Haemophilia A With InhibitorsPhase 3explorer7NCT04083781
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors
A Phase 3 study testing Concizumab in people with Haemophilia A With Inhibitors and Haemophilia B With Inhibitors, run by Novo Nordisk A/S at 99 sites in 29 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 with 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?
- Any age, men only
- Conditions the study is for
- Haemophilia A With Inhibitors, Haemophilia B With 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
- 134 participants
- Where
- 99 sitesin 29 countries
- Study dates
- 21 Oct 2019to 21 Feb 2027
- Phase
- Phase 3
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
- 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 Haemophilia A or B of any severity with documented history of inhibitor (equal to or above 0.6 Bethesda Units (BU).
- Patient has been prescribed, or in need of, treatment with bypassing agents in the last 24 weeks prior to screening (for patients not previously enrolled in NN7415-4310 (explorer 4)).
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.
- Ongoing or planned Immune Tolerance Induction treatment.
- History of thromboembolic disease (includes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion). Current clinical signs of, or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events.)
Trial locations and contacts
99 sites in 29 countries.
99 sites in 29 countries
Japan8 sites
Nagoya University Hospital_Blood Transfusion
Aichi, 466-8560
Kagoshima City Hospital_Pediatrics
Kagoshima, 890-8760
Kagoshima City Hospital
Kagoshima, 890-8760
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, 216-8511
Mie University Hospital_Dept.of Blood Trans.Med & Cell
Mie, 514-8507
Nara Medical University Hospital_Pediatrics
Nara, 634-8522
Saitama Medical Univ. Hospital_Dep of Int Med, Cent for Hemo
Saitama, 350-0495
Ogikubo Hospital_Pediatries & Blood
Tokyo, 167-0035
United States7 sites
Children's Healthcare Atlanta
Atlanta, Georgia, 30329
St. Jude Affiliate Clinic at Novant Health Hemby Children's
Charlotte, North Carolina, 28204
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260
TriStar Medical Group Children's Specialist
Nashville, Tennessee, 37203
Center for Inherited Blood Dis
Orange, California, 92868
University of Texas San Antonio
San Antonio, Texas, 78229
Washington University School of Medicine_St. Louis
St Louis, Missouri, 63110
India7 sites
St. John's Medical college and Hospital
Bangalore, Karnataka, 560034
All India Institute of Medical Sciences_New Dehli
New Dehli, New Delhi, 110029
All India Institute of Medical Sciences_New Dehli
New Delhi, 110029
Sahyadri Speciality Hospital
Pune, Maharashtra, 411004
Sahyadri Super Speciality Hospital
Pune, Maharashtra, 411004
CMCV
Ranipet, Tamil Nadu, 632 517
CMCV
Ranipet, Tamil Nadu, 632517
Italy7 sites
Istituto Oncologico Veneto - Oncoematologia IOV
Castelfranco Veneto, 31033
Oncoematologia IOV
Castelfranco Veneto, 31033
Dipartimento di Ematologia Univ. Firenze
Florence, FI, 50134
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano
Milan, 20124
Azienda OU "S.Maria della Misericordia"
Udine, 33100
Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Donna Bambino Borgo Trento - U.O.C. Oncoematologia Pediatrica
Verona, 37126
Ospedale Donna Bambino U.O.C. Oncoematologia Pediatrica
Verona, 37126
Spain7 sites
Hospital Vall d'Hebron
Barcelona, 08035
Hospital Universitario La Paz
Madrid, 28046
Hospital Regional Universitario de Málaga
Málaga, 29010
Hospital Universitario Regional de Málaga
Málaga, 29010
Hospital Central de Asturias
Oviedo, 33011
Hospital Univ. Central de Asturias
Oviedo, 33011
Hospital Virgen del Rocío
Seville, 41013
Poland6 sites
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
Krakow, Lesser Poland Voivodeship, 30-688
SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
Lublin, 20-081
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
Lublin, 20-081
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
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
Serbia5 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
Institute for Health Care of Children and Adolescents
Novi Sad, 21000
United Kingdom5 sites
Queen Elizabeth Hospital, Birmingham - Haemophilia
Birmingham, B15 2TH
Great Ormond Street Hospital for Children
London, WC1N 3HR
Manchester Royal Infirmary_Manchester
Manchester, M13 9WL
Queen's Medical Centre - Haemophilia Comprehensive Care Centre
Nottingham, NG7 2UH
Royal Hallamshire Hospital
Sheffield, S10 2JF
France4 sites
Hospices Civils de Lyon- Hopital Louis Pradel
Bron, 69500
Centre Hospitalier de Clermont-Ferrand-Hopital Estaing
Clermont-Ferrand, 63100
Ap-Hp-Hopital de Bicetre
Le Kremlin-Bicêtre, 94270
Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou
Rennes, 35000
Malaysia4 sites
Hospital Ampang
Ampang, Selangor, 68000
Hospital Ampang
Ampang, Selangor, 68000
Hospital Pulau Pinang_Georgetown, Penang
George Town, Pulau Pinang, 10450
Hospital Queen Elizabeth 1
Kota Kinabalu, Sabah, 88586
Turkey (Türkiye)4 sites
Acıbadem Adana Hastanesi-Hematoloji
Adana, 01130
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji
Adana, 01130
Akdeniz Üniversitesi Hastanesi- Hematoloji
Antalya, Konyaaltı/ Antalya, 01010
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-ISTANBUL, Capa-ISTANBUL, 34093
Australia3 sites
The Alfred
Melbourne, Victoria, 3004
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
Murdoch, Western Australia, 6150
The Royal Children's Hospital
Parkville, Victoria, 3052
Croatia3 sites
KBC Zagreb, Rebro, Hemofilija centar
Zagreb, 10 000
KBC Zagreb_Hematology
Zagreb, 10 000
KBC Zagreb, Zavod za pedijatrijsku hematologiju
Zagreb, 10000
South Africa3 sites
Haematology Clinic
Durban, KwaZulu-Natal, 4013
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, 2193
Pietersburg Hospital
Polokwane, Limpopo, 0699
Thailand3 sites
Ramathibodi Hospital_Department of Haematology
Bangkok, 10400
Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology
Chiang Mai, 50200
Sunpasitthiprasong Hospital_Pediatrics Department
Ubon Ratchathani, Mueang Distirct,, 34000
Algeria2 sites
Haematology and Blood Bank Department
Algiers, 16000
CHU Constantine BEN BADIS/ Hematology department
Constantine, 25000
Czechia2 sites
Ustav Hematologie a krevni tranfuze
Prague, 12000
Fakultni Nemocnice Motol A Homolka
Prague, 150 18
South Korea2 sites
Daejeon Eulji Medical Center, Eulji University
Daejeon, 35233
Severance Hospital, Yonsei University Health System
Seoul, 03722
Sweden2 sites
Skåne US - Koagulationsmottagning
Malmö, 214 28
KS Solna - 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
Austria1 site
Klin. Abt. f. Hämatologie und Hämostaseologie, AKH Wien
Vienna, 1090
Bulgaria1 site
UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology
Sofia, 1527
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
Slovakia1 site
Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz
Bratislava, 851 07
About this study
Official title: Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B With Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group, participants will get study medicine from the start of the study. In the other group, participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will get 1 injection with the study medicine every day under the skin. This participants will have to do themselves and can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for about seven years. The length of time the participants will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (31 December 2026 at the latest). The time between visits will be approximately 4 weeks for the first 6 to 12 months, depending on the group participants are in and approximately 8 weeks for the rest of the study. Participants will be asked to record information into an electronic diary during the study and may also be asked to wear an activity tracker.
- Being tested
- Concizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ConcizumabDrug
Concizumab will be administered daily subcutaneously (s.c., under the skin). When patients are randomised to concizumab prophylaxis they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week 0: arm 2, 3 \& 4) or visit 9a (week 24: arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (week 6: arm 2, 3 \& 4) or 9a.3 (week 30: arm 1) and will be based on the concizumab exposure level measured at the previous visit 4a (week 4) or 9a.2 (week 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual bypassing product until visit 9a (week 24: end of main part for arm 1).
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: No prophylaxis | Experimental | Haemophilia A with inhibitors (HAwI) and haemophilia B with inhibitors (HBwI) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension part, patients in arm 1 will receive daily concizumab subcutaneous (s.c., under the skin) injections. Receives: Drug: Concizumab |
| Arm 2: Concizumab prophylaxis | Experimental | HAwI and HBwI 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 HAwI and HBwI patients enrolled into the concizumab phase 2 trial (NN7415-4310) at time of transfer will be offered enrolment into this trial. It is required that these patients are on concizumab prophylaxis up until enrolment into the trial. These patients will continue concizumab prophylaxis. Receives: Drug: Concizumab |
| Arm 4: Concizumab prophylaxis | Experimental | Patients previously on prophylaxis with by-passing agents and on-demand patients who are screened at a timepoint where the required number of patients in arms 1 and 2 have been randomised. These patients will, if eligible, be enrolled into the trial and will initiate concizumab prophylaxis at visit 2a (week 0). Receives: Drug: Concizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Treated Spontaneous and Traumatic Bleeding Episodes
Rate of treated spontaneous and traumatic bleeding episodes is presented. The observation period used for reporting this endpoint is on-treatment without ancillary therapy excl. data on initial regimen for participants exposed to both regimens (OTwoATexIR). It is defined as the time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of annualised bleeding rate (ABR). Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2): From week 0 up until the primary analysis cut-off (at least 32 weeks)
Secondary outcomes (18)
Rate of Treated Spontaneous Bleeding Episodes
Rate of treated spontaneous bleeding episodes is presented. The observation period used for reporting this endpoint is OTwoATexIR. It is defined as the time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of Treated Spontaneous and Traumatic Joint Bleeds
Rate of treated spontaneous and traumatic joint bleeds is presented. Observation period used for reporting this endpoint is OTwoATexIR. It is defined as time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of ABR. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Rate of Treated Spontaneous and Traumatic Target Joint Bleeds
Rate of treated spontaneous and traumatic target joint bleeds is presented. Observation period used for reporting the endpoint is OTwoATexIR. It is defined as time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in terms of ABR. Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.
Measured: On demand (arm 1): From week 0 up until start of concizumab treatment (at least 24 weeks) Extension concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off Concizumab (arm 2): From week 0 up until week 56 cut-off
Change in 36-item Short Form Health Survey (SF-36v2) Bodily Pain
Change in 36-item SF-36v2 bodily pain from baseline (week 0) to week 24 is presented. SF-36 v2 Health Survey is 36-item generic patient-reported outcome (PRO) instrument measuring health-related quality of life and general health status across disease areas. SF-36 v2 scores are norm-based scores, i.e. transformed to a scale where the 2009 US general population has a mean of 50 and an SD of 10. Lowest and highest scores for bodily pain are 21.68 to 62.0. Higher values indicate better functional health and well-being. Observation period for reporting the data is on-treatment without data on initial regimen (OTexIR) which is defined as time period where participants are considered affected by on demand treatment or treatment with new concizumab dosing regimen. Week 0 is defined as time of randomisation to on-demand administration or start of new concizumab dosing regimen.
Measured: Baseline (week 0), Week 24
Change in SF36v2 Physical Functioning
Change in 36-item SF-36v2 physical functioning from baseline (week 0) to week 24 is presented. SF-36 v2 Health Survey is 36-item generic PRO instrument measuring health-related quality of life and general health status across disease areas. SF-36 v2 scores are norm-based scores, i.e. transformed to a scale where the 2009 US general population has a mean of 50 and an SD of 10. Lowest and highest scores for physical functioning are 19.26 to 57.54. Higher values indicate better functional health and well-being. Observation period used for reporting the data is OTexIR which is defined as time period where participants are considered affected by on demand treatment or treatment with new concizumab dosing regimen. Week 0 is defined as time of randomisation to on-demand administration or start of new concizumab dosing regimen.
Measured: Baseline (week 0), Week 24
Number of Thromboembolic Events
Number of thromboembolic events is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Thromboembolic Events
Number of thromboembolic events is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: From week 0 to end of trial (week 167)
Number of Hypersensitivity Type Reactions
Number of hypersensitivity type reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Hypersensitivity Type Reactions
Number of hypersensitivity type reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: From week 0 to end of trial (week 167)
Number of Injection Site Reactions
Number of injection site reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: On demand (arm 1 ): From week 0 until start of concizumab treatment (atleast 24 weeks) Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Injection Site Reactions
Number of injection site reactions is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: From week 0 to end of trial (week 167)
Number of Participants With Antibodies to Concizumab
Number of participants with antibodies to concizumab is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: Concizumab (arms 2-4): From week 0 up until week 56 cut-off Extension Concizumab (arm 1): From start of new concizumab dosing regimen (week 25) up until week 56 cut-off
Number of Participants With Antibodies to Concizumab
Number of participants with antibodies to concizumab is presented. The observation period used for reporting the endpoint is on-treatment period which is defined as the time period where participants are considered to be affected by on-demand treatment or concizumab treatment.
Measured: From week 0 to end of trial (week 167)
Pre-dose (Trough) Concizumab Plasma Concentration (Ctrough)
Pre-dose (trough) concizumab plasma concentration is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Measured: Pre-dose (prior to concizumab administration at week 56)
Pre-dose Thrombin Peak
Pre-dose thrombin peak for concizumab is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Measured: Pre-dose (prior to concizumab administration at week 56)
Pre-dose Free Tissue Factor Pathway Inhibitor (TFPI) Concentration
Pre-dose free TFPI concentration for concizumab is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Measured: Pre-dose (prior to concizumab administration at week 56)
Maximum Concizumab Plasma Concentration (Cmax)
Maximum concizumab plasma concentration is presented. The observation period used for reporting the endpoint is on OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Measured: Week 24: Predose, 3 hours (h), 6h, 9h, 24h
Area Under the Concizumab Plasma Concentration-time Curve (AUC)
Area under the concizumab plasma concentration-time curve is presented. The observation period used for reporting the endpoint is OTexIR. It is defined as the time period where participants are considered to be affected by on demand treatment or treatment with the new concizumab dosing regimen.
Measured: Week 24: Predose, 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 posted10 Sep 2019
- Study start21 Oct 2019
- Primary completion27 Dec 2021
- Study completion21 Feb 2027 (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 With 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
- Participants will be randomised to concizumab prophylaxis (ppx) or no ppx or assigned into non-randomised treatment arms, based on their treatment regimen before entering the trial. Main part of trial is completed when participant has completed at least 24 weeks of participation in arm 1 or 32 weeks in arms 2, 3 and 4. After main part, all participants will be offered to continue in extension part and receive treatment until concizumab is commercially available in their countries or until 31 December 2026 for up to 332 weeks (arms 1-4) or up to 324 weeks (randomised to arm 1 before the pause). After extension part, participant will enter safety follow-up part on visit 26a, which defines end-of-treatment. Participant will receive last dose of trial drug at home on day prior to visit 26a. On visit 26a, participants will either start up commercially available concizumab or revert to previous ppx schedule or on-demand regimen. Follow-up part will start on visit 26a and lasts for 7 weeks.
- Masking
- None (open label)
- Enrolment
- 134 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
- NCT04083781
- Sponsor's protocol ID
- NN7415-4311
- Other IDs
- U1111-1225-9670, 2018-004889-34, 2018
- Acronym
- explorer7
- Status last verified
- Sep 2026
- First submitted
- 6 Sep 2019
- Last updated
- 9 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
Mahlangu J, Boban A, Bruzelius M, Castaman G, Hampton K, Knoebl P, Lebreton A, Linari S, Lopez-Jaime FJ, Martins Mazini Tavares C, Nekkal MS, Nogami K, Rhode Hogh Nielsen A, Shapiro A, d'Oiron R. Concizumab in hemophilia with inhibitors: longer-term efficacy and safety results from the phase 3 explorer7 study. Blood Adv. 2026 Mar 24;10(6):1854-1863. doi: 10.1182/bloodadvances.2025018264.
PubMed 41499759Tran H, von Mackensen S, Abraham A, Castaman G, Hampton K, Knoebl P, Linari S, Odgaard-Jensen J, Neergaard JS, Stasyshyn O, Thaung Zaw JJ, Zulfikar B, Shapiro A. Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study. Res Pract Thromb Haemost. 2024 Jun 17;8(4):102476. doi: 10.1016/j.rpth.2024.102476. eCollection 2024 May.
PubMed 39099801Matsushita T, Shapiro A, Abraham A, Angchaisuksiri P, Castaman G, Cepo K, d'Oiron R, Frei-Jones M, Goh AS, Haaning J, Hald Jacobsen S, Mahlangu J, Mathias M, Nogami K, Skovgaard Rasmussen J, Stasyshyn O, Tran H, Vilchevska K, Villarreal Martinez L, Windyga J, You CW, Zozulya N, Zulfikar B, Jimenez-Yuste V; explorer7 Investigators. Phase 3 Trial of Concizumab in Hemophilia with Inhibitors. N Engl J Med. 2023 Aug 31;389(9):783-794. doi: 10.1056/NEJMoa2216455.
PubMed 37646676