Health Condition 1: null- Previously Treated Pediatric Subjects with Hemophilia B State the primary health condition(s) or problem(s) studied
Conditions
Interventions
Intervention1: investigational agent-IB1001: Intervention is the investigational agent IB1001 with no comparator starting from PK phase and for a minimum of about 6 months (50 exposure days) for the t
product should be infused over a period of approximately five minutes
Control Intervention1: Not Applicable: Not Applicable
Sponsors
Aptevo Bio Therapeutics LLC
JSS Medical Research India Limited
Eligibility
Inclusion criteria
Inclusion criteria: 1. Subjectâ??s parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects >=7 years of age, assent will be obtained if required by the institution. For subjects 2. Severe (factor IX activity episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis. In children under 5 years of age, severity may be indicated by the occurrence of one or more joint bleeds at any point in the childâ??s history requiring infusion(s) to replace factor IX. 3. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents 4. Previously treated patients with a minimum of 50 exposure days to a factor IX preparation 8 of 48 Confidential and Proprietary 5. Platelet count at least 150,000/mm3 6. Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] 7. Total bilirubin 8. Renal function: serum creatinine 9. Willingness to participate in the trial for approximately 6 months (50 exposures) 10. Age 11. Hemoglobin >=7 g/dL at the time of the blood draw
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. On medications that could impact hemostasis, such as aspirin 6. 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 7. History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subjectâ??s ability to treat bleeding episodes with a factor IX product 8. Hypersensitivity to the active substance or to any excipients in the investigational products 9. Known allergic reaction to hamster proteins 10. In Poland children weighing
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophiliaTimepoint: 6 Months | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety Assessments Investigators and subject/parent/guardian will report AEs that occur anytime during the study Timepoint: 6 Months | — |
Countries
India, United Kingdom, United States of America
Contacts
Public ContactDr Shariq Anwar
JSS Medical Research India Limited
Outcome results
None listed