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Clinical Trials/2023-506832-33-00
2023-506832-33-00
Active, not recruiting
Phase 3

Efficacy and Safety of Concizumab prophylaxis in patients with haemophilia A or B with inhibitors

Novo Nordisk A/S18 sites in 8 countries43 target enrollmentStarted: January 31, 2024Last updated:
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Overview

Phase
Phase 3
Status
Active, not recruiting
Enrollment
43
Locations
18
Primary Endpoint
The number of treated spontaneous and traumatic bleeding episodes. On demand (arm 1) From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
OverviewEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

Brief Summary

To compare the effect of concizumab prophylaxis to no prophylaxis (on-demand treatment with bypassing agents) in reducing the number of bleeding episodes in adult and adolescent patients with haemophilia A or B with inhibitors

Eligibility Criteria

Ages
0 years to 65+ years (0-17 Years, 65+ Years, 18-64 Years)
Sex
Male
Accepts Healthy Volunteers
No

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 at the time of signing informed consent.
  • •Body weight >25 kg at screening
  • •Congenital Haemophilia A or B of any severity with documented history of inhibitor (≥0.6 BU).
  • •Patient has been prescribed, or in need of, treatment with bypassing agents in the last 24 weeks prior to screening (for patients not previously enrolled in NN7415-4310).

Exclusion Criteria

  • •Known or suspected hypersensitivity to any constituent of the trial product or related products
  • •A known systemic inflammatory condition requiring systemic treatment at screening
  • •Treatment with emicizumab within 180 days before screening.
  • •Ongoing or planned Immune Tolerance Induction treatment
  • •Any disorder, except for conditions associated with haemophilia, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol
  • •Previous participation in this trial. Participation is defined as signed informed consent. However, this is not applicable for patients who were screen failed at Sponsor’s decision due to the treatment pause.
  • •Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer (not applicable for patients from NN7415-4310).
  • •Platelets ≤ 100x109/L at screening
  • •Fibrinogen below laboratory lower normal limit at screening
  • •Hepatic dysfunction defined as AST and/or ALT > 3 times the upper limit combined with total bilirubin > 1,5 times the upper limit at screening

Outcomes

Primary Outcomes

The number of treated spontaneous and traumatic bleeding episodes. On demand (arm 1) From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)

The number of treated spontaneous and traumatic bleeding episodes. On demand (arm 1) From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)

Secondary Outcomes

  • Change in SF36v2 bodily pain from start of treatment (week 0) until week 24
  • Change in SF36v2 physical functioning From start of treatment (week 0) until week 24
  • Number of treated spontaneous bleeding episodes On demand (arm 1) From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
  • Number of treated spontaneous and traumatic joint bleeds On demand (arm 1) From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
  • Number of treated spontaneous and traumatic target joint bleeds On demand (arm 1) From randomisation (week 0) up until start of concizumab treatment (at least 24 weeks) Concizumab (arm 2) From start of the new concizumab dosing regimen (week 0) up until the primary analysis cut-off (at least 32 weeks)
  • Number of thromboembolic 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 as well as After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of the new concizumab dosing regimen (visit 9a) up
  • Number of thromboembolic events Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 339 weeks)
  • Number 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 as well as After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of the new concizumab dosing regimen (visi
  • Number of hypersensitivity type reactions Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 339 weeks)
  • Number 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 0b) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment (week 0a) up until the primary analysis cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of the new concizumab dosing regimen (visit 9a)
  • Number of injection site reactions Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (up to 339 weeks)
  • Number of patients with antibodies to concizumab Concizumab (arms 2-4) Before the pause: From start of concizumab treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment (week 0) up until the primary analysis cut-off (at least 32 weeks) Concizumab (arm 1 extension part) From start of the new concizumab dosing regimen (visit 9a) up until the primary analysis cut-off
  • Number of patients with antibodies to concizumab Concizumab Before the pause: From start of treatment (week 0) up until 7 weeks after the treatment was paused as well as After the pause: From start of concizumab treatment up until the end of trial (339 weeks)
  • Pre-dose (trough) concizumab plasma concentration (Ctrough) Prior to the concizumab administration at week 24 (after restart)
  • Pre-dose thrombin peak Prior to the concizumab administration at week 24 (after restart)
  • Pre-dose free TFPI concentration Prior to the concizumab administration at week 24 (after restart)
  • Maximum concizumab plasma concentration (Cmax) From 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart)
  • Area under the concizumab plasma concentration-time curve (AUC) From 0 to 24 hours where 0 is time of the concizumab dose at week 24 (after restart)

Investigators

Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

EU Submission Hub

Scientific

Novo Nordisk A/S

Study Sites (18)

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