites
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, 2193
Pietersburg Hospital
Polokwane, Limpopo, 0699
Sweden2 sites
Skåne US - Koagulationsmottagning
Malmö, 214 28
Koagulationsmottagningen
Solna, 171 64
Ukraine2 sites
National specialized children's hospital 'OHMATDYT' - Haemostasis centre
Kyiv, 01135
Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery
Lviv, 79044
Bosnia and Herzegovina1 site
University Clinical Center of Republic Srpska (205)
Banja Luka, 78000
Israel1 site
Sheba MC The Israeli National Hemophilia Center
Tel HaShomer, 52621
Mexico1 site
Hospital Universitario Dr. José Eleuterio González
Monterrey, Nuevo León, 64460
Portugal1 site
ULS São João, E.P.E._H.São João_Imunohemoterapia
Porto, 4200-319
Switzerland1 site
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
Zurich, 8091
About this study
Official title: Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B Without Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without 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 have to inject themselves with the study medicine 1 time every day under the skin. This 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 up to 8 years. The length of time the participant will be in the study depends on when they agreed to take part and when the medicine is available for purchase in their country (or 31 December 2027 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. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.
- Being tested
- Concizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ConcizumabDrug
When patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 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 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 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 replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: No prophylaxis (PPX) | Experimental | Haemophilia A (HA) and haemophilia B (HB) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension phase, this group will receive treatment with concizumab. Receives: Drug: Concizumab |
| Arm 2: Concizumab prophylaxis | Experimental | HA and HB patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. Receives: Drug: Concizumab |
| Arm 3: Concizumab prophylaxis | Experimental | The HA patients enrolled into the concizumab phase 2 trial NN7415-4255 (explorer 5) will be offered enrolment into this arm. Receives: Drug: Concizumab |
| Arm 4: Concizumab prophylaxis | Experimental | Arm 4 will include patients previously on prophylaxis with factor products with a minimum of 24 weeks observation in NN7415-4322 (explorer 6) (at least 30 HA and 30 HB patients).
In addition, arm 4 will also include: 1) Patients who were randomised to arms 1 and 2 before the treatment pause. 2) HA patients who were in NN7415-4255 (explorer 5) at the time of the treatment pause, and who have now completed explorer 5. 3) On demand patients included after arms 1 and 2 are closed. Receives: Drug: Concizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Rate of treated spontaneous and traumatic bleeding episodes for haemophilia A participants without inhibitors is presented. The observation period used for reporting this endpoint is on-treatment without ancillary therapy excluding data before restart (OTwoATexBR). It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of annualised bleeding rate (ABR). Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen. Confirmatory analyses cut-off was defined as when all participants on no PPX (arm 1) had completed the 24-week visit or withdrawn and all participants on concizumab PPX (in arms 2 and 4) had completed the 32-week visit or withdrawn.
Measured: On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Rate of treated spontaneous and traumatic bleeding episodes for haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen. Confirmatory analyses cut-off was defined as when all participants on no PPX (arm 1) had completed the 24-week visit or withdrawn and all participants on concizumab PPX (in arms 2 and 4) had completed the 32-week visit or withdrawn.
Measured: On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off
Secondary outcomes (21)
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Rate of treated spontaneous and traumatic bleeding episodes for haemophilia A participants without inhibitors in arm 4 participants who had been on stable PPX at least 24 weeks in study 4322 is presented. The observation period used for reporting this endpoint is on stable treatment without ancillary therapy excluding data before restart (OT stable woATexBR). It is defined as the time period where participants are on stable PPX in study NN7415-4322 combined with the time period after the treatment pause in NN7415-4307 where the same participants are on the maintenance dose and have not used factor-containing products not related to treatment of a bleed. The data is reported in the terms of ABR.
Measured: For previous PPX (study 4322): After an initial period on PPX treatment of at least 24 weeks until end of study (maximum 115 weeks) For concizumab PPX (trial 4307): After an initial 5-8 weeks dose adjustment period and up until 56 week cut off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Rate of treated spontaneous and traumatic bleeding episodes for haemophilia B participants without inhibitors in arm 4 participants who had been on stable PPX at least 24 weeks in study 4322 is presented. The observation period used for reporting this endpoint is OT stable woATexBR. It is defined as the time period where participants are on stable PPX in study NN7415-4322 combined with the time period after the treatment pause in NN7415-4307 where the same participants are on the maintenance dose and have not used factor-containing products not related to treatment of a bleed. The data is reported in the terms of ABR.
Measured: For previous PPX (study 4322): After an initial period on PPX treatment of at least 24 weeks until end of study (maximum 115 weeks) For concizumab PPX (trial 4307): After an initial 5-8 weeks dose adjustment period and up until 56 week cut off
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous Bleeding Episodes
Rate of treated spontaneous bleeding episodes for haemophilia A participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous Bleeding Episodes
Rate of treated spontaneous bleeding episodes for haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Joint Bleeding Episodes
Rate of treated spontaneous and traumatic joint bleeding episodes for haemophilia A participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Joint Bleeding Episodes
Rate of treated spontaneous and traumatic joint bleeding episodes for haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Target Joint Bleeding Episodes
Rate of treated spontaneous and traumatic target joint bleeding episodes for haemophilia A participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Target Joint Bleeding Episodes
Rate of treated spontaneous and traumatic target joint bleeding episodes for haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Thromboembolic Events
Number of thromboembolic events in haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is on-treatment (OT). It is defined as the time period where participants were considered to be affected by no PPX (on-demand treatment) or concizumab PPX. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): from week 0 until start of concizumab treatment (week 24). Concizumab (arms 2-4): From week 0 up until the 56 week cut-off. Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Thromboembolic Events
Measured: From week 0 to end of trial (up to 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Hypersensitivity Type Reactions
Number of hypersensitivity type reactions in haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OT. It is defined as the time period where participants were considered to be affected by no PPX (on-demand treatment) or concizumab PPX. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): from week 0 until start of concizumab treatment (week 24). Concizumab (arms 2-4): From week 0 up until the 56 week cut-off. Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Hypersensitivity Type Reactions
Measured: From week 0 to end of trial (up until 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Injection Site Reactions
Number of injection site reactions in haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OT. It is defined as the time period where participants were considered to be affected by no PPX (on-demand treatment) or concizumab PPX. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1 main part): from randomisation (week 0) until start of concizumab treatment (week 24). Concizumab (arms 2-4): From week 0 up until the 56 week cut-off. Concizumab (arm 1 extension part): From week 25 up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Injection Site Reactions
Measured: From week 0 to end of trial (up until 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Participants With Antibodies to Concizumab
Measured: Concizumab (arms 2-4): From start of concizumab treatment (week 0) up until the 56 week cut-off. Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Participants With Antibodies to Concizumab
Measured: From week 0 to end of trial (up until 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Pre-dose (Trough) Concizumab Plasma Concentration (Ctrough)
Ctrough for haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is on-treatment without data before re-start (OTexBR). It is defined as the time period after restart where participants were considered to be affected by no PPX (on-demand treatment) or treatment with the new concizumab regimen.
Measured: Pre-dose (prior to the concizumab administration at week 24)
Haemophila A and Haemophilia B Participants Without Inhibitors: Pre-dose Thrombin Peak
Pre-dose thrombin peak for haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTexBR. It is defined as the time period after restart where participants were considered to be affected by no PPX (on-demand treatment) or treatment with the new concizumab regimen.
Measured: Pre-dose (prior to the concizumab administration at week 24)
Haemophila A and Haemophilia B Participants Without Inhibitors: Pre-dose Free Tissue Factor Pathway Inhibitor (TFPI) Concentration
Pre-dose free TFPI for haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTexBR. It is defined as the time period after restart where participants were considered to be affected by no PPX (on-demand treatment) or treatment with the new concizumab regimen.
Measured: Pre-dose (prior to the concizumab administration at week 24)
Haemophila A and Haemophilia B Participants Without Inhibitors: Maximum Concizumab Plasma Concentration (Cmax)
Cmax for haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTexBR. It is defined as the time period after restart where participants were considered to be affected by no PPX (on-demand treatment) or treatment with the new concizumab regimen.
Measured: Pre-dose, Week 24: 3 hours (h), 6h, 9h, 24h
Haemophila A and Haemophilia B Participants Without Inhibitors: Area Under the Concizumab Plasma Concentration-time Curve (AUC)
AUC for haemophilia A and haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTexBR. It is defined as the time period after restart where participants were considered to be affected by no PPX (on-demand treatment) or treatment with the new concizumab regimen.
Measured: Pre-dose, Week 24: 3 hours (h), 6h, 9h, 24h
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2019
- Study start13 Nov 2019
- Primary completion12 Jul 2022
- Study completion21 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Haemophilia A 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients will be randomised to concizumab PPX/no PPX/ assigned into non-randomised arms, based on treatment before trial. Upon restart, patients randomised to arms 1/2 before pause will enter arm 4. Patients allocated to arms 3 \& 4 before pause will re-enter initially allocated arm. Randomisation into arms 1/2 will be restarted with new patients. Main part is completed when patient completed 24 wks (excluding screening) in arm 1 or 32 wks (excluding screening) in arms 2-4. After main part, patients will have offer to continue in extension (ext.) part and receive treatment with product until concizumab is commercially available in their countries or until 31-Dec-2027 at latest. Patients will be in the extension part for up to 345 weeks (arms 2-4) or up to 353 weeks (arm 1). Patient will receive last dose at home on day prior to visit 26a. Follow-up part will start on visit 26a and lasts for 7 wks and include reporting of bleeding episodes until visit 27a.
- Masking
- None (open label)
- Enrolment
- 156 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor and Disclosure (1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04082429
- Sponsor's protocol ID
- NN7415-4307
- Other IDs
- U1111-1225-9722, 2018-004891-36, 2018
- Acronym
- explorer8
- Status last verified
- Sep 2026
- First submitted
- 5 Sep 2019
- Last updated
- 16 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- 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
Publications
Young G, Angchaisuksiri P, Apte SJ, Brown Frandsen R, Chan AK, Chowdary P, Eichler H, Lyu CJ, Martinez Garcia MF, Matsushita T, Trakymiene SS, Tran HA, Trinchero A, Windyga J, Astermark J. Concizumab in patients with hemophilia A or B without inhibitors: 56-week cut-off results of the phase 3 explorer8 study. Blood Adv. 2026 Jun 24:bloodadvances.2026019931. doi: 10.1182/bloodadvances.2026019931. Online ahead of print.
PubMed 42341325Angchaisuksiri P, von Mackensen S, Apte S, Benson G, Eichler H, Findley A, Matsushita T, Mazini Tavares CM, Puggaard Ravn M, Sathar J, Villarreal Martinez L, Young G. Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study. Res Pract Thromb Haemost. 2025 Feb 20;9(2):102705. doi: 10.1016/j.rpth.2025.102705. eCollection 2025 Feb.
PubMed 40166710Chowdary P, Angchaisuksiri P, Apte S, Astermark J, Benson G, Chan AKC, Jimenez Yuste V, Matsushita T, Hogh Nielsen AR, Sathar J, Sutton C, Saulyte Trakymiene S, Tran H, Villarreal Martinez L, Wheeler AP, Windyga J, Young G, Thaung Zaw JJ, Eichler H. Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial. Lancet Haematol. 2024 Dec;11(12):e891-e904. doi: 10.1016/S2352-3026(24)00307-7. Epub 2024 Nov 6.
PubMed 39521008
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B Without Inhibitors
- ClinicalTrials.gov ID
- NCT04082429
- Acronym / sponsor ID
- explorer8 / NN7415-4307
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 156 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 13 Nov 2019 (actual)
- Primary completion
- 12 Jul 2022 (actual)
- Study completion
- 21 Feb 2028 (estimated)
- First posted
- 9 Sep 2019 (actual)
- Results
- Posted 31 Oct 2025
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 in the body of people with haemophilia A or B without 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 have to inject themselves with the study medicine 1 time every day under the skin. This 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 up to 8 years. The length of time the participant will be in the study depends on when they agreed to take part and when the medicine is available for purchase in their country (or 31 December 2027 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. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Patients will be randomised to concizumab PPX/no PPX/ assigned into non-randomised arms, based on treatment before trial. Upon restart, patients randomised to arms 1/2 before pause will enter arm 4. Patients allocated to arms 3 \& 4 before pause will re-enter initially allocated arm. Randomisation into arms 1/2 will be restarted with new patients. Main part is completed when patient completed 24 wks (excluding screening) in arm 1 or 32 wks (excluding screening) in arms 2-4. After main part, patients will have offer to continue in extension (ext.) part and receive treatment with product until concizumab is commercially available in their countries or until 31-Dec-2027 at latest. Patients will be in the extension part for up to 345 weeks (arms 2-4) or up to 353 weeks (arm 1). Patient will receive last dose at home on day prior to visit 26a. Follow-up part will start on visit 26a and lasts for 7 wks and include reporting of bleeding episodes until visit 27a.
| Arm | Type | Receives |
|---|---|---|
| Arm 1: No prophylaxis (PPX) | Experimental | Drug: ConcizumabHaemophilia A (HA) and haemophilia B (HB) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension phase, this group will receive treatment with concizumab. |
| Arm 2: Concizumab prophylaxis | Experimental | Drug: ConcizumabHA and HB patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. |
| Arm 3: Concizumab prophylaxis | Experimental | Drug: ConcizumabThe HA patients enrolled into the concizumab phase 2 trial NN7415-4255 (explorer 5) will be offered enrolment into this arm. |
| Arm 4: Concizumab prophylaxis | Experimental | Drug: ConcizumabArm 4 will include patients previously on prophylaxis with factor products with a minimum of 24 weeks observation in NN7415-4322 (explorer 6) (at least 30 HA and 30 HB patients). In addition, arm 4 will also include: 1) Patients who were randomised to arms 1 and 2 before the treatment pause. 2) HA patients who were in NN7415-4255 (explorer 5) at the time of the treatment pause, and who have now completed explorer 5. 3) On demand patients included after arms 1 and 2 are closed. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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 severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%).
Key exclusion criteria (4 listed)
- Known or suspected hypersensitivity to any constituent of the trial product or related products.
- Known inherited or acquired coagulation disorder other than congenital haemophilia.
- Presence of confirmed inhibitors 0.6 BU or greater at screening.
- 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.)
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 |
|---|---|---|
| ConcizumabWhen patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 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 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 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 replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections. | Drug | Arm 1: No prophylaxis (PPX), Arm 2: Concizumab prophylaxis, Arm 3: Concizumab prophylaxis, Arm 4: Concizumab prophylaxis |
Primary outcome
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Time frame: On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off
Rate of treated spontaneous and traumatic bleeding episodes for haemophilia A participants without inhibitors is presented. The observation period used for reporting this endpoint is on-treatment without ancillary therapy excluding data before restart (OTwoATexBR). It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of annualised bleeding rate (ABR). Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen. Confirmatory analyses cut-off was defined as when all participants on no PPX (arm 1) had completed the 24-week visit or withdrawn and all participants on concizumab PPX (in arms 2 and 4) had completed the 32-week visit or withdrawn.
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Time frame: On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off
Rate of treated spontaneous and traumatic bleeding episodes for haemophilia B participants without inhibitors is presented. The observation period used for reporting this endpoint is OTwoATexBR. It is defined as the time period after the restart where participants are treated by concizumab treatment or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleeding episode. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen. Confirmatory analyses cut-off was defined as when all participants on no PPX (arm 1) had completed the 24-week visit or withdrawn and all participants on concizumab PPX (in arms 2 and 4) had completed the 32-week visit or withdrawn.
21 secondary outcomes
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Time frame: For previous PPX (study 4322): After an initial period on PPX treatment of at least 24 weeks until end of study (maximum 115 weeks) For concizumab PPX (trial 4307): After an initial 5-8 weeks dose adjustment period and up until 56 week cut off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Time frame: For previous PPX (study 4322): After an initial period on PPX treatment of at least 24 weeks until end of study (maximum 115 weeks) For concizumab PPX (trial 4307): After an initial 5-8 weeks dose adjustment period and up until 56 week cut off
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous Bleeding Episodes
Time frame: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous Bleeding Episodes
Time frame: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Joint Bleeding Episodes
Time frame: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Joint Bleeding Episodes
Time frame: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Target Joint Bleeding Episodes
Time frame: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophilia B Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Target Joint Bleeding Episodes
Time frame: On demand (arm 1): From randomisation after the pause (week 0) up until start of concizumab treatment (week 24) Concizumab (arm 2): From start of the new concizumab dosing regimen (week 0) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Thromboembolic Events
Time frame: On demand (arm 1): from week 0 until start of concizumab treatment (week 24). Concizumab (arms 2-4): From week 0 up until the 56 week cut-off. Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Thromboembolic Events
Time frame: From week 0 to end of trial (up to 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Hypersensitivity Type Reactions
Time frame: On demand (arm 1): from week 0 until start of concizumab treatment (week 24). Concizumab (arms 2-4): From week 0 up until the 56 week cut-off. Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Hypersensitivity Type Reactions
Time frame: From week 0 to end of trial (up until 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Injection Site Reactions
Time frame: On demand (arm 1 main part): from randomisation (week 0) until start of concizumab treatment (week 24). Concizumab (arms 2-4): From week 0 up until the 56 week cut-off. Concizumab (arm 1 extension part): From week 25 up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Injection Site Reactions
Time frame: From week 0 to end of trial (up until 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Participants With Antibodies to Concizumab
Time frame: Concizumab (arms 2-4): From start of concizumab treatment (week 0) up until the 56 week cut-off. Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the 56 week cut-off
Haemophila A and Haemophilia B Participants Without Inhibitors: Number of Participants With Antibodies to Concizumab
Time frame: From week 0 to end of trial (up until 384 weeks)
Haemophila A and Haemophilia B Participants Without Inhibitors: Pre-dose (Trough) Concizumab Plasma Concentration (Ctrough)
Time frame: Pre-dose (prior to the concizumab administration at week 24)
Haemophila A and Haemophilia B Participants Without Inhibitors: Pre-dose Thrombin Peak
Time frame: Pre-dose (prior to the concizumab administration at week 24)
Haemophila A and Haemophilia B Participants Without Inhibitors: Pre-dose Free Tissue Factor Pathway Inhibitor (TFPI) Concentration
Time frame: Pre-dose (prior to the concizumab administration at week 24)
Haemophila A and Haemophilia B Participants Without Inhibitors: Maximum Concizumab Plasma Concentration (Cmax)
Time frame: Pre-dose, Week 24: 3 hours (h), 6h, 9h, 24h
Haemophila A and Haemophilia B Participants Without Inhibitors: Area Under the Concizumab Plasma Concentration-time Curve (AUC)
Time frame: Pre-dose, Week 24: 3 hours (h), 6h, 9h, 24h
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42341325Young G, Angchaisuksiri P, Apte SJ, Brown Frandsen R, Chan AK, Chowdary P, Eichler H, Lyu CJ, Martinez Garcia MF, Matsushita T, Trakymiene SS, Tran HA, Trinchero A, Windyga J, Astermark J. Concizumab in patients with hemophilia A or B without inhibitors: 56-week cut-off results of the phase 3 explorer8 study. Blood Adv. 2026 Jun 24:bloodadvances.2026019931. doi: 10.1182/bloodadvances.2026019931. Online ahead of print.
- Derived from this study: PubMed 40166710Angchaisuksiri P, von Mackensen S, Apte S, Benson G, Eichler H, Findley A, Matsushita T, Mazini Tavares CM, Puggaard Ravn M, Sathar J, Villarreal Martinez L, Young G. Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study. Res Pract Thromb Haemost. 2025 Feb 20;9(2):102705. doi: 10.1016/j.rpth.2025.102705. eCollection 2025 Feb.
- Derived from this study: PubMed 39521008Chowdary P, Angchaisuksiri P, Apte S, Astermark J, Benson G, Chan AKC, Jimenez Yuste V, Matsushita T, Hogh Nielsen AR, Sathar J, Sutton C, Saulyte Trakymiene S, Tran H, Villarreal Martinez L, Wheeler AP, Windyga J, Young G, Thaung Zaw JJ, Eichler H. Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial. Lancet Haematol. 2024 Dec;11(12):e891-e904. doi: 10.1016/S2352-3026(24)00307-7. Epub 2024 Nov 6.
- 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 Reporting Anchor and Disclosure (1452) study director
Novo Nordisk A/S
113 sites in 33 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 Without Inhibitors and Haemophilia B Without Inhibitors, run by Novo Nordisk A/S at 113 sites in 33 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 3 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 16 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 113 sites in Japan, United States, Turkey (Türkiye), France, Poland and 28 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?
Yes. Results are posted in the registry (first posted 31 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Haemophilia A Participants Without Inhibitors: Rate of Treated Spontaneous and Traumatic Bleeding Episodes (time frame: On demand (arm 1): From week 0 up until start of concizumab treatment (week 24); Concizumab (arm 2): From week 0 up until the confirmatory analyses cut-off). 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?
On the ClinicalTrials.gov record for NCT04082429, which also lists 3 linked publications.
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 16 Sep 2026.