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Набор скоро начнётся NCT07692217

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

— Наблюдательное Hemophilia A Factor VIII (FVIII)

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy.
Кому может быть актуально
Состояния в реестре: Hemophilia A, Factor VIII (FVIII). Базовые параметры: от 6 мес. · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
США
Следующий шаг
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Официальное название

Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (TG-INSIGHT With Joint POCUS)

Обзор

This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII.

Подробное описание

This research study aims to compare the differences in the way children with moderate or severe hemophilia A respond when being treated with prophylactic extended half-life factor VIII based replacement therapy (such as Altuviiio) vs non-FVIII based replacement therapy (such as Hemlibra) as part of their hemophilia treatment to prevent spontaneous joint bleeds. This study will collect information about the patient's joint health, patient outcomes (using questionnaires), and laboratory testing at specific time points over 3 years.

The study will use an overall approach of comparative effectiveness research (CER) methodology. We plan to recruit participants with severe (FVIII \<1%) and moderate (FVIII 1-4%) HA who take extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy as their clinically prescribed treatment. Both are standard of care treatment for persons with Hemophilia A (PwHA). Using CER principals, this study will analyze differences in 1) hemostatic potential and annualized bleeding rates (Aim 1-3) and 4) joint health (structural and functional) using the Hemophilia Joint Health Score (HJHS) and imaging markers to identify subclinical bleeding and early joint tissue changes in PwHA on novel FVIII and non-FVIII therapies already in use in practice (Aim 4). The proposed study will be the first systematic observational comparative effectiveness study in PwHA on two conventional, standard-of-care novel therapies with differing mechanisms of action to evaluate the overall effectiveness of treatment.

Вмешательства

  • Препарат Half-life factor VIII based replacement therapy
    Children with moderate or severe hemophilia A being treated with prophylactic extended half-life factor VIII based replacement therapy (such as Altuviiio) as part of their hemophilia treatment to prevent spontaneous joint bleeds.
  • Препарат Non-FVIII based replacement therapy
    Children with moderate or severe hemophilia A being treated with non-FVIII based replacement therapy (such as Hemlibra) as part of their hemophilia treatment to prevent spontaneous joint bleeds.

Первичные конечные точки

  • Thrombin Generation [Срок оценки: Thrombin generation assay will be measured at 5 time points within a week of receiving dose of medication]
  • Hemophilia Joint Health Score [Срок оценки: Obtained at baseline and every 6 months]
Вторичные конечные точки (5)
  • Annualized bleeding rate [Срок оценки: From enrollment, every 6 months, until 3 years]
  • Health related quality of life - CHO-KLAT [Срок оценки: From enrollment, every 6 months, until 3 years]
  • Health related quality of life - PedHAL [Срок оценки: From enrollment, every 6 months, until 3 years]
  • Health related quality of life - CATCH [Срок оценки: From enrollment, every 6 months, until 3 years]
  • Joint Tissue Changes [Срок оценки: From enrollment, every 6 months, until 3 years]

Критерии участия

Критерии включения

  • Participants with moderate or severe HA who are on prophylaxis with either weekly, biweekly, or every 4-weeks emicizumab or weekly efanesoctocog alfa for at least 2 months.
  • >6 months of age

Критерии исключения

  • Participants with active FVIII inhibitor (>0.5 BU/mL)
  • Presence of an additional bleeding disorder other than hemophilia A

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

США · 1 центр
  • The University of Texas Southwestern Medical Center — Dallas

Идентификаторы

NCT: NCT07692217 · STU20260378

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗