A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A
- Conditions
- Haemophilia AMedDRA version: 14.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 14.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2011-001142-15-IT
- Lead Sponsor
- OVO NORDISK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 186
- Male patients with severe congenital haemophilia A (FVIII activity <1%, according to medical records) - Documented history of at least 150 EDs to other FVIII products - Age = 12 years and body weight = 35 kg (except for Croatia and The Netherlands where the lower age limit will be 18 years)
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 117
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Previous participation in this trial defined as withdrawal after administration N8-GP - Any history of FVIII inhibitors - FVIII inhibitors = 0.6 BU/mL at screening XML File Identifier: qi8L5HhhFIvo/IgCi/fXk85pGo8= Page 10/22 - HIV positive, defined by medical records with CD4+ count =200/µL or a viral load of >400000 copies/mL If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit - Congenital or acquired coagulation disorders other than haemophilia A - Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records - Platelet count < 50,000 platelets/µL (laboratory value at the screening visit) - ALAT > 3 times the upper limit of normal reference ranges at central laboratory - Creatinine level = 1.5 times above upper normal limit (according to central laboratory reference ranges) - Ongoing immune modulating or chemotherapeutic medication
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the immunogenicity of NNC 0129-0000-1003 (hereafter referred to as N8-GP) in previously treated patients with Haemophilia A - To evaluate the clinical efficacy of N8-GP in bleeding prophylaxis (number of bleeds during prophylaxis);Secondary Objective: - To evaluate the clinical efficacy of N8-GP when treating bleeds in patients with haemophilia A - To evaluate the safety of N8-GP when used for prevention of bleeds and treatment of bleeds in patients with haemophilia A - To evaluate PK properties of N8-GP - To evaluate Patient Reported Outcomes - To evaluate the health economic impact of N8-GP treatment - Generation of a population based PK-model for N8-GP;Primary end point(s): - The Incidence rate of FVIII-inhibitors =0.6 BU - Annualised bleeding rate in the prophylaxis arm;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 19 months.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Haemostatic effect of N8-GP when used for treatment of bleeds, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) by counting excellent and good as success and moderate and none as failure.;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 19 months.