A Prospective, Multicenter, Open-label, Phase IV Post-authorization Safety Study Conducted in India to Assess the Safety and Treatment Outcomes of Octocog Alfa in Real-world Practice for On-demand Treatment of Acute Bleeds in Previously Treated Severe Hemophilia A Patients in India
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 33
- 试验地点
- 6
- 主要终点
- Duration of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
研究概览
简要总结
Hemophilia A is a genetic condition that makes it hard for blood to clot properly. This happens because the body does not have enough of a protein called Factor VIII, which helps stop bleeding. The main goal of treating someone with hemophilia is to stop and prevent bleeding by giving them the missing Factor VIII. This treatment can be given when a person starts bleeding (called on-demand treatment), or it can be given regularly to prevent bleeding (called prophylactic therapy). In India, most people with hemophilia A get treatment only when they have a bleeding episode, and only a few receive regular preventive treatment. Octocog alfa (also known as BAY 81-8973) is a modern, laboratory-made version of Factor VIII. It is made without using any human or animal materials and has special features that help it work better in the body. In India, Octocog alfa is approved for use in adults and children with hemophilia A to:
- Treat and control bleeding episodes when they happen
- Manage bleeding during surgery
- Prevent bleeding by giving regular treatment The safety and effectiveness of Octocog alfa have been shown in several global studies. This new study is required by Indian health authorities to collect information about how safe Octocog alfa is and how well it works in people with hemophilia A who have already received treatment. The study will look at how Octocog alfa is used in real-life medical practice in India, including how doctors prescribe it, how patients use it, and what treatment results they have.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients aged 18 years or older with a documented diagnosis of severe Hemophilia A, defined by a baseline Factor VIII (FVIII) activity level of less than 1% (<0.01 IU/mL) in accordance with the Hemophilia Severity Classification
- •Previously treated with FVIII concentrate(s) (plasma derived or recombinant, including Octocog alfa) either on-demand or prophylactically for at least 100 Exposure Days (EDs).
- •Patients for whom the decision to initiate on-demand treatment with Octocog alfa for acute bleeding was made as per the investigator's routine treatment practice. This will include patients who are already on on-demand treatment with Octocog alfa as well.
- •Written informed consent from the patient or legal representative
排除标准
- •Known contraindication according to the local prescriber information
- •Patients who are participating in an investigational program with interventions outside of routine clinical practice.
- •Patients with any other diagnosis of bleeding/coagulation disorder other than Hemophilia A.
- •Patients who are on ongoing prophylactic treatment with any FVIII concentrate or non-factor treatments like emicizumab.
- •Patients exhibiting any of the following laboratory abnormalities at screening:
- •Known platelet count <100,000 mm3
- •Known Serum Creatinine >2 folds upper the normal limit
- •Known Hepatic AST or ALT >5 folds upper the normal limit
- •Patients who have received an on-demand infusion with any other FVIII (different to Octocog alfa), FVII or Activated prothrombin complex concentrate product (aPCC/FEIBA) 72 hours before the enrollment.
- •History or presence of FVIII inhibitor with a titer ≥ 0.6 with Nijmegen modified Bethesda Assay (NBA), or a clinical history suggestive of an inhibitor necessitating changes to treatment
- •Patient concerns or other barriers precluding adequate understanding or cooperation.
研究组 & 干预措施
Group 1
Male adult patients (aged ≥18 years) with severe Hemophilia A in India, who have been previously treated for at least 100 exposure days to FVIII concentrate(s) and are prescribed Octocog alfa for managing bleeding episodes
干预措施: Octocog alfa (Drug)
结局指标
主要结局
Duration of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
时间窗: 12 weeks
Severity of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
时间窗: 12 weeks
The severity (or intensity) of an AE will be evaluated by the Investigator in accordance with the CTCAE v 5.0. \- Grade 1 (Milde): Asymptomatic or mild symptoms ; clinical or diagnostic observations only ; intervention not indicated. - Grade 2 (Moderate): Minimal, local or invasive intervention indicated, limiting age-appropriate instrumental activities of daily living. - Grade 3 (Severe): Severe or medically significant but not immediately life threatening; hospitalization or prolongation of existing hospitalization indicated ; disabling; limiting self-care activities of daily living. - Grade 4 (Life threatening Consequences): Urgent intervention indicated. -Grade 5 (Death): Related to Adverse Event
Treatment administered for treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
时间窗: 12 weeks
Outcome of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs)
时间窗: 12 weeks
The outcome is defined by the following categories. - Recovered or Resolved: The subject has completely recovered or resolved from the Serious Adverse Event. - Recovered or Resolved with sequelae: As a result of AE , the subject suffered persistent and significant disability / incapacity (e.g., blind, deaf and paralysed ). Any AE recovered with sequelae should be rated as an SAE . - Recovering or Resolving: The subject has begin to recover from the condition or injury , but the event has considered ongoing at a reduced intensity. - Not Recovered or Not Resolved: The AE itself is still present and observable. - Fatal: Death due to SAE, mention the cause of death. Unknown: This term should only be used in cases where the subject is lost to follow up .
Laboratory test results related to adverse events of special interest (AESIs)rse events (TESAEs)
时间窗: 12 weeks
hypersensitivity, inhibitor development Tests used will be as per the routine clinical practice followed by the investigator at his/her hospital
次要结局
- Type of bleeds(12 weeks)
- Investigator rating of response(12 weeks)
- Total number of infusions per bleed(12 weeks)
- Dose of Octocog alfa (IU/kg) per bleed(12 weeks)
- Location of bleeds(12 weeks)
- Severity of bleeds(12 weeks)
- Duration of bleeds(12 weeks)