A prospective single-arm open label study to evaluate Pharmacokinetics properties of Human Plasma Derived Factor IX in severe Factor IX Deficiency
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- To determine the pharmacokinetics of Factor IX in subjects with severe Hemophilia B
研究概览
简要总结
A prospective, single-arm, open-label study to evaluate Pharmacokinetics properties of Human Plasma-Derived Factor IX in severe Factor IX Deficiency.
Primary Objective is to determine the pharmacokinetics of Factor IX in subjects with severe Hemophilia B (FIX deficiency, FIX coagulant levels of <1%).
Secondary Objectives is to assess the safety of Factor IX in subjects with severe Hemophilia B
12 adult male subjects with severe Hemophilia B will be enrolled into the study.
PK will be evaluated following a 10-minute intravenous infusion of a single dose of 50 IU/kg
body weight. For PK assessment, patients should not have received Factor IX or any other
concerned therapy for bleeding event within 7 days prior to dosing.
Screening period validity will be of three weeks. Subjects will receive a single infusion
of factor IX at a dose of 50 IU/ kg of body weight on Day 1 of PK testing (PK-Day 1).
Before start of the PK phase, there must be an at least 7 days wash-out period of any
Factor IX containing product or any other product used for bleeding events.
A total of 10 blood samples (3 mL each) will be collected for adult male subjects.
First sample will be collected within 1 hour prior to drug administration (0.00 hour) and
subsequent samples will be collected at 10 minutes and 0.5, 1, 2, 4, 8, 24, 32 and 48
hours post infusion.
Before the start of infusion, vital signs will be recorded within 1 hour. Blood will be
withdrawn for baseline Factor IX levels assessment. The vital signs will be recorded at 1,
3, 6, 24 and 48 hours’ post-infusion.
The adverse events and concomitant medications will be recorded, including potential
thromboembolic events.
The pharmacokinetic phase of the study will end at the last PK sample, i.e. 48 hours post infusion.
After completing the last PK sample at 48 hours post infusion, all the participating
subjects will enter into the observational phase of the study from next day, i.e. 72 hours
post infusion. During this observational phase, subjects will report to the site about any
bleeding events occurring over the next at least 5-6 months. All the subjects will receive
Factor IX treatment for their bleeding episodes during this observational study period.
Any other treatment will be at the investigator’s discretion. The site can enroll additional
subjects in this observational phase in order to have total 18 subjects in this study phase
(including 12 subjects from the PK phase of the study).
At the end of the observational study phase, all the patients from this observational
study could be evaluated and considered for further participation in an upcoming gene
therapy clinical trial program depending on their willingness and eligibility. The
observational study will conclude once the gene therapy clinical trial is initiated and all
study related activities will also be completed. All the subjects who are selected for the
gene therapy clinical trial program will then undergo activities for that trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 18.00 Year(s) 至 90.00 Year(s)（—）
- 性别
- Male
入选标准
- •Male subjects aged 18 years and above at the start of treatment Documented diagnosis of severe Hemophilia B FIX deficiency FIX coagulant levels of less than 1percent Negative for serology testing of HIV 1 and 2 If serology test results of HBsAg or Hepatitis C is positive subject enrolment will be allowed only if no active disease/virus is confirmed Written and signed informed consent form by adult subject Able to adhere to study schedules and requirements.
排除标准
- •Subjects with history of inhibitors to FIX Any other inherited or acquired bleeding disorder Subjects who had not been previously exposed to pdFIX Subjects with abnormal laboratory parameters like Serum creatinine less than 1 point 5 times of upper normal limit AST or ALT greater than 3 times of upper normal limit Platelet count less than 100000 per micro litre Hemoglobin less than 10 gm per dL Neutrophils less than 1 point 5 into 103 per micro litre Presence or history of Hypersensitivity to pdFIX Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment for the bleeding episode or surgery Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode or surgery Hypersensitivity to human plasma proteins Oesophageal varicose bleeding End-stage liver disease ie Child-Pugh score B or C Ongoing break-through bleeding Positive result for HIV 1 and 2 Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery History of any clinically significant diseases or any acute or chronic medical condition which may in the opinion of investigator affect the conduct of the study including subjects receiving immune-modulating drugs other than antiretroviral chemotherapy such as alpha-interferon prednisone equivalent to 10 mg per day or similar drugs at study start Subject participation in any other clinical trial 30 days prior to administration of IP Any other condition which investigator feels would affect interpretation of the results or render the subject at high risk.
结局指标
主要结局
To determine the pharmacokinetics of Factor IX in subjects with severe Hemophilia B
时间窗: A total of 10 blood samples 3 mL each will be collected First sample will be collected within 1 hour prior to drug administration 0 hour and subsequent samples will be collected at 10 minutes and half hour 1hr 2hr 4hr 8hr 24hr 32hr and 48 hours after infusion
FIX deficiency FIX coagulant levels of less than 1 percent
时间窗: A total of 10 blood samples 3 mL each will be collected First sample will be collected within 1 hour prior to drug administration 0 hour and subsequent samples will be collected at 10 minutes and half hour 1hr 2hr 4hr 8hr 24hr 32hr and 48 hours after infusion
次要结局
- To assess the safety of Factor IX in subjects with severe Hemophilia B(Vital signs will be assessed at screening & within 1 hour prior to infusion of Factor IX & then at 1hr 3hr 6hr 24hr & 48 hours post infusion)
研究者
Dr Nimish Narkar
K J SOMAIYA HOSPITAL AND RESEARCH CENTRE