Primary objective:To investigate the safety of turoctocog alfa pegol during continuous use for prevention and treatment of bleeding episodes of previously turoctocog alfa pegol treated severe haemophilia A patients.Secondary objectives:To…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary endpoint:
Number of adverse events reported
Secondary outcome
Key secondary endpoints:
* Incidence of FVIII inhibitors *0.6 BU
* Number of bleeding episodes on prophylaxis
* Number of spontaneous bleeding episodes on prophylaxis
* Haemostatic effect of turoctocog alfa pegol when used for treatment of
bleeding episodes assessed as: Excellent, Good, Moderate, or None
* Number of turoctocog alfa pegol injections required per bleeding episode
Background summary
The rationale for performing this trial is to allow the continued evaluation of
the safety and efficacy of turoctocog alfa pegol in order to obtain additional
data on long-term use. Introducing a twice or three times weekly prophylactic
dosing regimen to the majority of patients is intended to show potential
improvement in clinical outcomes by converting patients to a milder bleeding
phenotype. Joint health and target joints will be assessed and evaluated at
inclusion and at end of trial.
Study objective
Primary objective:
To investigate the safety of turoctocog alfa pegol during continuous use for
prevention and treatment of bleeding episodes of previously turoctocog alfa
pegol treated severe haemophilia A patients.
Secondary objectives:
To investigate the following in severe haemophilia A patients previously
treated with turoctocog alfa pegol:
* Development of FVIII inhibitors
* Efficacy of turoctocog alfa pegol prophylaxis
* Haemostatic efficacy of turoctocog alfa pegol when used for treatment of
bleeds
Study design
This phase 3 trial is a multi-centre, multi-national, open-label,
non-randomised trial evaluating safety and efficacy of turoctocog alfa pegol
during prophylaxis treatment and treatment of bleeds. There will be three
turoctocog alfa pegol treatment arms (dosing once weekly, twice weekly, and
three times weekly) and no comparator.
Intervention
Intravenous injection of turoctocog alfa pegol once, twice or three times
weekly in order to prevent bleeding episodes. Additionally, breakthrough bleeds
will also be treated with an intravenous injection of turoctocog alfa pegol.
Study burden and risks
The trial will provide assessments of longer-term safety of N8-GP and establish
information on joint health and target joints.
Currently available clinical data from the pathfinder programme support that
N8*GP has the intended haemostatic potential. The key risks associated with
N8-GP administration are inhibitor development and allergic/hypersensitivity
reactions, which are well-known class effects for FVIII products.
Flemingweg 18
Alphen a/d Rijn 2408 AV
NL
Flemingweg 18
Alphen a/d Rijn 2408 AV
NL
Listed location countries
Age
Inclusion criteria
1. Male patients of all ages with the diagnosis of severe congenital haemophilia A (FVIII activity <1%) based on medical records
2. On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer
Exclusion criteria
1. Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
2. Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol
3. Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or nonapproved investigational medicinal product
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2017-003788-36-NL |
| CCMO | NL64362.078.18 |