Primary Objective: To evaluate the haemostatic effect of N9-GP during surgery procedures in patients with haemophilia B.Key Secondary Objectives• To evaluate the haemostatic effect of N9-GP during surgery and the postoperative period.• To evaluate…
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 a four-point
response scale (Excellent, good, moderate poor), assessed by the
Investigator/Surgeon at the day of surgery
Secondary outcome
Key Secondary Endpoints:
• Consumption of N9-GP (U/kg Body Weight) during surgery and post-operative
period
• Transfusion requirements during surgery and the post-operative period
• Haemoglobin pre and post surgery start (0, 1 h, 24 h and every 24 hours in
the post-operative period)
• AE and SAEs reported during the trial period until the last visit
• Incidence of inhibitors against FIX (>=0.6 BU) until the last visit
Background summary
The rationale for this trial is to investigate efficacy and safety of N9-GP
during surgery in haemophilia B patients. A surgery trial is in accordance with
the draft European Medicines Agency (EMA) guideline on clinical investigation
of recombinant and human plasma-derived Factor IX products. The trial will
provide information on the bleeding-preventive effect during surgery, the
bleeding-arresting haemostatic effect during and after these procedures and the
safety profile of N9-GP in patients with haemophilia B. Based on clinical and
non-clinical studies conducted, N9-GP is a promising drug candidate for
prevention/prophylaxis and on-demand treatment of bleedings in haemophilia B
patients. The completed phase 1 trial showed a mean t* of 93 hours which is
approximately 5 times higher than commercially available FIX concentrates.
Study objective
Primary Objective: To evaluate the haemostatic effect of N9-GP during surgery
procedures in patients with haemophilia B.
Key Secondary Objectives
• To evaluate the haemostatic effect of N9-GP during surgery and the
postoperative period.
• To evaluate the general safety, including immunogenicity of N9-GP, when used
for prevention and treatment of bleeding during surgery and the post-operative
period.
Study design
The design is open-label, multi-centre, un-controlled, efficacy and safety
trial evaluating N9-GP in minimum 10 major surgeries in 5-10 patients.
This trial will provide information on the bleeding-preventive efficacy and
safety profile of N9-GP when administered before, during and after surgery.
Patients enrolled in this trial can be recruited from the pivotal trial
(Paradigm2) or the extension trial (Paradigm4). In addition, new patients can
also be recruited into the present trial. Patients will be offered to continue
on prophylactic treatment or on-demand treatment in the extension trial
(Paradigm4).
The trial will consist of a Screening visit (for new patients) or a Pre-surgery
visit (for patients transferred from the pivotal trial (Paradigm2) and the
extension trial (Paradigm4)), Day of surgery (Visit 2), and a post-operative
period (Day 1-13; Visit 3).
Intervention
Weekly injections with N9-GP in the period prior to the surgery.
An initial injection with N9-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 N9-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 N9-GP.
There is a risk of development of antibodies against N9-GP and/or FIX that
could decrease the effectiveness of future treatments with FIX products.
Flemingweg 18
2408 AV Alphen a/d Rijn
NL
Flemingweg 18
2408 AV Alphen a/d Rijn
NL
Listed location countries
Age
Inclusion criteria
• Male patients, aged 13-70 years, with moderately severe or severe congenital haemophilia B with a FIX activity <=2% according to medical records
NB: In The Netherlands only patients aged 18-70 years will be included
• History of at least 150 exposure days to other FIX products
• Scheduled major surgery
Exclusion criteria
• Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews
• Current FIX inhibitors >=0.6 BU (central laboratory)
• 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)
• ALT >3 times the upper limit of normal reference ranges at screening (central laboratory)
• 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 | EUCTR2010-023070-40-NL |
| ClinicalTrials.gov | NCT01386528 |
| CCMO | NL35219.041.11 |