Precision Medicine for Stem Cell Transplantation

Обзор

A study of patients undergoing haematopoietic stem cell transplantation, a procedure in which patients are infused with stem cells from a donor, resulting in a new immune system that eliminates cancer or replaces diseased bone marrow. This study aims to develop new blood tests that predict the onset of acute graft-versus-host disease (aGvHD) and leukaemia relapse, two life-threatening complications that frequently limit the success of treatment. Predictive tests would allow doctors to individualise prophylaxis and intervene early to abort complications before they develop. The study will also create a large collection of clinically annotated blood samples from 300 transplant recipients to support future research and provide a resource to the transplant research community.

Подробное описание

Precision Medicine for Stem Cell Transplantation (PM-SCT) is a prospective cohort study of patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT). The study aims to develop novel biomarkers that predict the onset of acute graft-versus-host disease (aGvHD) and post-transplant relapse of acute myeloid leukaemia (AML). The study will also build a collection of clinically annotated longitudinal blood samples from 300 HSCT recipients to support additional mechanistic research and provide a resource to the transplant research community.

Patients will be recruited and consented prior to admission for HSCT. Enrolled patients will undergo sequential blood collection beginning prior to conditioning, then on the day of transplant (day 0), followed by days 7, 14, 21, 28, 56 (2 months) and 90 (3 months) post-transplant. Samples will be collected, processed, and stored by the MCRC Biobank. The period of observation for each patient is 6-months. Clinical data will be collected prospectively: on admission, with each blood sample, and at 6-months post-transplant. Bone marrow aspirates taken within this period as part of routine care will also be collected, these are typically performed around day 100 and whenever there is suspicion of disease recurrence. The study aims to identify all patients who develop aGvHD, collecting an additional blood sample at the onset of treatment for those who receive systemic corticosteroids (PO or IV steroid equivalent to ≥0.5mg/kg prednisolone). Additional clinical data will be collected at treatment onset and 7, 14, 21 and 28 days after starting systemic corticosteroids. These assessments will provide the clinical data necessary to establish diagnostic confidence, severity at onset/peak, response to treatment and outcome.

Вмешательства

• Процедура Blood sample collection
  Enrolled patients will undergo sequential blood collection beginning prior to conditioning, then on the day of transplant (day 0), followed by days 7, 14, 21, 28, 56 (2 months) and 90 (3 months) post-transplant. Bone marrow aspirates taken within this period as part of routine care will also be collected, these are typically performed around day 100 and whenever there is suspicion of disease recurrence. The study will also collect an additional blood sample at the onset of treatment for aGvHD
• Процедура Bone marrow aspirate
  Bone marrow aspirates taken within this period as part of routine care will also be collected, these are typically performed around day 100 and whenever there is suspicion of disease recurrence.

Первичные конечные точки

• Development of biomarkers that predict the onset of acute graft-versus-host disease (aGvHD) [Срок оценки: 5 years]

Вторичные конечные точки (1)

• Development of biomarkers that predict the relapse of acute myeloid leukaemia. [Срок оценки: 2.5 years]

Критерии участия

Критерии включения

• Any recipient of allogeneic haematopoietic stem cell transplantation (HSCT)
• Children/infants may participate, there is no age restriction
• Patients participating in other clinical trials remain eligible

Критерии исключения

• Weight <5kg
• Recipients of autologous stem cell transplants

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Великобритания · 3 центра

• Manchester Royal Infirmary — Manchester
• Royal Manchester Children's Hospital — Manchester
• The Christie NHS Foundation Trust — Manchester

Идентификаторы

NCT: NCT05567289 · IRAS ID: 262284

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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