Severe Hemophilia B MedDRA version: 16.0 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Interventions
Product Name: IB1001
Product Code: IB1001
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: trenonacog alfa
CAS Number: 1232401-60-3
Current Sponsor code: IB1001
Sponsors
Cangene Europe Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age of at least 12 years 2. Body Mass Index of = 29, with a minimum body weight of 40 kg 3. Written Institutional Review Board (IRB)/Ethics Committee (EC)-approved informed consent (ICF) 4. Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months) 5. Severe (factor IX activity =2 U/dL) hemophilia B subjects with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior to being placed on prophylaxis 6. Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the PK and Treatment/Continuation Phase of the study 7. Previously treated patients with a minimum of 150 exposure days to a factor IX preparation 8. Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior to PK evaluations 9. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents 10. Platelet count at least 150,000/mm3 11. Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) =2 times the upper limit of the normal range 12. Total bilirubin =1.5 times the upper limit of the normal range 13. Renal function: serum creatinine =1.25 times the upper limit of the normal range 14. Hemoglobin =7 g/dL at the time of the blood draw Are the trial subjects under 18? yes Number of subjects for this age range: 4 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1. History of factor IX inhibitor =0.6 Bethesda units (BU) 2. Existence of another coagulation disorder 3. Evidence of thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC) 4. Use of an investigational drug within 30 days prior to study entry 5. Previous use of IB1001 6. Use of medications that could impact hemostasis, such as aspirin 7. Hypersensitivity to the active substance or to any of the excipients in the investigational products 8. Known allergic reaction to hamster proteins 9. History of poor compliance, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol 10. History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject’s ability to treat bleeding episodes with a factor IX product
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: PK: 2 interim analyses Efficacy: - ABR: end of participation - Others: PK post-infusion, treatment phase visits (5 exposure days; 1, 2, 3 & 6 months) - Surgery: post-surgery Safety: - AEs: PK & repeat PK pre-infusion, all PK & repeat PK timepoints, PK post-infusion, treatment phase visits, pre- & post-surgery - (Non-)inhibitory FIX, anti-CHOP antibodies: screening, PK pre-infusion, PK post-infusion, treatment phase visits, pre- & post-surgery - Thrombogenicity: PK & repeat PK pre-infusion, some PK & repeat PK timepoints - Lab values: screening, PK & repeat PK pre-infusion, some PK & repeat PK timepoints, PK post-infusion, treatment phase visits - Vital signs: screening; same as AEs - Physical exam: screening, PK & repeat PK pre-infusion, PK post-infusion, treatment phase visits;Main Objective: - to evaluate safety of IB1001, - to determine IB1001 pharmacokinetics (PK), and - to evaluate efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and control of hemorrhaging in subjects with severe hemophilia B;Secondary Objective: - to evaluate long-term safety of IB1001, and - to evaluate long-term efficacy of IB1001;Primary end point(s): PK: - Maximum plasma concentration (Cmax) - Area under the curve (AUC0-72, AUC0-8) - Area under the moment curve (AUMC) - Clearance (Cl) - Rate of elimination for the terminal phase (?z) - Terminal half-life (t½) - Incremental recovery - In-vitro recovery (IVR) - Mean residence time (MRT) - Volume of distribution at steady state (Vdss) Efficacy: - Annualized bleeding rate (ABR) - Degree of hemorrhage control - Time from onset of treatment until the bleeding episode stops (as defined by ‘change in pain’ and ‘change in swelling’) - Number of infusions required to treat the bleeding episode - Physical therapy assessment of target joint(s) - Subject’s product tolerance > The following efficacy endpoints will be evaluated for surgery: - Estimated blood loss at time of surgery - Po | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Long-term Efficacy and Safety: cf. primary endpoints for details;Timepoint(s) of evaluation of this end point: Efficacy: continuation phase visits (every 3 months during 6 months), end of study/early withdrawal Safety: - AEs: continuation phase visits, end of study/early withdrawal - (Non-)inhibitory FIX, anti-CHOP antibodies: continuation phase visits, end of study/early withdrawal - Laboratory values: continuation phase visits, end of study/early withdrawal - Vital signs: continuation phase visits, end of study/early withdrawal - Physical exams: continuation phase visits, end of study/early withdrawal | — |
Countries
India, United Kingdom
Contacts
Public ContactClinical Trial Unit
Voisin Consulting
Outcome results
None listed