Clinical trial NCT05567289: Precision Medicine for Stem Cell Transplantation
Official title: Precision Medicine for Stem Cell Transplantation
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Haematopoietic Stem Cell Transplantation
Lead sponsor: University of Manchester
Sponsor class: OTHER
Collaborators: name: The Christie NHS Foundation Trust
First posted: 2022-10-05
Enrollment: 300
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patient undergoing allogeneic haematopoietic stem cell transplantation (HSCT).

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* Weight \<5kg
* Recipients of autologous stem cell transplants

Primary outcomes:
measure: Development of biomarkers that predict the onset of acute graft-versus-host disease (aGvHD)
timeFrame: 5 years
Secondary outcomes:
measure: Development of biomarkers that predict the relapse of acute myeloid leukaemia.
timeFrame: 2.5 years
Other outcomes:
Locations:
city: Manchester
country: United Kingdom
facility: Manchester Royal Infirmary
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
city: Manchester
country: United Kingdom
facility: The Christie NHS Foundation Trust
Study officials:
affiliation: CRUK Manchester Institute
name: Mark Williams
role: PRINCIPAL_INVESTIGATOR
References: