A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Patients with Hemophilia B
- Conditions
- Hemophilia BMedDRA version: 20.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2012-005489-37-DE
- Lead Sponsor
- CSL Behring GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 96
Main study inclusion criteria:
For previously treated subjects, either:
• Completed a CSL-sponsored rIX-FP (CSL654) study, including study CSL654_3001 [NCT01496274] or study CSL654_3002 [NCT01662531].
Or:
• Scheduled to have a major non-emergency surgery within approximately 8 weeks from the anticipated date of receiving the first rIX-FP injection.
• Not previously completed a CSL-sponsored rIX-FP lead-in study.
• Male, 12 to 70 years of age.
• Documented severe hemophilia B (FIX activity of = 2%), or confirmed at screening by the central laboratory.
• Subjects who have received FIX products (plasma-derived and / or recombinant FIX) for > 150 EDs, confirmed by their treating physician.
• No confirmed history of FIX inhibitor formation at screening by the central laboratory
For previously untreated subjects:
• Male, up to 18 years of age.
• Documented severe hemophilia B (FIX activity of = 2%), or confirmed at screening by the central laboratory.
• Never previously been treated with FIX clotting factor products (except previous exposure to blood components).
• No confirmed history of FIX inhibitor formation
Surgery substudy inclusion criterion:
• Must require non-emergency surgery
Subcutaneous substudy inclusion criteria:
• Male, at least 18 years of age.
• Subjects currently enrolled in Study CSL654_3003
• Subjects who have received rIX-FP for = 100 EDs (single-dose cohorts) or for = 50 EDs (repeated-dose cohort)
Are the trial subjects under 18? yes
Number of subjects for this age range: 49
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 66
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Main study exclusion criteria:
• Currently receiving a therapy not permitted during the study.
• Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.
For subjects who have previously completed a CSL-sponsored rIX-FP study:
• Unwilling to participate in the study for a total of 100 exposure days.
For subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study:
• Known hypersensitivity (ie, allergic reaction or anaphylaxis) to any FIX product or hamster protein.
• Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
• Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
• Low platelet count, kidney or liver disease.
• Human immunodeficiency virus positive with a CD4 count < 200/mm3.
For previously untreated subjects:
• Known congenital or acquired coagulation disorder other than congenital FIX deficiency (except for vitamin K deficiency of the newborn).
• Known kidney or liver dysfunction or any condition which, in the investigator’s opinion, place the patient at unjustifiable risk.
The surgical sub-study does not have any additional exclusion criteria, although subject(s) in France will not be eligible for the surgery sub-study.
Subcutaneous substudy exclusion criteria:
• Intravenous use of rIX-FP within 14 days of subcutaneous administration of rIX-FP.
•Life-threatening bleeding episode or major surgery during the 3 months prior to substudy entry
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