Hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850
Conditions
Interventions
Trade Name: ELOCTA
Product Name: ELOCTA
Product Code: rFVIIIFc
Pharmaceutical Form: Powder and solvent for solution for injection
INN or
Sponsors
Bioverativ Therapeutics Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1.Ability of the subject or his 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) in accordance with national and local subject privacy regulations. Subjects may provide assent in addition to the parental/guardian consent, if appropriate. 2. Male, age =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Prior history of inhibitor as defined by the reporting laboratory. The historical positive inhibitor test is defined as per local laboratory Bethesda value for a positive inhibitor test (i.e., equal to or above lower level of detection). 2. Measurable inhibitor activity at the Screening Visit, measured using the Nijmegen-modified Bethesda assay performed at the central laboratory. A negative inhibitor test result at the local laboratory may be used initially to determine subject eligibility; however, any subject who is enrolled based on results of the local laboratory must be withdrawn if the central laboratory screening results indicate a positive inhibitor. Subjects actively bleeding and requiring emergent treatment may be enrolled and receive study drug after samples for inhibitor testing at the central laboratory have been obtained, with results pending. However, any such subject must be withdrawn if the central laboratory screening results indicate a positive inhibitor. 3. History of hypersensitivity reactions associated with any IV immunoglobulin administration. 4. Injection with any FVIII replacement product or any blood component prior to confirmation of eligibility. 5. Injection with rFVIIIFc prior to confirmation of eligibility. 6. Other coagulation disorder(s) in addition to hemophilia A. 7. Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the subject unsuitable for enrollment. 8. Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of corticosteroids for the treatment of asthma or management of acute allergic episodes is allowed with the exception of systemic corticosteroid treatment given to children daily or on alternate days at =2 mg/kg per day of prednisone or its equivalent or =20 mg/day if they weigh more than 10 kg with a duration of longer than 14 days. 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 Biogen Idec, make the subject unsuitable for enrollment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Subjects will be tested for inhibitor formation: - at each clinic visit including screening and baseline. - 5 (±1), 10-15, 20-25 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 of protocol).If this does not align with a scheduled visit, an additional visit must be scheduled. 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 the subject will be tested for inhibitors by the central lab Additional unscheduled testing for inhibitors may be performed, if required by local standards of care ;Main Objective: The primary objective of the study is to evaluate the safety of rFVIIIFc in previously untreated subjects with severe hemophilia A.; Secondary Objective: -To evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in PUPs -To evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs -To describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in subjects with inhibitors ;Primary end point(s): The primary endpoint of the study is the occurrence of inhibitor development. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -The annualized number of bleeding episodes per subject - The annualized number of spontaneous joint bleeding episodes per subject - The number of injections and dose per injection of rFVIIIFc required to resolve a bleeding episode - Assessments of response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale - The total number of EDs per subject per year - Total annualized rFVIIIFc consumption per subject for the prevention and treatment of bleeding episodes - rFVIIIFc incremental recovery (IR) - Response to ITI with rFVIIIFc (success, partial success, failure, early withdrawal) ;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 rFVIIIFc is injected, blood samples will be taken to assess incremental recovery. | — |
Countries
Australia, Brazil, Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, New Zealand, Poland, Spain, Sweden, United Kingdom, United States
Outcome results
None listed