4322 - Explorer6 - haem patients with/out inhibitors routine practice
Research type
Research Study
Full title
A prospective, multi-national, non-interventional study in haemophilia A and B patients with or without inhibitors treated according to routine clinical treatment practice (explorer™6)
IRAS ID
249959
Contact name
Gary Benson
Contact email
Sponsor organisation
Novo Nordisk Ltd
Clinicaltrials.gov Identifier
U1111-1182-3359, UTN
Duration of Study in the UK
1 years, 8 months, 9 days
Research summary
This non-interventional/basic science study (NIS), explorer™6 (NN7415-4322), is designed to prospectively collect comprehensive data on bleeding episodes and health-related quality of life as well as on physical activity in patients with severe congenital haemophilia A (HA) and B (HB), with or without inhibitors under routine local clinical practice, irrespective of the treatment regimen.
Haemophilia A is characterised by a lack of Factor VIII (eight) and Haemophilia B is characeterised by a lack of Factor IX (nine) in the blood. If these factors are not present in the blood patients the clotting cascade cannot be completed successfully and so people who suffer from these conditions can bleed. The regular treatment for these conditions is to replace the missing factor but sometimes people can develop inhibitors (proteins) to these in the blood which can render these replacement factors useless.
Data collected in explorer™6 is intended to be used for within-patient comparisons with data obtained in subsequent clinical trials with concizumab, a new drug substance developed for subcutaneous (injection under the skin) prophylaxis treatment (treamment given regularly to prevent bleeds occurring) of haemophilia. Therefore, patients participating in explorer™6 will be offered screening for eligibility to participate in the subsequent clinical trials for concizumab, following their participation in explorer™6 and provided the study site participates in the subsequent trial programme. It is expected that the majority of the participating patients will join subsequent concizumab trials and thus may benefit from bleeding prophylaxis with this new investigational treatment option.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
18/EM/0242
Date of REC Opinion
24 Aug 2018
REC opinion
Further Information Favourable Opinion