Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8)

• ClinicalTrials.gov Identifier: NCT04082429
• Recruitment Status: Active, not recruiting
• First Posted: September 9, 2019
• Last Update Posted: April 26, 2024
• Sponsor: Novo Nordisk A/S
• Information provided by (Responsible Party): Novo Nordisk A/S

Study Description

Brief Summary:

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with the study medicine 1 time every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for up to 6.5 years. The length of time the participant will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (21 April 2026 at the latest). Participants will have to come to the clinic for up to 40 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months depending on the group participants are in, and approximately 8 weeks for the rest of the study. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.

Condition or disease	Intervention/treatment	Phase
Haemophilia A Without Inhibitors; Haemophilia B Without Inhibitors	Drug: Concizumab	Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 158 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: Patients will be randomised to concizumab PPX/no PPX/ assigned into non-randomised arms, based on treatment before trial. Upon restart, patients randomised to arms 1/2 before pause will enter arm 4. Patients allocated to arms 3 & 4 before pause will re-enter initially allocated arm. Randomisation into arms 1/2 will be restarted with new patients. Main part is completed when patient completed 24 wks (excluding screening) in arm 1 or 32 wks (excluding screening) in arms 2-4. After main part, patients will have offer to continue in extension (ext.) part and receive treatment with product until concizumab is commercially available in their countries or until 21-Apr-26 at latest. Patients will be in ext. part for up to 257 (arms 2-4) or 265 wks (arm 1) for up to additional 128 (arms 2-4) or 136 wks (arm 1). Patient will receive last dose at home on day prior to visit 26a. Follow-up part will start on visit 26a and lasts for 7 wks and include reporting of bleeding episodes until visit 27a.
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B Without Inhibitors
• Actual Study Start Date: November 13, 2019
• Actual Primary Completion Date: July 12, 2022
• Estimated Study Completion Date: June 9, 2026

Arms and Interventions

Arm	Intervention/treatment
Experimental: Arm 1: No prophylaxis (PPX); Haemophilia A (HA) and haemophilia B (HB) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension phase, this group will receive treatment with concizumab.	Drug: Concizumab; When patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 24) (arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections.
Experimental: Arm 2: Concizumab prophylaxis; HA and HB patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis.	Drug: Concizumab; When patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 24) (arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections.
Experimental: Arm 3: Concizumab prophylaxis; The HA patients enrolled into the concizumab phase 2 trial NN7415-4255 (explorer 5) will be offered enrolment into this arm.	Drug: Concizumab; When patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 24) (arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections.
Experimental: Arm 4: Concizumab prophylaxis; Arm 4 will include patients previously on prophylaxis with factor products with a minimum of 24 weeks observation in NN7415-4322 (explorer 6) (at least 30 HA and 30 HB patients). In addition, arm 4 will also include: 1) Patients who were randomised to arms 1 and 2 before the treatment pause. 2) HA patients who were in NN7415-4255 (explorer 5) at the time of the treatment pause, and who have now completed explorer 5. 3) On demand patients included after arms 1 and 2 are closed.	Drug: Concizumab; When patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 24) (arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections.

Outcome Measures

Primary Outcome Measures :

1. For haemophilia A patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
2. For haemophilia B patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.

Secondary Outcome Measures :

1. For haemophilia A patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes [ Time Frame: Time frame is presented under 'outcome measure description' ]
   This will be presented as 'count of episodes'. Arm 4 patients who have been on stable PPX at least 24 weeks in study 4322. For previous PPX (study 4322): From the point in time where PPX is stable and up until the end of study. Stable is defined as the time after an initial period on PPX treatment of at least 24 weeks. For concizumab PPX (trial 4307): From the point in time where the concizumab maintenance dose is confirmed, increased or decreased and up until the confirmatory analyses cut-off (at least 24 weeks).
2. For haemophilia B patients without inhibitors: The number of treated spontaneous and traumatic bleeding episodes [ Time Frame: Time frame is presented under 'outcome measure description' ]
   This will be presented as 'count of episodes'. Arm 4 patients who have been on stable PPX at least 24 weeks in study 4322. For previous PPX (study 4322): From the point in time where PPX is stable and up until the end of study. Stable is defined as the time after an initial period on PPX treatment of at least 24 weeks. For concizumab PPX (trial 4307): From the point in time where the concizumab maintenance dose is confirmed, increased or decreased and up until the confirmatory analyses cut-off (at least 24 weeks).
3. For haemophilia A patients without inhibitors: Number of treated spontaneous bleeding episodes [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
4. For haemophilia B patients without inhibitors: Number of treated spontaneous bleeding episodes [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
5. For haemophilia A patients without inhibitors: Number of treated spontaneous and traumatic joint bleeds [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
6. For haemophilia B patients without inhibitors: Number of treated spontaneous and traumatic joint bleeds [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
7. For haemophilia A patients without inhibitors: Number of treated spontaneous and traumatic target joint bleeds [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
8. For haemophilia B patients without inhibitors: Number of treated spontaneous and traumatic target joint bleeds [ Time Frame: On demand (arm 1): From randomisation after the pause (Wk 0) up until start of concizumab treatment (Wk 24). Concizumab (arm 2): From start of the new concizumab dosing regimen (Wk 0) up until the confirmatory analyses cut-off (at least 32 weeks) ]
   This will be presented as 'count of episodes'.
9. Number of thromboembolic events [ Time Frame: Time frame is presented under 'outcome measure description'. ]
   This will be presented as 'count of events'. On demand (arm 1 main part): from randomisation to on demand treatment up until start of concizumab treatment. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the confirmatory analyses cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the confirmatory analysis cut-off. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day). Week 0 after the pause is defined as the time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
10. Number of thromboembolic events [ Time Frame: Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 296 weeks). ]
    This will be presented as 'count of events'. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day).
11. Number of hypersensitivity type reactions [ Time Frame: Time frame is presented under 'outcome measure description'. ]
    This will be presented as 'count of hypersensitivity type reactions'. On demand (arm 1 main part): from randomisation to on demand treatment up until start of concizumab treatment. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the confirmatory analyses cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the confirmatory analysis cut-off. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day). Week 0 after the pause is defined as the time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
12. Number of hypersensitivity type reactions [ Time Frame: Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 296 weeks). ]
    This will be presented as 'count of hypersensitivity type reactions'. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day).
13. Number of injection site reactions [ Time Frame: Time frame is presented under 'outcome measure description'. ]
    This will be presented as 'count of injection site reactions'. On demand (arm 1 main part): from randomisation to on demand treatment up until start of concizumab treatment. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the confirmatory analyses cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the confirmatory analysis cut-off. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day). Week 0 after the pause is defined as the time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
14. Number of injection site reactions [ Time Frame: Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 296 weeks). ]
    This will be presented as 'count of injection site reactions'. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day).
15. Number of patients with antibodies to concizumab [ Time Frame: Time frame is presented under 'outcome measure description'. ]
    This will be presented as 'count of participants'. Concizumab (arms 2-4): Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment (week 0) up until the confirmatory analyses cut-off (at least 32 weeks). Concizumab (arm 1 extension part): From start of concizumab treatment (week 25) up until the confirmatory analysis cut-off. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day). Week 0 after the pause is defined as the time of randomisation to on-demand administration after the pause or time of start of the new concizumab dosing regimen.
16. Number of patients with antibodies to concizumab [ Time Frame: Concizumab: Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused. After the pause: From start of concizumab treatment up until the end of trial (up to 296 weeks). ]
    This will be presented as 'count of participants'. Week 0 before the pause is defined as the time of the initial randomisation to on-demand administration or time of start of the previous concizumab dosing regimen (0.25 mg/kg/day).
17. Pre-dose (trough) concizumab plasma concentration (Ctrough) [ Time Frame: Prior to the concizumab administration at week 24 (after restart) ]
    This will be measured in 'ng/mL'.
18. Pre-dose thrombin peak [ Time Frame: Prior to the concizumab administration at week 24 (after restart) ]
    This will be measured in 'nmol/L'.
19. Pre-dose free tissue factor pathway inhibitor (TFPI) concentration [ Time Frame: Prior to the concizumab administration at week 24 (after restart) ]
    This will be measured in 'ng/mL'.
20. Maximum concizumab plasma concentration (Cmax) [ Time Frame: From 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart) ]
    This will be measured in 'ng/mL'.
21. Area under the concizumab plasma concentration-time curve (AUC) [ Time Frame: From 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart) ]
    This will be measured in 'ng*hr/mL'.

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
• Male aged 12 years or older at the time of signing informed consent.
• Congenital severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%).

Exclusion Criteria:

• Known or suspected hypersensitivity to any constituent of the trial product or related products.
• Known inherited or acquired coagulation disorder other than congenital haemophilia.
• Presence of confirmed inhibitors 0.6 BU or greater at screening.
• History of thromboembolic disease (includes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion). Current clinical signs of, or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events.)

Contacts and Locations

Locations

United States, California

Children's Hosp-Los Angeles

Los Angeles, California, United States, 90027

Center for Inherited Blood Disorders

Orange, California, United States, 92868

United States, Michigan

Children's Hospital of Michigan

Detroit, Michigan, United States, 48201

United States, Pennsylvania

M.S. Hershey Medical Center

Hershey, Pennsylvania, United States, 17033

United States, Tennessee

Vanderbilt University Medical Center_Nashville_0

Nashville, Tennessee, United States, 37212

United States, Texas

University of Texas San Antonio

San Antonio, Texas, United States, 78229

United States, Wisconsin

Versiti, CCBD

Milwaukee, Wisconsin, United States, 53226

Algeria

Haematology and Blood Bank Department

Algiers, Algeria, 16000

CHU Constantine BEN BADIS/ Hematology department

Constantine, Algeria, 25000

Australia, Victoria

The Alfred

Melbourne, Victoria, Australia, 3004

Royal Children's Hospital

Parkville, Victoria, Australia, 3052

Australia, Western Australia

Fiona Stanley Hospital - Haemophilia and Haemostasis Centre

Murdoch, Western Australia, Australia, 6150

Bosnia and Herzegovina

University Clinical Center of Republic Srpska (205)

Banja Luka, Bosnia and Herzegovina, 78000

Bulgaria

UMHAT "Tsaritsa Yoanna-ISUL"

Sofia, Bulgaria, 1527

Canada, Ontario

Hamltn Hth Sci/McMstr Child Hosp

Hamilton, Ontario, Canada, L8N 3Z5

Croatia

KBC Zagreb, Rebro, Hemofilija centar

Zagreb, Croatia, 10 000

Denmark

Copenhagen Center for Heamatology

København, Denmark, 2100

Estonia

North Estonia Medical Centre Foundation

Tallinn, Estonia, 13419

France

Centre Hospitalier Regional Et Universitaire de Brest-Hopital Morvan

Brest, France, 29200

CHU de Caen - Côte de Nacre

Caen, France, 14033

Hopital de Bicetre

Le Kremlin Bicetre, France, 94270

Hôpital Pontchaillou

Rennes, France, 35033

Germany

UKS - Hämostaseologie und Transfusionsmedizin

Homburg, Germany, 66421

Hungary

MH Eü. Központ -Orszagos Haemophilia Kozpont

Budapest, Hungary, H-1134

India

St. John's Medical college and Hospital

Bangalore, Karnataka, India, 560034

Sahyadri Speciality Hospital

Pune, Maharashtra, India, 411004

Sahyadri Super Speciality Hospital

Pune, Maharashtra, India, 411004

J K Lon Hospital

Jaipur, Rajasthan, India, 302004

Israel

Sheba MC The Israeli National Hemophilia Center

Tel-Hashomer, Israel, 52621

Italy

A.O.U Città Salute Scienza Torino

Torino, Italy, 10126

Japan

Nagoya University Hospital_Blood Transfusion

Aichi, Japan, 466-8560

Hiroshima University Hospital, Hematology

Hiroshima, Japan, 734-8551

Hyogo prefectural kobe children's hospital

Hyogo, Japan, 654-0047

Osaka National Hospital

Osaka, Japan, 546-0006

Saitama Children's Med Centre_Hematology-Oncology

Saitama, Japan, 330-8777

Saitama Medical Univ. Hospital

Saitama, Japan, 350-0495

Shizuoka Children's Hospital

Shizuoka, Japan, 420-8660

National Center for Child Health and Development_Hematology

Tokyo, Japan, 157-8535

National Center for Child Health and Development

Tokyo, Japan, 157-8535

Ogikubo Hospital_Pediatries & Blood

Tokyo, Japan, 167-0035

Korea, Republic of

Daejeon Eulji University Hospital

Daejeon, Korea, Republic of, 35233

Jeju National University Hospital

Jeju-do, Korea, Republic of, 63241

Severance Hospital, Yonsei University Health System

Seoul, Korea, Republic of, 03722

Lithuania

Children Oncohaematology department Children's Hospital,

Vilnius, Lithuania, 08406

Vilnius University hospital Santaros klinikos

Vilnius, Lithuania, LT-08661

Malaysia

Hospital Ampang

Ampang, Selangor, Malaysia, 68000

Mexico

Hospital Universitario Dr. José Eleuterio González

Monterrey, Nuevo León, Mexico, 64460

Poland

Intytut Hematologii i Transfuzjologii

Warszawa, Mazowieckie, Poland, 02-776

Szpital Uniwersytecki, Oddzial Kliniczny Hematologii

Kraków, Małopolskie, Poland, 30-688

Uniwersytecki Szpital Kliniczny W Poznaniu

Poznań, Wielkopolskie, Poland, 60-569

SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku

Lublin, Poland, 20-081

Portugal

Centro Hospitalar de São João_Porto

Porto, Portugal, 4200-319

Russian Federation

Children Regional Clinical Hospital

Krasnodar, Russian Federation, 350007

Morozovskaya municipal children hospital

Moscow, Russian Federation, 119049

National Medical Research institution of haemotology

Moscow, Russian Federation, 125167

Republican Hospital n.a. V. A. Baranov

Petrozavodsk, Russian Federation, 185019

City out-patient clinic 37, City Hemophilia Centre

Saint-Petersburg, Russian Federation, 191186

Serbia

Institute for Mother and Child Health Care of Serbia

Belgrade, Serbia, 11070

University Clinical Centre Kragujevac

Kragujevac, Serbia, 34000

Clinical Centre of Vojvodina, Haematology Clinic

Novi Sad, Serbia, 21000

Slovakia

Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz

Bratislava, Slovakia, 851 07

South Africa

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng, South Africa, 2193

Pietersburg Hospital

Polokwane, Limpopo, South Africa, 0699

Spain

Hospital Universitario La Paz

Madrid, Spain, 28046

Hospital Universitario Regional de Málaga

Málaga, Spain, 29010

Hospital Virgen del Rocío

Sevilla, Spain, 41013

Hospital La Fe - Hemostasia y Trombosis

Valencia, Spain, 46026

Sweden

Koagulationsmottagning

Malmö, Sweden, 205 02

Koagulationsmottagningen

Solna, Sweden, 171 64

Switzerland

USZ Klinik für Medizinische Onkologie und Hämatologie

Zürich, Switzerland, 8091

Thailand

Ramathibodi Hospital_Bangkok_0

Bangkok, Thailand, 10400

Turkey

Gazi University

Ankara, Beşevler/Ankara, Turkey, 06500

Acibadem Adana Hastanesi

Adana, Turkey, 01130

Hacettepe University Medical Faculty

Ankara, Turkey, 06230

Istanbul University Oncology Institute

Capa-ISTANBUL, Turkey, 34093

Trakya University

Edirne, Turkey, 22030

Ege Universitesi Tip Fakultesi

Izmir, Turkey, 35100

Ondokuz Mayis University Medical Faculty Ped. Haematology

Samsun, Turkey, 55139

Ukraine

National specialized children clinic "OHMATDYT"

Kyiv, Ukraine, 01135

Institute of blood pathology and transfusion medicine

Lviv, Ukraine, 79044

United Kingdom

Belfast City Hospital

Belfast, United Kingdom, BT9 7AB

Royal Free Haemophilia Comprehensive Care Center

London, United Kingdom, NW3 2QG

Royal Hallamshire Hospital

Sheffield, United Kingdom, S10 2JF

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

• Study Director: Clinical Reporting Anchor and Disclosure (1452); Novo Nordisk A/S

More Information

• Responsible Party: Novo Nordisk A/S
• ClinicalTrials.gov Identifier: NCT04082429
• Other Study ID Numbers: NN7415-4307; U1111-1225-9722 ( Other Identifier: World Health Organization (WHO) ); 2018-004891-36 ( Registry Identifier: European Medicines Agency (EudraCT) )
• First Posted: September 9, 2019
• Last Update Posted: April 26, 2024
• Last Verified: April 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
• URL: http://novonordisk-trials.com

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemophilia A; Hemophilia B; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked