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A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors (Explorer10)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05135559
Recruitment Status : Recruiting
First Posted : November 26, 2021
Last Update Posted : April 29, 2022
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:

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 about 2 years.


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

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 24, 2022
Estimated Primary Completion Date : December 1, 2024
Estimated Study Completion Date : April 20, 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: Concizumab-naïve patients
Concizumab-naïve participants below 12 years of age at the time of consent/assent
Drug: Concizumab

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.


Experimental: Patients comming from compassionate use
Patients previously treated with concizumab via compassionate use, either on an individual patient basis or through the concizumab compassionate use programme NN7415-4807
Drug: Concizumab

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 Outcome Measures :
  1. The number of treated spontaneous and traumatic bleeding episodes [ Time Frame: From start of treatment (week 0) up until the analysis cut-off (at least 32 weeks) ]
    Count of episode(s)


Secondary Outcome Measures :
  1. The number of all bleeding episodes (spontaneous and traumatic) [ Time Frame: From start of treatment (week 0) or from the point in time when the concizumab maintenance dose is set until the analysis cut-off (at least 32 weeks) ]
    Count of episode(s)

  2. Number of treated spontaneous bleeding episodes [ Time Frame: From start of treatment (week 0) or from the point in time when the concizumab maintenance dose is set until the analysis cut-off (at least 32 weeks) ]
    Count of episode(s)

  3. Number of treated joint bleeding episodes [ Time Frame: From start of treatment (week 0) or from the point in time when the concizumab maintenance dose is set until the analysis cut-off (at least 32 weeks) ]
    Count of episode(s)

  4. Number of treated bleeding episodes in baseline target joints [ Time Frame: From start of treatment (week 0) or from the point in time when the concizumab maintenance dose is set until the analysis cut-off (at least 32 weeks) ]
    Count of episode(s)

  5. The number of treated spontaneous and traumatic bleeding episodes [ Time Frame: From the point in time when the concizumab maintenance dose is set until the analysis cut-off (at least 32 weeks) ]
    Count of episode(s)

  6. Number of treatment emergent adverse events [ Time Frame: From start of treatment (week 0) up until the analysis cut-off (at least 32 weeks) ]
    Count of event(s)

  7. Number of thromboembolic events [ Time Frame: From start of treatment (week 0) up until the analysis cut-off (at least 32 weeks) ]
    Count of event(s)

  8. Number of hypersensitivity type reactions [ Time Frame: From start of treatment (week 0) up until the analysis cut-off (at least 32 weeks) ]
    Count of event(s)

  9. Number of injection site reactions [ Time Frame: From start of treatment (week 0) up until the analysis cut-off (at least 32 weeks) ]
    Count of event(s)

  10. Number of patients who develop antibodies to concizumab - yes/no [ Time Frame: From start of treatment (week 0) up until the analysis cut-off (at least 32 weeks) ]
    Count of event(s)

  11. Concizumab plasma concentrations prior to dosing [ Time Frame: Week 32 ]
    messured in ng/mL

  12. Peak thrombin generation prior to dosing [ Time Frame: Week 32 ]
    messured in nM

  13. Free TFPI concentration prior to dosing [ Time Frame: Week 32 ]
    messured in ng/mL

  14. Pre-dose (trough) concizumab plasma concentration (Ctrough) (in a subgroup of participants) [ Time Frame: Prior to the concizumab administration at week 20 ]
    messured in ng/mL

  15. Maximum concizumab plasma concentration (Cmax) (in a subgroup of participants) [ Time Frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20 ]
    messured in ng/mL

  16. Area under the concizumab plasma concentration-time curve (AUC) (in a subgroup of participants) [ Time Frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20 ]
    messured in ng*hr/mL



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

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 medical records of a total of at least 26 weeks of treatment within the last 52 weeks prior to enrolment (For patients that have been diagnosed with haemophilia below 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 HAwI (haemophilia A with inhibitors) with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment.
    • Patients with HBwI (haemophilia B with inhibitors) with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment
    • Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment
    • Patients without inhibitors with medical records of a total of at least 26 weeks of PPX (prophylaxis) treatment within the last 52 weeks prior to enrolment
  • 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05135559


Contacts
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Contact: Novo Nordisk (+1) 866-867-7178 clinicaltrials@novonordisk.com

Locations
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Japan
Novo Nordisk Investigational Site Recruiting
Kanagawa, Japan, 216-8511
Russian Federation
Novo Nordisk Investigational Site Not yet recruiting
Moscow, Russian Federation, 119049
Novo Nordisk Investigational Site Not yet recruiting
Saint-Petersburg, Russian Federation, 191186
Spain
Novo Nordisk Investigational Site Not yet recruiting
Barcelona, Spain, 08035
Novo Nordisk Investigational Site Not yet recruiting
Madrid, Spain, 28046
Novo Nordisk Investigational Site Not yet recruiting
Málaga, Spain, 29009
United Kingdom
Novo Nordisk Investigational Site Not yet recruiting
Birmingham, United Kingdom, B4 6NH
Novo Nordisk Investigational Site Recruiting
London, United Kingdom, WC1N 3HR
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Clinical Transparency (dept. 2834) Novo Nordisk A/S
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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT05135559    
Other Study ID Numbers: NN7415-4616
U1111-1247-7330 ( Other Identifier: World Health Organization (WHO) )
2020-000504-11 ( EudraCT Number )
First Posted: November 26, 2021    Key Record Dates
Last Update Posted: April 29, 2022
Last Verified: April 2022
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn