Precision Medicine for Stem Cell Transplantation (PM-SCT)

March 31, 2025 updated by: Mark Williams, University of Manchester
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

300

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United Kingdom
      • Manchester, United Kingdom, M20 4BX
        • Recruiting
        • The Christie NHS Foundation Trust
        • Contact:
        • Contact:
          • Adrian Blloor
      • Manchester, United Kingdom, M13 9WL
        • Recruiting
        • Manchester Royal Infirmary
        • Contact:
        • Contact:
          • Fiona Dignan
      • Manchester, United Kingdom, M139WL
        • Recruiting
        • Royal Manchester Children's Hospital
        • Contact:
          • Robert Wynn
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patient undergoing allogeneic haematopoietic stem cell transplantation (HSCT).

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Development of biomarkers that predict the onset of acute graft-versus-host disease (aGvHD)
Time Frame: 5 years
GvHD staging in this study is based on the approach of Harris et al., (2016). The weekly aGvHD assessments will provide the clinical data necessary to establish the confidence of diagnosis, severity at onset, peak severity and outcome. Four weeks of follow-up will determine whether patients achieve a complete response (CR) or very good partial response (VGPR) by day 28. In addition, the 6-month assessment will identify patients who died as a result of aGvHD and those who are alive and off immunosuppression at six months. These validated clinical endpoints will be used to identify biomarker signatures that predict aGvHD severity and response to therapy. Calendar-driven assessments will also be used to identify patients given topical steroids for grade I aGvHD as these patients may need to be excluded from use as negative controls or included as edge cases in future validation studies.
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Development of biomarkers that predict the relapse of acute myeloid leukaemia.
Time Frame: 2.5 years
Relapse remains the leading cause of death for most transplant recipients. Approximately 40% of AML patients suffer disease recurrence, with a median time to relapse of 7 months. The 6-month follow-up assessment will therefore identify many cases of early relapse. The study will remain open for 3 years after the last assessment to allow regular updating of clinical outcome data to identify later cases of relapse. These data and the accompanying clinical samples will be used to identify cytomic and proteomic signatures of immune dysfunction and AML relapse and provide an invaluable resource for studying mechanisms of disease recurrence.
2.5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Manchester

Collaborators

The Christie NHS Foundation Trust

Investigators

  • Principal Investigator: Mark Williams, CRUK Manchester Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 6, 2023

Primary Completion (Estimated)

August 1, 2025

Study Completion (Estimated)

August 1, 2028

Study Registration Dates

First Submitted

September 28, 2022

First Submitted That Met QC Criteria

October 4, 2022

First Posted (Actual)

October 5, 2022

Study Record Updates

Last Update Posted (Actual)

April 3, 2025

Last Update Submitted That Met QC Criteria

March 31, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Other Study ID Numbers

  • IRAS ID: 262284

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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