A research study investigating nonacog beta pegol (N9-GP) for treatment and prevention of bleedings in Chinese people with haemophilia B
- Conditions
- Haemophilia BMedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2021-004947-25-Outside-EU/EEA
- Lead Sponsor
- ovo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Male
- Target Recruitment
- Not specified
1. Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
2. Male Chinese patient with moderate to severe congenital haemophilia B with a FIX activity =2% according to medical records.
3. Aged 12-70 years (both inclusive) at the time of signing informed consent.
4. History of at least 100 EDs to products containing FIX.
5. Patients currently on prophylaxis or patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months.
6. The patient, legally authorised representative (LAR) and/or caregiver are capable of assessing a bleeding episode, keeping a diary, performing home treatment of bleeding episodes and otherwise following the trial procedures.
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 13
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2
1. Known or suspected hypersensitivity to trial product or related products.
2. Previous participation in this trial. Participation is defined as signed informed consent.
3. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer.
4. Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews.
5. Current FIX inhibitors =0.6 BU.
6. HIV positive, defined by medical records, with CD4+ count =200/µL and a viral load >200 particles/µL or >400000 copies/mL within 6 months of the trial entry. If the data are not available in the medical records within the last 6 months, then the test must be performed at the screening visit.
7. Congenital or acquired coagulation disorder other than haemophilia B.
8. 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).
9. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >3 times the upper limit of normal combined with total bilirubin >1.5 times the upper limit of normal at screening.
10. Renal impairment defined as estimated glomerular filtration rate (eGFR) =30 mL/min/1.73 m^2 for serum creatinine measured at screening.
11. Any disorder, except for conditions associated with haemophilia B, which in the investigator’s opinion might jeopardise the patient’s safety or compliance with the protocol.
12. Platelet count <50×10*9/L at screening.
13. Immune modulating or chemotherapeutic medication.
14. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method