Precision Medicine for Stem Cell Transplantation

NCT05567289 | Recruiting

• 1 other identifier: IRAS ID: 262284
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 3

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.

Conditions

Haematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• Development of biomarkers that predict the onset of acute graft-versus-host disease (aGvHD)

  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 Outcomes (1)

• Development of biomarkers that predict the relapse of acute myeloid leukaemia.

  2.5 years

Interventions

Blood sample collection PROCEDURE

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 for those who receive systemic corticosteroids (PO or IV steroid equivalent to ≥0.5mg/kg prednisolone).

Bone marrow aspirate PROCEDURE

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patient undergoing allogeneic haematopoietic stem cell transplantation (HSCT).

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• University of Manchester: lead
• The Christie NHS Foundation Trust: collaborator

Study Sites (3)

Manchester Royal Infirmary

Manchester, m13 9wl, United Kingdom

RECRUITING

Royal Manchester Children's Hospital

Manchester, m139wl, United Kingdom

RECRUITING

The Christie NHS Foundation Trust

Manchester, M20 4BX, United Kingdom

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Cryopreserved plasma and peripheral blood mononuclear cells.

Study Officials

• Mark Williams

  CRUK Manchester Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mark Williams

CONTACT

+441613063240; mark.williams-4@cruk.manchester.ac.uk

Abbey Walker

CONTACT

0161 446 8201; abbey.walker@manchester.ac.uk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 28, 2022
• First Posted: October 5, 2022
• Study Start: June 6, 2023
• Primary Completion: August 1, 2025
• Study Completion (Estimated): August 1, 2028
• Last Updated: April 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

GB (3)