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

• Register: EUCTR
• Last refreshed on: 26 March 2024
• Main ID: EUCTR2016-000510-30-SE
• Date of registration: 02/06/2017
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors
• Scientific title: 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
• Date of first enrolment: 08/06/2017
• Target sample size: 26
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000510-30
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: yes Other trial design description: With potential dose escalation If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Global Clinical Registry (GCR,1452)
• Affiliation: Novo Nordisk A/S

• Name: Global Clinical Registry (GCR,1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion 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:
- 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


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

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: 100000004850
MedDRA version: 20.0 Level: LLT Classification code 10053752 Term: Hemophilia B with anti factor IX System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

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 milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Trade Name: NovoSeven 5 mg
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: eptacog alfa (activated)
CAS Number: 102786-61-8
Other descriptive name: EPTACOG ALFA (ACTIVATED)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1.0-

Primary Outcome(s)

Primary end point(s): The number of bleeding episodes

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

Timepoint(s) of evaluation of this end point: During at least 24 weeks from treatment onset

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

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1.+ 2. During at least 24 weeks from treatment onset

Secondary end point(s): 1. The number of spontaneous bleeding episodes
2. Number of treatment emergent adverse events (TEAEs)

Secondary ID(s)

NN7415-4310

Source(s) of Monetary Support

Novo Nordisk A/S

Ethics review

Status: Approved
Approval date: 10/05/2017
Contact:

Results

• Results available: Yes
• Date Posted: 14/02/2021
• Date Completed: 31/01/2020
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-000510-30/results