Haematopoietic Stem Cell TransplantationPM-SCTNCT05567289
Precision Medicine for Stem Cell Transplantation
An observational study of Haematopoietic Stem Cell Transplantation, run by University of Manchester at 3 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haematopoietic Stem Cell Transplantation
- What is being tested?
- Blood sample collection and Bone marrow aspirate
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 300 participants
- Where
- 3 sitesUnited Kingdom
- Study dates
- 6 Jun 2023to Aug 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patient undergoing allogeneic haematopoietic stem cell transplantation (HSCT).
Who may be able to join
- 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
Who cannot join
- Weight <5kg
- Recipients of autologous stem cell transplants
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom3 sites, 3 recruiting
- Recruiting
Manchester Royal Infirmary
Manchester, m13 9wl
James Yates, james.yates@mft.nhs.uk
Fiona Dignan
- Recruiting
Royal Manchester Children's Hospital
Manchester, m139wl
james Yates, james.yates@mft.nhs.uk
Robert Wynn
- Recruiting
The Christie NHS Foundation Trust
Manchester, M20 4BX
Ellen Clarke, ellen.clarke4@nhs.net
Adrian Blloor
About this study
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.
Read the 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.
- Conditions
- Haematopoietic Stem Cell Transplantation
- Being tested
- Blood sample collection, Bone marrow aspirate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood sample collectionProcedure or surgery
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 aspirateProcedure or surgery
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 5 years
Secondary outcomes (1)
Development of biomarkers that predict the relapse of acute myeloid leukaemia.
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.
Measured: 2.5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2022
- Study start6 Jun 2023
- Primary completionAug 2025 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haematopoietic Stem Cell TransplantationStudies of the same condition, open and under way.
- More trials run by University of ManchesterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Manchester
- Sponsor type
- University, hospital or other
- Collaborators
- The Christie NHS Foundation Trust
- Responsible party
- Principal investigator, Mark Williams, University of Manchester
- Investigators
- Mark Williams, Principal investigator, CRUK Manchester Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05567289
- Sponsor's protocol ID
- IRAS ID: 262284
- Acronym
- PM-SCT
- Status last verified
- Jan 2025
- First submitted
- 28 Sep 2022
- Last updated
- 3 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Precision Medicine for Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT05567289
- Acronym / sponsor ID
- PM-SCT / IRAS ID: 262284
- Conditions
- Haematopoietic Stem Cell Transplantation
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- University of ManchesterUniversity, hospital or other
- Collaborators
- The Christie NHS Foundation Trust
- Study start
- 6 Jun 2023 (actual)
- Primary completion
- Aug 2025 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 5 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patient undergoing allogeneic haematopoietic stem cell transplantation (HSCT).
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- Weight <5kg
- Recipients of autologous stem cell transplants
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blood sample collectionEnrolled 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). | Procedure or surgery | – |
| Bone marrow aspirateBone 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. | Procedure or surgery | – |
Primary outcome
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.
1 secondary outcome
Development of biomarkers that predict the relapse of acute myeloid leukaemia.
Time frame: 2.5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2025.
- Individual participant dataSharing planned: No
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Not yet recruitingPhase 2
Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers
Core Binding Factor Alpha Subunits, Hematologic Neoplasms
Not yet recruitingPhase 1
Expanded Donor Stem Cells for Poor Graft Function After Stem Cell Transplantation in Children
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Vaginal Microbiome and the Development of Vulvovaginal Graft Versus Host Disease
Bone Marrow Transplant Complications
RecruitingPhase 1
225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Mark Williams principal investigator
CRUK Manchester Institute
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haematopoietic Stem Cell Transplantation, run by University of Manchester at 3 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mark Williams (+441613063240, mark.williams-4@cruk.manchester.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Development of biomarkers that predict the onset of acute graft-versus-host disease (aGvHD) (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2025.