Haemophilia B MedDRA version: 14.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: IB1001
Product Code: IB1001
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: NONACOG GAMMA
CAS Number: 1232401-60-3
Current Sponsor code: IB1001
Ot
Sponsors
Inspiration Biopharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Subject’s parent or legal guardian must give written 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 400/mm3) and not receiving immune modulating or chemotherapeutic agents 4) Previously treated subjects with a minimum of 50 exposure days to a factor IX preparation 5) Platelet count at least 150,000/mm3 6) Liver function: alanine transaminase and aspartate transaminase =2 times the upper limit of the normal range 7) Total bilirubin =1.5 times the upper limit of the normal range 8) Renal function: serum creatinine =1.25 times the upper limit of the normal range 9) Willingness to participate in the trial for approximately 6 months (50 exposure days) 10) Age =12 years 11) Hemoglobin =7 g/dL at the time of the screening blood draw Are the trial subjects under 18? yes Number of subjects for this age range: 22 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1) History of factor IX inhibitor = 0.6 Bethesda units 2) Existence of another coagulation disorder 3) Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation 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 subject’s ability to treat bleeding episodes with a factor IX product
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the pharmacokinetics, safety and efficacy of IB1001 in previously treated pediatric subjects with hemophilia B;Secondary Objective: 1. To obtain tolerance and compliance information on pediatric subject response to an intravenously delivered recombinant factor IX product, IB1001 2. To evaluate the safety of IB1001 during the 50 exposure days treatment course ;Primary end point(s): PK: - Cmax - Incremental recovery - AUC - Clearance - t1/2 Efficacy: - Control of breakthrough bleeding; - Degree of hemorrhage control; - Factor IX consumption - Annualized bleeding rates Safety: - Adverse events (acute effects) - Inhibitor development - Thrombogenicity ;Timepoint(s) of evaluation of this end point: PK: samples for factor IX level will be drawn pre-infusion and at 15-30 minutes, 4-6 hours, 24-26 hours and 68-72 hours post-infusion; additional time points at 1-3 hours and 10-14 hours will be drawn when possible Efficacy: A diary to record information about each bleeding episode will be maintained during the treatment and reviewed by the investigator at visits every three months. Safety: Inhibitors: Prior to the first infusion of IB1001, after the first 5 infusions, during the intervals ED 10-15, at the three month visit (approximately ED 25), during the interval ED 50-75, and at study termination Adverse events: at each study visit Thrombogenicity markers: (D-dimer, F1+2, TAT) pre-infusion and at 15-30 minutes , 4-6 hours and 24-26 hours post infusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety: adverse events (long term tolerance and compliance) ;Timepoint(s) of evaluation of this end point: Subject diary will be evaluated at each study visit every three months. | — |
Countries
Brazil, India, Poland, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Operations Europe
Voisin Consulting
Outcome results
None listed