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Идёт набор NCT05277272

INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)

— Наблюдательное Hemophagocytic Lymphohistiocytoses

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
Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
Кому может быть актуально
Состояния в реестре: Hemophagocytic Lymphohistiocytoses. Базовые параметры: Без ограничений · 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
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

INTO-HLH- Insight Into the Natural History and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis (HLH): A Disease Registry for Patients With HLH

Обзор

The purpose of this observational study is to collect data on the natural history of disease of patients with Hemophagocytic Lymphohistiocytosis (HLH) including diagnosis, treatments, responses, and outcomes.

Подробное описание

Hemophagocytic Lymphohistiocytosis (HLH) is a complex, hyperinflammatory syndrome resulting from the interplay of genetic predisposition and various environmental factors. Despite available treatment options for HLH, approximately 30% of patients do not respond to therapy. Moreover, the standard therapy is constrained by its toxicities, and safer treatments are pursued.

There is an unmet need for a deeper understanding of the natural history, clinical/etiologic diversity, complications, and treatment outcomes of patients with HLH, specifically from North America. The proposed study, a collaboration between Cincinnati Children's Hospital Medical Center (CCHMC), Texas Children's Hospital, and Sobi Inc. aims to establish a robust registry that will enable investigators to better define the natural history of HLH.

Первичные конечные точки

  • Time to HLH diagnosis from the initial presentation [Срок оценки: Interval between date of presentation, as defined as the day of appearance of initial HLH symptom, and the date of full HLH diagnosis, as defined by fulfilling the HLH diagnostic criteria, will be measured. Timeframes up to 6 months will be assessed.]
  • Number of patients with an autoimmune disease at the time of HLH diagnosis [Срок оценки: Up to 1 month from HLH diagnosis]
  • Number of patients with malignancy at the time of HLH diagnosis [Срок оценки: Up to 1 month from HLH diagnosis.]
  • Number of patients treated with immune-activating agents before HLH diagnosis [Срок оценки: Up to 1 month before HLH diagnosis.]
  • Number of patients with central nervous system (CNS) involvement during the HLH disease course. [Срок оценки: Up to 1 month from HLH diagnosis.]
  • Frequency of a genetic diagnosis underlying the HLH. [Срок оценки: Up to 1 month from HLH diagnosis.]
  • Number of patients with infections (e.g., EBV, CMV, HHV6, HIV, fungal, bacterial) at the time of diagnosis. [Срок оценки: Up to 1 month from HLH diagnosis.]
  • Number of patients with organ failure. [Срок оценки: Up to 1 year from HLH diagnosis.]
  • Number of patients with long-term disease-related complications. [Срок оценки: Up to 5 years from HLH diagnosis.]
Вторичные конечные точки (6)
  • Treatment response rate to HLH-related treatments. [Срок оценки: Week two from the start of treatment.]
  • Time to response to HLH-related therapy for patients in the registry. [Срок оценки: Assessed up to 12 weeks from start of treatment.]
  • The survival probability of patients in the registry [Срок оценки: From HLH diagnosis to last follow-up or death, whichever comes first, assessed up to 5 years post-HLH diagnosis.]
  • Number of patients who received hematopoietic stem cell transplantation (HSCT) [Срок оценки: From HLH diagnoses up to 5 years post-HLH diagnosis.]
  • Frequency of hematopoietic stem cell transplantation (HSCT) related complications [Срок оценки: From HSCT up to 5 years post HSCT.]
  • Number of participants with treatment-related adverse events >/= 3 as assessed by CTCAE 5.0 [Срок оценки: From initiation of HLH related treatment up to 30 days following discontinuation of treatment.]

Критерии участия

Критерии включения

  • Patients with clinically suspected or confirmed HLH, including those meeting the HLH-2004 diagnostic criteria (primary or secondary forms, including malignancy) and other forms of HLH (macrophage activation syndrome \[MAS\], cytokine release syndrome \[CRS\], etc.)
  • Signed and dated informed consent and assent (adolescents)

Критерии исключения

  • None

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
  • Cincinnati Children's Hospital Medical Center — Cincinnati

Идентификаторы

NCT: NCT05277272 · 2021-0821 · Sobi.HLH-RWE102

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗