Hemophilia B (congenital coagulation factor IX [FIX] deficiency
Conditions
Interventions
Trade Name: Alprolix
Product Name: Alprolix
Product Code: rFIXFc
Pharmaceutical Form: Powder and solvent for solution for injection
INN
Sponsors
Bioverativ Therapeutics Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Ability of the subject or their parent or legal guardian to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) or equivalent, and/or to provide assent in accordance with national and local regulations. 2. Male, age 2% of normal. Are the trial subjects under 18? yes Number of subjects for this age range: 35 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. History of positive inhibitor testing. A prior history of inhibitors is defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (i.e., equal to or above lower limit of detection). 2. History of hypersensitivity reactions associated with any rFIXFc administration. 3. Exposure to blood components or injection with a FIX concentrate (including plasma derived) other than rFIXFc. 4. Injection with commercially available rFIXFc more than 28 days prior to Screening. 5. More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility. 6. Other coagulation disorder(s) in addition to hemophilia B. 7. Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the subject unsuitable for enrollment (e.g., HIV infection with CD4 lymphocyte count 200 particles/µL, or any other known congenital or acquired immunodeficiency). 8. Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes is allowed. Treatment in these circumstances should not exceed a 14-day duration. 9. Participation within the past 30 days in any other clinical study involving investigational treatment. 10. Current enrollment in any other clinical study involving investigational treatment. 11. Inability to comply with study requirements. 12. Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, make the subject unsuitable for enrolment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to evaluate the safety of rFIXFc in previously untreated subjects with severe hemophilia B.; Secondary Objective: - To evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in previoulsy untreated patients (PUPs). - To evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs. ;Primary end point(s): The primary endpoint of the study is the occurrence of inhibitor development.; Timepoint(s) of evaluation of this end point: Subjects will be tested for inhibitor formation: - at each clinic visit. - at 5 (±1), 10 (±1), 15 (±1), 20 (±1) and 50 EDs - 2 to 4 weeks prior to elective major surgery, preoperatively, postoperatively, and at the Last Postoperative Visit (see Section 4.2.2). For minor surgeries, testing for inhibitors will only be performed if indicated by the nature of the procedure, according to local standard of care. - if inhibitor development is suspected at any time during the study Additional unscheduled testing for inhibitors may be performed, if required by local standards of care | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - the annualized number of bleeding episodes (spontaneous and traumatic) per subject - the annualized number of spontaneous joint bleeding episodes per subject - assessments of response to treatment with rFIXFc for bleeding episodes, using the 4-point bleeding response scale (Investigator assessment for bleeding episodes treated in the clinic; parent or caregiver assessment for all other bleeding episodes) - the total number of EDs per subject per year - total annualized rFIXFc consumption per subject for the prevention and treatment of bleeding episodes - the number of injections and dose per injection of rFIXFc required to resolve a bleeding episode - rFIXFc incremental recovery ;Timepoint(s) of evaluation of this end point: Incidence of bleeding episodes, dose and number of injections, and response to treatment will be monitored by the physician on an ongoing basis and will be obtained from electronic patient diaries (EPDs), electronic case report forms (eCRFs), and medical records. During clinic visits when rFIXFc is injected, blood samples will be taken to assess incremental recovery. | — |
Countries
Australia, Belgium, Czech Republic, Denmark, France, Germany, Ireland, Italy, Netherlands, New Zealand, Poland, Spain, Sweden, Switzerland, United Kingdom, United States
Outcome results
None listed