A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
- Conditions
- Health Condition 1: D67- Hereditary factor IX deficiencyHealth Condition 2: D66- Hereditary factor VIII deficiency
- Registration Number
- CTRI/2022/05/042825
- Lead Sponsor
- ovo Nordisk AS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
Participants are eligible to be included in the study only if all the following criteria apply:
1. Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
2. Diagnosis of congenital severe haemophilia A (FVIII <1%) or moderate/severe congenital haemophilia B (FIX =2%), or congenital haemophilia with inhibitors.
3. For arm 1 only: Male aged <12 years of age at the time of signing informed consent.
4. For arm 1 only: Patients with inhibitors (HAwI or HBwI):
a. Patients with HAwI with historical medical records of a total of at least 26 weeks of on-demand treatment within the last 52 weeks prior to enrolment
b. Patients with HBwI with historical medical records of a total of at least 26 weeks of on-demand treatment within the last 52 weeks prior to enrolment
c. Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment
5. For arm 1 only: Patients without inhibitors (HA or HB):
a. Patients with historical medical records of at least 52 weeks of on-demand treatment during the last year prior to enrolment and with at least 3 documented treated bleeds during this period
b. Patients with historical medical records of a total of at least 26 weeks of PPX treatment within the last 52 weeks prior to enrolment
6. For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.
Participants are excluded from the study if any of the following criteria apply:
1. Known or suspected hypersensitivity to study intervention or related products.
2. Previous participation in this study. Participation is defined as signed informed Consent.
3. Participation (i.e., signed informed consent) in any study or programme with investigational
drug other than concizumab within 5 half-lives or 30 days before screening, whichever is
longer.
4. Platelets = 100x109/L at screening.
5. Fibrinogen below laboratory lower normal limit at screening.
6. Hepatic dysfunction defined as AST and/or ALT >3 times the upper limit combined with total
bilirubin >1.5 times the upper limit at screening.
7. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) =30 ml/min/1.73 m2 calculated from serum creatinine measured at screening.
8. Known inherited or acquired coagulation disorder other than congenital haemophilia.
History of thromboembolic diseasea. Current clinical signs of or treatment for thromboembolic
disease. Patients who in the judgement of the investigator are considered at high risk of
thromboembolic events.
10. A known systemic inflammatory condition requiring systemic treatment at screening.
11. Treatment with emicizumab within 180 days before treatment with concizumab.
12. Ongoing or planned Immune Tolerance Induction treatment.
13. Any disorder, except for conditions associated with haemophilia, which in the investigator’s
opinion might jeopardise participant’s safety or compliance with the protocol.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method For inhibitor patients with at least 26 <br/ ><br>weeks on demand treatment during <br/ ><br>the last 52 weeks prior enrolment: <br/ ><br>The number of treated spontaneous <br/ ><br>and traumatic bleeding episodes <br/ ><br> <br/ ><br>For non-inhibitor patients treated on demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodesTimepoint: From start of treatment (week 0) up until the analysis cut-off (week 32 for inhibitor patients) <br/ ><br> <br/ ><br> <br/ ><br>
- Secondary Outcome Measures
Name Time Method