Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Octocog alfa
  unmodified, full-length recombinant human FVIII (rFVIII)

Первичные конечные точки

• Duration of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs) [Срок оценки: 12 weeks]
• Severity of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs) [Срок оценки: 12 weeks]
• Outcome of treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs) [Срок оценки: 12 weeks]
• Treatment administered for treatment emergent adverse event (TEAEs) and serious adverse events (TESAEs) [Срок оценки: 12 weeks]
• Laboratory test results related to adverse events of special interest (AESIs)rse events (TESAEs) [Срок оценки: 12 weeks]

Вторичные конечные точки (7)

• Total number of infusions per bleed [Срок оценки: 12 weeks]
• Dose of Octocog alfa (IU/kg) per bleed [Срок оценки: 12 weeks]
• Location of bleeds [Срок оценки: 12 weeks]
• Type of bleeds [Срок оценки: 12 weeks]
• Severity of bleeds [Срок оценки: 12 weeks]
• Duration of bleeds [Срок оценки: 12 weeks]
• Investigator rating of response [Срок оценки: 12 weeks]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

Индия · 6 центров

• Department of Medicine Assam Medical College & Hospital — Dibrugarh
• Government of Medical College Kozhikode — Kozhikode
• Sanjay Gandhi Post Graduate Institute & Medical Sciences — Lucknow
• Christian Medical College & Hospital — Ludhiana
• All India Institute Of Medical Sciences — New Delhi
• Sahyadri Super Speciality Hospital — Pune

Идентификаторы

NCT: NCT07446010 · 23153

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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