Primary Objective: * To evaluate the haemostatic effect of N8-GP during surgical procedures in patients with haemophilia A. Secondary Objectives * To evaluate the general safety including immunogenicity of N8-GP when used for prevention and…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary Endpoint:
* Haemostatic effect during surgery evaluated by the four-point scale
(excellent, good, moderate or none), assessed by the Investigator/surgeon at
the day of surgery
Secondary outcome
Secondary Endpoints:
* Average consumption of N8-GP during surgery
* Haemostatic effect of N8-GP during the post-operative period Days 1-6 and 7-14
* Average consumption of N8-GP during the post-operative period Days 1-6
* Incidence rate of inhibitors against factor VIII (FVIII) (*0.6 BU/mL)
Background summary
The rationale for this trial is to investigate efficacy and safety of N8-GP
during surgery, and in the Post-operative Period in patients with haemophilia
A. A surgery trial is in accordance with the CHMP guideline on Clinical
investigation of recombinant and human plasma-derived Factor VIII products
(EMA/CHMP/BPWP/144533/2009). The trial will provide information on the
bleeding-preventive effect during surgery, the haemostatic effect during and
after these procedures and the safety profile of N8-GP in patients with
haemophilia A.
One phase 1 trial has been completed with 26 PTPs dosed with N8-GP and had
undergone a safety and PK investigation. The phase 1 trial has successfully
been concluded with no safety concerns. No FVIII inhibitors were detected and
no treatment related SAEs were reported. The half-life was pro-longed with
approximately 1.6-fold compared to the patients previous FVIII product.
Study objective
Primary Objective:
* To evaluate the haemostatic effect of N8-GP during surgical procedures in
patients with haemophilia A.
Secondary Objectives
* To evaluate the general safety including immunogenicity of N8-GP when used
for prevention and treatment of bleeding throughout the surgical period
* To evaluate the haemostatic effect of N8-GP during the post-operative period
* To evaluate health economic resource use (hospitalisation days) due to
surgery
Study design
The trial is a multi-centre, multi-national, open-label, non-randomised, single
arm, efficacy and safety trial evaluating N8-GP during surgical procedures in
patients with severe (FVIII activity <1%) haemophilia A. In total a minimum of
15 major surgical procedures need to be evaluated in 10-15 patients.
This trial will provide information on the bleeding-preventive efficacy and
safety profile of N8-GP when administered before, during and after surgery.
Patients enrolled in this trial can be recruited from the pivotal trial
(pathfinder2).
This surgery trial consists of a Screening Visit (Visit 1), Day of Surgery (Day
0: Visit 2), Post-operative Period (Days 1-6: Visit 3, Days 7-14: Visit 4) and
an EOT Visit (Visit 5).
Intervention
Continued treatment with N8-GP according to treatment regime in pathfinder2
trial in the period prior to the surgery. An initial injection with N8-GP on
the day of surgery, and afterwards depending on the patients needs. Besides,
surgery related interventions.
Study burden and risks
It's possible that bloodwithdrawals or injections with N8-GP can cause
haemorrhages or discomfort. There is also a very small chance of infection on
the injection site. The patient could also experience side effects from N8-GP.
There is a risk of development of antibodies against N8-GP and/or Factor VIII
that could decrease the effectiveness of future treatments with Factor VIII
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
- Ongoing participation in the pathfinder2 (NN7088-3859) trial and having received *5 doses of N8-GP
- Undergoing major surgery
Exclusion criteria
- Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
- Previous withdrawal from the pathfinder2 (NN7088-3859) trial after administration of trial product, except interruption due to inclusion in this pathfinder3 trial (NN7088-3860)
- The receipt of any investigational medicinal product (except N8-GP) within 30 days prior to enrolment into the trial.
- FVIII inhibitors * 0.6 BU/mL at screening
- Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
- Immune modulating or chemotherapeutic medication
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 | EUCTR2011-001144-30-NL |
| ClinicalTrials.gov | NCT01489111 |
| CCMO | NL38634.078.11 |