A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A without Inhibitors

• Register: EUCTR
• Last refreshed on: 14 April 2020
• Main ID: EUCTR2016-000614-29-SE
• Date of registration: 01/06/2017
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A without Inhibitors
• Scientific title: A Multi-Centre Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A without Inhibitors - explorer™5
• Date of first enrolment: 12/06/2017
• Target sample size: 33
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000614-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

European Union, Germany, Japan, Spain, Sweden, Thailand, Turkey, 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 patients aged 18 years or older at the time of signing informed consent, diagnosed with severe haemophilia A (FVIII activity below 1%), based on medical records or results at screening
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 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion criteria:
- Known or suspected hypersensitivity to trial product(s) or related products
- Known inherited or acquired bleeding disorder other than haemophilia A
- Presence of inhibitors (neutralising antibodies) to Factor VIII (equal to or above 0.6 Bethesda Units) at screening measured by the Nijmegen method


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

Health Condition(s) or Problem(s) studied

Haemophilia A
MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) 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-

Primary Outcome(s)

Main Objective: To assess the efficacy of concizumab administered s.c. once daily in preventing bleeding episodes in patients with severe haemophilia A without inhibitors

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

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

Secondary Objective: - To assess the longer-term efficacy of concizumab in patients with severe haemophilia A without inhibitors
- To assess the safety of concizumab in patients with severe haemophilia A without inhibitors
- To assess the immunogenicity of concizumab in patients with severe haemophilia A without inhibitors

Secondary Outcome(s)

Secondary end point(s): 1. The number of spontaneous bleeding episodes
2. The 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

Secondary ID(s)

NN7415-4255

Source(s) of Monetary Support

Novo Nordisk A/S

Ethics review

Status: Approved
Approval date: 17/05/2017
Contact: