NN7088-4410 N8-GP in PTP with Severe Haemophilia A
Research type
Research Study
Full title
Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated Patients with Severe Haemophilia A
IRAS ID
242431
Contact name
Sarah Mangles
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2017-003788-36
Duration of Study in the UK
2 years, 7 months, 3 days
Research summary
The aim of this trial is to investigate the safety and efficacy of the continuous use of turoctocog alfa pegol (N8-GP) in the prevention and treatment of bleeding episodes in patients with severe haemophilia A. All patients enrolled will have previously been treated with this investigational product.
This global open label trial aims to enrol 170 patients (adults and children) across 25 countries. There are 3 treatment arms (once, twice or three times a week) and it will be up to the Investigator to decide which treatment arm the patient should be allocated ensuring to take into account the patient’s previous treatment regimen and bleeding tendencies. Only patients however currently on once-weekly dosing or on-demand treatment upon entry into this trial will be allowed to enter the once-weekly treatment arm. In addition children (< 12 years of age) will only be treated either twice or three times weekly with N8-GP prophylaxis.Enrolment into this trial is only open to those who are currently taking part in pathfinder2 and pathfinder5. Patients will be evaluated over a two year treatment period which will involve 8 site visits as well as an end of trial (EOT) visit. Further, patients undergoing both major and minor surgeries during the trial will use N8-GP during these procedures.
If a patient discontinues then they will be asked to attend the EOT visit and a follow up visit approximately 1 month later. If a patient develops inhibitors to the trial product then they will be asked to attend the EOT visit and additional follow up visits for monitoring by the Investigator.
This trial will be conducted at the same clinics as are currently being used for pathfinder2 and pathfinder5 with N8-GP being supplied as a freeze-dried powder which is re-constituted with sodium chloride solution for I.V. injection.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
18/EE/0067
Date of REC Opinion
5 Apr 2018
REC opinion
Further Information Favourable Opinion