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NCT Number: NCT05135559

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.

Participants will have to inject the study medicine every day under the skin with a pen-injector.

The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Haemophilia A and B With and Without Inhibitors Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Haematology and Blood Bank Department, Algiers, Algeria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
  • Diagnosis of congenital severe haemophilia A (FVIII below 1%) or moderate/severe congenital haemophilia B (FIX (coagulation factor IX) below or equal to 2%), or congenital haemophilia with inhibitors.
  • For arm 1 only: Male aged below 12 years of age at the time of signing informed consent.
  • For arm 1 only: Patients with inhibitors (haemophilia A with inhibitors or haemophilia B with inhibitors)
  • Patients with HAwI (haemophilia A with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia <1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
  • Patients with HBwI (haemophilia B with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia <1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
  • Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products)
  • For arm 1 only: Patients without inhibitors (haemophilia A or haemophilia B)
  • Patients with historical medical records of at least 52 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products; Surgery related PPX or short-term PPX (e.g., in relation to a severe bleed) is not allowed) during the last year prior to enrolment and with at least 3 documented treated bleeds (For participants less than (<) 2 years of age there is no limitation for number of documented treated bleeds in the medical history) during this period
  • Patients with historical medical records of a total of at least 26 weeks of PPX (prophylaxis) treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia <1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available)
  • For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.

Exclusion criteria

  • Known or suspected hypersensitivity to study intervention or related products.
  • Known inherited or acquired coagulation disorder other than congenital haemophilia.
  • Ongoing or planned Immune Tolerance Induction treatment.
  • History of thromboembolic disease (aIncludes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events).

Treatment and study plan

Concizumab

Drug

Participants in Arm 1 will be assigned to concizumab prophylaxis starting with a loading dose on treatment day 0 followed by daily injections of an individual maintenance dose.

Participants in Arm 2 will be assigned to concizumab prophylaxis with daily injections of an individual maintenance dose.

Primary outcomes

  1. For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  2. For non-inhibitor patients treated on demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

Secondary outcomes

  1. For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  2. For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  3. For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  4. For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  5. For non-inhibitor patients treated on-demand during at least the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  6. For non-inhibitor patients treated on-demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  7. For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated joint bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  8. For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  9. For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  10. For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  11. For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  12. For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  13. For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of episode(s)

  14. Concizumab-naïve pateints - Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of event(s)

  15. Number of thromboembolic events, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of event(s)

  16. Number of hypersensitivity type reactions, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of event(s)

  17. Number of injection site reactions, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of event(s)

  18. Number of patients who develop antibodies to concizumab - yes/no, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of patient(s)

  19. Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

    Count of event(s)

  20. Concizumab plasma concentrations prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: Week 32

    Measured in ng/mL

  21. Peak thrombin generation prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: Week 32

    Measured in nM

  22. Free TFPI concentration prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: Week 32

    Measured in ng/mL

  23. Pre-dose (trough) concizumab plasma concentration (Ctrough), reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: Prior to the concizumab administration at week 20

    Measured in ng/mL

  24. Maximum concizumab plasma concentration (Cmax), reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20

    Measured in ng/mL

  25. Area under the concizumab plasma concentration-time curve (AUC), reported both separately for inhibitor and non-inhibitor patients and combined

    Time frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20

    Measured in ng*hr/mL

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

Acronym: Explorer10

Important dates

Study start
2022
Primary completion
2026
Study completion
2029
First posted
Nov 26, 2021
Registry last updated
Jun 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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