Hemophilia B MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Interventions
Trade Name: IDELVION
Product Name: Recombinant fusion protein linking coagulation factor IX with Albumin, IDELVION
Product Code: CSL654
Pharmaceutical Form: Powder for solution for injection/infusion
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of rIX-FP as measured by new cases of inhibitors against FIX in subjects, including previously untreated patients (PUPs), with severe hemophilia B. Pharmacokinetic parameters in PUPs will also be investigated.;Secondary Objective: The secondary objectives of the study are: • To evaluate the efficacy of rIX-FP routine prophylaxis when administered at various treatment intervals. • To compare the efficacy of rIX-FP routine prophylaxis between 2 different treatment intervals and versus on-demand treatment. • To further evaluate the safety of rIX-FP. • To evaluate the efficacy of treatment for bleeding episodes in PUPs. ;Primary end point(s): 1. Main study: Total number of subjects who develop inhibitors against factor IX (FIX); 2. Surgery substudy: Investigator’s overall clinical assessment of hemostatic efficacy for surgical prophylaxis; 3. Incremental recovery in previously untreated patients (PUPs). ;Timepoint(s) of evaluation of this end point: to 1: Main study: Approximately 5 years; to 2: Surgery substudy: Immediately after surgery (0 hours) and at up to 3 timepoints thereafter up to 72 hours or discharge, whichever is earliest; to 3: PUPs: Approximately 30 minutes after infusion. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Comparison of annualized bleeding rate between different prophylaxis treatment intervals; 2. Comparison of annualized bleeding rate between 2 different prophylaxis treatment intervals and on-demand treatment; 3. rIX-FP consumed per month per subject during routine prophylaxis treatment; 4. Surgery substudy: The frequency of AEs related to rIX-FP; 5. Surgery substudy: Total number of subjects who develop inhibitors against FIX; 6. Surgery substudy: Total number of subjects who develop antibodies against rIX-FP; 7. Surgery substudy: Predicted and intraoperative estimated blood loss; 8. Surgery substudy: Predicted and actual transfusion requirements; 9. Surgery substudy: Change in hemoglobin levels between baseline, intraoperatively and postoperatively (major surgery only); 10.Overall adverse events (AEs) and rIX-FP-related AEs; 11. Hemostatic response to rIX-FP treatment in PUPs. ;Timepoint(s) of evaluation of this end point: to 1,2 and 3: Approximately 5 years; to 4, 5 and 6: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery); to 7: Predicted blood loss and intraoperative estimated blood loss to be determined before surgery and at the end of surgery, respectively; to 8: Predicted and actual transfusion requirements to be determined before and at the end of surgery, respectively; to 9: Before, during and up to 28 days after surgery; to 10: Approximately 5 years; to 11: Hemostatic response of rIX-FP treatment during the course of the study (up to 5 years). | — |
Countries
Australia, Austria, Bulgaria, Czechia, Czech Republic, France, Germany, Israel, Italy, Japan, Malaysia, Philippines, South Africa, Spain, United States
Contacts
Public ContactClinical Trial Coordinator
CSL Behring GmbH
Outcome results
None listed