NN7415-4255 Concizumab Explorer 5

Principal Investigator: Hassan Yaish
Keywords: Concizumab , Severe Haemophilia A Department: Pediatric Administration
IRB Number: 00104196 Co Investigator:  
Specialty: Pediatrics, General
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Rebbecca Hanshew
Rebbecca.Perez@hsc.utah.edu
801-213-9105

Brief Summary

Four phase 1 clinical trials with concizumab have been finalised. Key safety and preliminary
efficacy results from these phase 1 trials support further development of concizumab in haemophilia patients. Therefore, the main objective in the phase 2 of concizumab development is to assess
efficacy and safety and provide data that will guide for the confirmatory phase 3 concizumab trials.

The main objective for the phase 2 trial NN7415-4255, explorer™5, is to assess the efficacy of
concizumab administered s.c. once daily to prevent bleeding episodes in patients with severe
haemophilia A without inhibitors. Furthermore, this trial aims to assess the longer-term efficacy and
safety of concizumab in severe haemophilia A patients without inhibitors.

Primary objective

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

Secondary objectives
 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
Primary endpoint
 The number of bleeding episodes during at least 24 weeks from treatment onset..
Key secondary endpoints
 The number of spontaneous bleeding episodes during at least 24 weeks from treatment onset
 The number of treatment emergent adverse events (TEAEs) during at least 24 weeks from
treatment onset.

Inclusion Criteria

Inclusion criteria
For an eligible patient, all inclusion criteria must be answered “yes”.
1. 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.
2. Male patients aged ≥ 18 years at the time of signing informed consent, diagnosed with
severe haemophilia A (FVIII activity <1%), based on medical records or results at
screening.
3. For patients being treated on-demand with FVIII replacement therapy, a minimum of six
documented and treated bleeding episodes during the 24 weeks (or twelve bleeds during 52
weeks) prior to screening.

Exclusion Criteria

Exclusion criteria
For an eligible patient, all exclusion criteria must be answered “no”.
1. Known or suspected hypersensitivity to trial product(s) or related products.
2. Previous participation in this trial. Participation is defined as signed informed consent.
3. Participation in any clinical trial of an approved or non-approved investigational medicinal
product within the last 30 days or 5 half-lives (whichever is longer) from the last drug
administration before screening.
4. Any disorder, which in the investigator’s opinion might jeopardise patient’s safety or
compliance with the protocol.
5. Known inherited or acquired bleeding disorder other than haemophilia A.
6. Major surgery conducted within one month prior to the initiation of trial activities or major
surgery planned to occur during the trial.
7. Previous history of thromboembolic disease. Current clinical signs of thromboembolic
disease, or patients who in the judgement of the investigator are considered at high risk of
thromboembolic events.