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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1225-9670 | Other Identifier | World Health Organization (WHO) | |
| 2018-004889-34 | Registry Identifier | European Medicines Agency (EudraCT) |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Arm 2: Concizumab prophylaxis | Experimental | HAwI and HBwI patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. |
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| 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. |
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| 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). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Concizumab | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Reporting Anchor and Disclosure (1452) | Novo Nordisk A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Center for Inherited Blood Dis | Orange | California | 92868 | United States | ||
| Children's Healthcare Atlanta |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41499759 | Derived | 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. | |
| 39099801 |
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According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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133 participants were actually randomised for the study. The data presented in the results form is till the 56 week cut off (except the data for primary outcome measure, patient reported outcome measures, maximum concizumab plasma concentration and area under the concizumab plasma concentration-time curve) as the study is still ongoing with the extension phase.
The trial was conducted in 27 countries. There were 70 sites that randomised participants. The details are as follows: Algeria (2), Australia (2), Austria (1), Bulgaria (1), Canada (0), Croatia (1), Czech Republic (1), Denmark (1), France (4), India (4), Italy (4), Japan (6), Republic of Korea (2), Malaysia (3), Mexico (1), Poland (4), Portugal (1), Russian Federation (5), Serbia (1), South Africa (2), Spain (5), Sweden (1), Thailand (3), Turkey (4), Ukraine (2), UK (3), United States (6).
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| ID | Title | Description |
|---|---|---|
| FG000 | Arm 1: Previous on Demand: No Prophylaxis | Participants with hemophilia A with inhibitor (HAwI) and hemophilia B with inhibitor (HAwB) received their on-demand treatment for a minimum of 24 weeks. Subsequently, following the main phase, participants transitioned to the new dosing regimen for concizumab and were scheduled to undergo concizumab prophylaxis in the trial's extension phase. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Main Part |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 18, 2021 | Dec 23, 2024 |
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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.
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|
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | Week 24: Predose, 3 hours (h), 6h, 9h, 24h |
| Atlanta |
| Georgia |
| 30329 |
| United States |
| Indiana Hemophilia-Thromb Ctr | Indianapolis | Indiana | 46260 | United States |
| Washington University School of Medicine_St. Louis | St Louis | Missouri | 63110 | United States |
| St. Jude Affiliate Clinic at Novant Health Hemby Children's | Charlotte | North Carolina | 28204 | United States |
| TriStar Medical Group Children's Specialist | Nashville | Tennessee | 37203 | United States |
| University of Texas San Antonio | San Antonio | Texas | 78229 | United States |
| Haematology and Blood Bank Department | Algiers | 16000 | Algeria |
| CHU Constantine BEN BADIS/ Hematology department | Constantine | 25000 | Algeria |
| The Alfred | Melbourne | Victoria | 3004 | Australia |
| The Royal Children's Hospital | Parkville | Victoria | 3052 | Australia |
| Fiona Stanley Hospital - Haemophilia and Haemostasis Centre | Murdoch | Western Australia | 6150 | Australia |
| Klin. Abt. f. Hämatologie und Hämostaseologie, AKH Wien | Vienna | 1090 | Austria |
| UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology | Sofia | 1527 | Bulgaria |
| Hamltn Hth Sci/McMstr Child Hosp | Hamilton | Ontario | L8N 3Z5 | Canada |
| KBC Zagreb, Rebro, Hemofilija centar | Zagreb | 10 000 | Croatia |
| KBC Zagreb_Hematology | Zagreb | 10 000 | Croatia |
| KBC Zagreb, Zavod za pedijatrijsku hematologiju | Zagreb | 10000 | Croatia |
| Ustav Hematologie a krevni tranfuze | Prague | 12000 | Czechia |
| Fakultni Nemocnice Motol A Homolka | Prague | 150 18 | Czechia |
| Skejby Blodsygdomme, blødercentret | Aarhus N | 8200 | Denmark |
| Hospices Civils de Lyon- Hopital Louis Pradel | Bron | 69500 | France |
| Centre Hospitalier de Clermont-Ferrand-Hopital Estaing | Clermont-Ferrand | 63100 | France |
| Ap-Hp-Hopital de Bicetre | Le Kremlin-Bicêtre | 94270 | France |
| Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou | Rennes | 35000 | France |
| St. John's Medical college and Hospital | Bangalore | Karnataka | 560034 | India |
| Sahyadri Speciality Hospital | Pune | Maharashtra | 411004 | India |
| Sahyadri Super Speciality Hospital | Pune | Maharashtra | 411004 | India |
| All India Institute of Medical Sciences_New Dehli | New Dehli | New Delhi | 110029 | India |
| CMCV | Ranipet | Tamil Nadu | 632 517 | India |
| CMCV | Ranipet | Tamil Nadu | 632517 | India |
| All India Institute of Medical Sciences_New Dehli | New Delhi | 110029 | India |
| Dipartimento di Ematologia Univ. Firenze | Florence | FI | 50134 | Italy |
| Istituto Oncologico Veneto - Oncoematologia IOV | Castelfranco Veneto | 31033 | Italy |
| Oncoematologia IOV | Castelfranco Veneto | 31033 | Italy |
| Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano | Milan | 20124 | Italy |
| Azienda OU "S.Maria della Misericordia" | Udine | 33100 | Italy |
| Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Donna Bambino Borgo Trento - U.O.C. Oncoematologia Pediatrica | Verona | 37126 | Italy |
| Ospedale Donna Bambino U.O.C. Oncoematologia Pediatrica | Verona | 37126 | Italy |
| Nagoya University Hospital_Blood Transfusion | Aichi | 466-8560 | Japan |
| Kagoshima City Hospital_Pediatrics | Kagoshima | 890-8760 | Japan |
| Kagoshima City Hospital | Kagoshima | 890-8760 | Japan |
| St. Marianna University School of Medicine Hospital_Pediatrics | Kanagawa | 216-8511 | Japan |
| Mie University Hospital_Dept.of Blood Trans.Med & Cell | Mie | 514-8507 | Japan |
| Nara Medical University Hospital_Pediatrics | Nara | 634-8522 | Japan |
| Saitama Medical Univ. Hospital_Dep of Int Med, Cent for Hemo | Saitama | 350-0495 | Japan |
| Ogikubo Hospital_Pediatries & Blood | Tokyo | 167-0035 | Japan |
| Hospital Pulau Pinang_Georgetown, Penang | George Town | Pulau Pinang | 10450 | Malaysia |
| Hospital Queen Elizabeth 1 | Kota Kinabalu | Sabah | 88586 | Malaysia |
| Hospital Ampang | Ampang | Selangor | 68000 | Malaysia |
| Hospital Ampang | Ampang, Selangor | 68000 | Malaysia |
| Hospital Universitario Dr. José Eleuterio González | Monterrey | Nuevo León | 64460 | Mexico |
| Rikshospitalet - avdeling for blodsykdommer | Oslo | 0027 | Norway |
| Uniwersytecki Szpital Kliniczny W Poznaniu | Poznan | Greater Poland Voivodeship | 60-569 | Poland |
| Szpital Uniwersytecki, Oddzial Kliniczny Hematologii | Krakow | Lesser Poland Voivodeship | 30-688 | Poland |
| Instytut Hematologii i Transfuzjologii | Warsaw | Masovian Voivodeship | 02-776 | Poland |
| SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku | Lublin | 20-081 | Poland |
| Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku | Lublin | 20-081 | Poland |
| Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu | Wroclaw | 50-367 | Poland |
| ULS São João, E.P.E._H.São João_Imunohemoterapia | Porto | 4200-319 | Portugal |
| Children Regional Clinical Hospital | Krasnodar | 350007 | Russia |
| Morozovskaya municipal children hospital | Moscow | 119049 | Russia |
| National Medical Research institution of haemotology | Moscow | 125167 | Russia |
| Republican Hospital n.a. V. A. Baranov | Petrozavodsk | 185019 | Russia |
| City out-patient clinic 37, City Hemophilia Centre | Saint Petersburg | 191186 | Russia |
| Clinical Centre of Serbia, Institute for Haematology | Belgrade | 11000 | Serbia |
| Institute for Mother and Child Health Care of Serbia | Belgrade | 11070 | Serbia |
| University Clinical Centre Kragujevac | Kragujevac | 34000 | Serbia |
| Clinical Centre of Vojvodina | Novi Sad | 21000 | Serbia |
| Institute for Health Care of Children and Adolescents | Novi Sad | 21000 | Serbia |
| Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz | Bratislava | 851 07 | Slovakia |
| Charlotte Maxeke Johannesburg Academic Hospital | Parktown, Johannesburg | Gauteng | 2193 | South Africa |
| Haematology Clinic | Durban | KwaZulu-Natal | 4013 | South Africa |
| Pietersburg Hospital | Polokwane | Limpopo | 0699 | South Africa |
| Daejeon Eulji Medical Center, Eulji University | Daejeon | 35233 | South Korea |
| Severance Hospital, Yonsei University Health System | Seoul | 03722 | South Korea |
| Hospital Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital Universitario La Paz | Madrid | 28046 | Spain |
| Hospital Regional Universitario de Málaga | Málaga | 29010 | Spain |
| Hospital Universitario Regional de Málaga | Málaga | 29010 | Spain |
| Hospital Central de Asturias | Oviedo | 33011 | Spain |
| Hospital Univ. Central de Asturias | Oviedo | 33011 | Spain |
| Hospital Virgen del RocÃo | Seville | 41013 | Spain |
| Skåne US - Koagulationsmottagning | Malmö | 214 28 | Sweden |
| KS Solna - Koagulationsmottagningen | Solna | 171 64 | Sweden |
| Sunpasitthiprasong Hospital_Pediatrics Department | Ubon Ratchathani | Mueang Distirct, | 34000 | Thailand |
| Ramathibodi Hospital_Department of Haematology | Bangkok | 10400 | Thailand |
| Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology | Chiang Mai | 50200 | Thailand |
| İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü | Capa-ISTANBUL | Capa-ISTANBUL | 34093 | Turkey (Türkiye) |
| Akdeniz Üniversitesi Hastanesi- Hematoloji | Antalya | Konyaaltı/ Antalya | 01010 | Turkey (Türkiye) |
| Acıbadem Adana Hastanesi-Hematoloji | Adana | 01130 | Turkey (Türkiye) |
| Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji | Adana | 01130 | Turkey (Türkiye) |
| National specialized children's hospital 'OHMATDYT' - Haemostasis centre | Kyiv | 01135 | Ukraine |
| Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery | Lviv | 79044 | Ukraine |
| Queen Elizabeth Hospital, Birmingham - Haemophilia | Birmingham | B15 2TH | United Kingdom |
| Great Ormond Street Hospital for Children | London | WC1N 3HR | United Kingdom |
| Manchester Royal Infirmary_Manchester | Manchester | M13 9WL | United Kingdom |
| Queen's Medical Centre - Haemophilia Comprehensive Care Centre | Nottingham | NG7 2UH | United Kingdom |
| Royal Hallamshire Hospital | Sheffield | S10 2JF | United Kingdom |
| Derived |
| Tran 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. |
| 37646676 | Derived | Matsushita 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. |
| FG001 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| FG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| FG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| Full Analysis Set (FAS) |
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| Safety Analysis Set (SAS) |
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| COMPLETED |
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| NOT COMPLETED |
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| Extension Part |
|
|
Baseline characteristic data is reported for on-treatment without data on initial regimen (OTexIR) analysis set which included the time period where participants were considered to be affected by on-demand treatment or treatment with the new concizumab dosing regimen excluding observation time during the initial concizumab PPX regimen.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm 1: Previous on Demand: No Prophylaxis | Participants with hemophilia A with inhibitor (HAwI) and hemophilia B with inhibitor (HAwB) received their on-demand treatment for a minimum of 24 weeks. Subsequently, following the main phase, participants transitioned to the new dosing regimen for concizumab and were scheduled to undergo concizumab prophylaxis in the trial's extension phase. |
| BG001 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| BG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| BG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Full analysis set (FAS) included all participants randomised to concizumab prophylaxis (PPX) or on-demand treatment or allocated to arms 3 or 4. This endpoint is only defined for arms 1 and 2 as per protocol. | Posted | Median | Inter-Quartile Range | Events per year | 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) |
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| Secondary | 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. | FAS included all participants randomised to concizumab prophylaxis (PPX) or on-demand treatment or allocated to arms 3 or 4. This endpoint is only defined for arms 1 and 2 as per protocol. | Posted | Median | Inter-Quartile Range | Events per year | 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 |
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| Secondary | 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. | FAS included all participants randomised to concizumab prophylaxis (PPX) or on-demand treatment or allocated to arms 3 or 4. This endpoint is only defined for arms 1 and 2 as per protocol. | Posted | Median | Inter-Quartile Range | Events per year | 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 |
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| Secondary | 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. | FAS included all participants randomised to concizumab prophylaxis (PPX) or on-demand treatment or allocated to arms 3 or 4. This endpoint is only defined for arms 1 and 2 as per protocol. | Posted | Median | Inter-Quartile Range | Events per year | 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 |
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| Secondary | 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. | FAS included all participants randomised to concizumab prophylaxis (PPX) or on-demand treatment or allocated to arms 3 or 4. Overall number of participants analyzed = participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | Score on a scale | Baseline (week 0), Week 24 |
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| Secondary | 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. | FAS included all participants randomised to concizumab prophylaxis (PPX) or on-demand treatment or allocated to arms 3 or 4. Overall number of participants analyzed = participants with available data for this outcome measure. | Posted | Mean | Standard Deviation | Score on a scale | Baseline (week 0), Week 24 |
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| Secondary | 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. | Safety analysis set (SAS) included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. | Posted | Number | Events | 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 |
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| Secondary | 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. | Not Posted | Dec 2026 | From week 0 to end of trial (week 167) | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. | Posted | Number | Reactions | 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 |
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| Secondary | 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. | Not Posted | Dec 2026 | From week 0 to end of trial (week 167) | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. | Posted | Number | Reactions | 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 |
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| Secondary | 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. | Not Posted | Dec 2026 | From week 0 to end of trial (week 167) | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. The data is presented in a combined manner across all Concizumab PPX arms (arm 2-4) and extension part of concizumab (arm 1) considering that the trial is still ongoing, and that incidence of immunogenicity can still change when the trial is completed, hence it was decided to provide now only total incidence. A more granular picture of immunogenicity per arm, will be provided once trial is finished. | Posted | Count of Participants | Participants | 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 |
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| Secondary | 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. | Not Posted | Dec 2026 | From week 0 to end of trial (week 167) | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. The endpoint is applicable for reported arms (extension part arm 1, arm 2, arm 3 and arm 4) only. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanograms per milliliter (ng/mL) | Pre-dose (prior to concizumab administration at week 56) |
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| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. The endpoint is applicable for reported arms (extension part arm 1, arm 2, arm 3 and arm 4) only. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanomoles per liter (nmol/L) | Pre-dose (prior to concizumab administration at week 56) |
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| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. The endpoint is applicable for reported arms (extension part arm 1, arm 2, arm 3 and arm 4) only. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/mL | Pre-dose (prior to concizumab administration at week 56) |
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| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. This endpoint is applicable for reported arms only. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/mL | Week 24: Predose, 3 hours (h), 6h, 9h, 24h |
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| Secondary | 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. | SAS included all participants exposed to concizumab PPX or randomised to on-demand treatment. Overall number of participants analyzed = participants with available data for this outcome measure. This endpoint is applicable for reported arms only. | Posted | Mean | Standard Deviation | nanogram*hour per milliliter (ng*hr/mL) | Week 24: Predose, 3 hours (h), 6h, 9h, 24h |
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Up to week 56 cut-off
Safety analysis set (SAS) included all participants exposed to concizumab prophylaxis (PPX) or randomised to no PPX. All presented AEs were treatment emergent AEs (TEAEs). TEAEs were defined as AEs which initiated or worsened during the time period where participants were considered to be affected by on demand treatment or concizumab treatment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm 1: Previous on Demand: No Prophylaxis | Participants with hemophilia A with inhibitor (HAwI) and hemophilia B with inhibitor (HAwB) received their on-demand treatment for a minimum of 24 weeks. Subsequently, following the main phase, participants transitioned to the new dosing regimen for concizumab and were scheduled to undergo concizumab prophylaxis in the trial's extension phase. | 1 | 19 | 3 | 19 | 8 | 19 |
| EG001 | Extension Arm 1: Previous on Demand: No Prophylaxis | Participants who completed at least 24 weeks of on demand treatment started with the new concizumab dosing regimen and were planned to receive concizumab prophylaxis (PPX) in the extension part of the trial. | 0 | 13 | 2 | 13 | 8 | 13 |
| EG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. | 4 | 33 | 9 | 33 | 15 | 33 |
| EG003 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. | 1 | 21 | 2 | 21 | 13 | 21 |
| EG004 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. | 1 | 60 | 9 | 60 | 35 | 60 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Alcoholic coma | Nervous system disorders | MedDRA 25 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Catheter site infection | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 25 | Systematic Assessment |
| |
| Encephalitis | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Fibrin D dimer increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Functional gastrointestinal disorder | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 25 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 25 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Hip arthroplasty | Surgical and medical procedures | MedDRA 25 | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 25 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Muscle haemorrhage | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 25 | Systematic Assessment |
| |
| Prothrombin fragment 1.2 increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Renal infarct | Renal and urinary disorders | MedDRA 25 | Systematic Assessment |
| |
| Retroperitoneal haematoma | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acarodermatitis | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Conjunctival hyperaemia | Eye disorders | MedDRA 25 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Fibrin D dimer increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Fibroma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Gilbert's syndrome | Congenital, familial and genetic disorders | MedDRA 25 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 25 | Systematic Assessment |
| |
| Haemophilic arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25 | Systematic Assessment |
| |
| Immunisation reaction | Immune system disorders | MedDRA 25 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 25 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 25 | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA 25 | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Molluscum contagiosum | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 25 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Prothrombin fragment 1.2 increased | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 25 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 25 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Subcutaneous abscess | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 25 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA 25 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 25 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25 | Systematic Assessment |
|
There was a pause in the concizumab clinical development programme during the period from March to August 2020, while thromboembolic events were investigated. As of 19 March 2020, all participants on concizumab had stopped treatment and switched to another available treatment as per investigator's discretion. Based on the investigation, risk mitigation actions (including new concizumab dosing regimen) were implemented and trial protocols updated before resuming.
At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Reporting Office (2834) | Novo Nordisk A/S | (+1) 866-867-7178 | clinicaltrials@novonordisk.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 27, 2022 | Dec 23, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| C574488 | concizumab |
Not provided
Not provided
Not provided
| Death |
|
| Withdrawal by Subject |
|
| Ongoing |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| OG001 |
| Extension Arm 1: Previous on Demand: No Prophylaxis |
Participants who completed at least 24 weeks of on demand treatment started with the new concizumab dosing regimen and were planned to receive concizumab prophylaxis (PPX) in the extension part of the trial. |
| OG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG001 |
| Extension Arm 1: Previous on Demand: No Prophylaxis |
Participants who completed at least 24 weeks of on demand treatment started with the new concizumab dosing regimen and were planned to receive concizumab prophylaxis (PPX) in the extension part of the trial. |
| OG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG001 |
| Extension Arm 1: Previous on Demand: No Prophylaxis |
Participants who completed at least 24 weeks of on demand treatment started with the new concizumab dosing regimen and were planned to receive concizumab prophylaxis (PPX) in the extension part of the trial. |
| OG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG001 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) |
Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG004 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG004 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX) | Participants with HAwI and HBwI received a loading dose of 1.0 milligrams per kilograms (mg/kg) concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG004 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
|
| OG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 3: Concizumab Non-naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose (participants entering from the phase 2 trial [N7415-4310] were not to receive a loading dose) of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
| OG003 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|
| OG002 | Arm 4: Concizumab Naive: Concizumab PPX | Participants with HAwI and HBwI received a loading dose of 1.0 mg/kg concizumab subcutaneously followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week maintenance dose setting period on 0.20 mg/kg concizumab, the participants could be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab or stayed at 0.20mg/kg. |
|
|