A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors

A Multi-Centre, Randomised, Open-Label, Controlled Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors - explorer™4

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-000510-30-GR

Enrollment

Registered

2017-07-04

Start date

2017-08-01

Completion date

Unknown

Last updated

2018-02-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemophilia A with inhibitors Haemophilia B with inhibitors MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000011928 MedDRA version: 20.0 Level: LLT Classification code 10053752 Term: Hemophilia B with anti factor IX System Organ Class: 100000011929

Interventions

Product Name: Concizumab B 100 mg/mL Pharmaceutical Form: Solution for injection INN or Proposed INN: concizumab CAS Number: *MASKED* Other descriptive name: CONCIZUMAB Concentration unit: mg/ml milli

Eligibility

Sex/Gender

Male

Inclusion 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 the suitability for the trial - Male haemophilia A or B patients with inhibitors aged 18 years or older at the time of signing informed consent - Patients currently in need of treatment with bypassing agents Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 24 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: - Known or suspected hypersensitivity to trial product(s) or related products - Known inherited or acquired bleeding disorder other than haemophilia - Ongoing or planned immune tolerance induction therapy or prophylaxis with FVIII or FIX

Design outcomes

Secondary

Measure	Time frame
Secondary end point(s): 1. The number of spontaneous bleeding episodes 2. Number of treatment emergent adverse events (TEAEs);Timepoint(s) of evaluation of this end point: 1.+ 2. During at least 24 weeks from treatment onset All endpoints referring to the time frame of at least 24 weeks will be evaluated in the main part of the trial, defined to end when the last patient has completed a minimum of 24 weeks of dosing treatment with trial product (or has withdrawn). In addition, number of bleeding episodes during 76 weeks of treatment with prophylactic concizumab will be analysed.	—

Measure

Time frame

Secondary end point(s): 1. The number of spontaneous bleeding episodes 2. Number of treatment emergent adverse events (TEAEs);Timepoint(s) of evaluation of this end point: 1.+ 2. During at least 24 weeks from treatment onset All endpoints referring to the time frame of at least 24 weeks will be evaluated in the main part of the trial, defined to end when the last patient has completed a minimum of 24 weeks of dosing treatment with trial product (or has withdrawn). In addition, number of bleeding episodes during 76 weeks of treatment with prophylactic concizumab will be analysed.

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Primary

Measure	Time frame
Main Objective: To assess the efficacy of concizumab administered s.c. once daily in preventing bleeding episodes in haemophilia A and B patients with inhibitors;Secondary Objective: 1. To assess the longer-term efficacy of concizumab in haemophilia patients with inhibitors 2. To assess the safety of concizumab in haemophilia patients with inhibitors 3. To assess the safety of administering recombinant factor VIIa (rFVIIa) to haemophilia patients with inhibitors that are exposed to concizumab 4. To assess the immunogenicity of concizumab in haemophilia patients with inhibitors;Primary end point(s): The number of bleeding episodes;Timepoint(s) of evaluation of this end point: During at least 24 weeks from treatment onset	—

Measure

Time frame

Main Objective: To assess the efficacy of concizumab administered s.c. once daily in preventing bleeding episodes in haemophilia A and B patients with inhibitors;Secondary Objective: 1. To assess the longer-term efficacy of concizumab in haemophilia patients with inhibitors 2. To assess the safety of concizumab in haemophilia patients with inhibitors 3. To assess the safety of administering recombinant factor VIIa (rFVIIa) to haemophilia patients with inhibitors that are exposed to concizumab 4. To assess the immunogenicity of concizumab in haemophilia patients with inhibitors;Primary end point(s): The number of bleeding episodes;Timepoint(s) of evaluation of this end point: During at least 24 weeks from treatment onset

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Countries

Austria, Canada, Croatia, Denmark, European Union, Greece, Israel, Japan, Malaysia, Spain, Sweden, Ukraine, United States

Contacts

Public ContactGlobal Clinical Registry (GCR,1452)

Novo Nordisk A/S

clinicaltrials@novonordisk.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026