Validating biomarkers for predicting chronic graft-versus-host disease in children after stem cell transplant

Biomarker Verification in Pediatric Chronic Graft-Versus-Host Disease: Applied Biomarkers to Minimize Long Term Effects of Childhood/Adolescent Cancer Treatment (ABLE) / Pediatric Transplantation & Cellular Therapy Consortium (PTCTC)

Observational University of British Columbia · NCT04372524

This study is testing a new way to predict which children and young adults might develop chronic graft-versus-host disease after a stem cell transplant, using blood samples collected before and after the procedure.

Quick facts

Study typeObservational
Enrollment350 (estimated)
Ages0 Years to 24 Years
SexAll
SponsorUniversity of British Columbia Academic / other
Drugs / interventionsalemtuzumab, cyclophosphamide
Locations16 sites (San Francisco, California and 15 other locations)
Trial IDNCT04372524 on ClinicalTrials.gov

What this trial studies

This observational study aims to validate a pediatric prognostic biomarker algorithm that predicts the risk of developing chronic graft-versus-host disease (cGvHD) in children and young adults undergoing allogeneic hematopoietic stem cell transplantation. Participants will be enrolled before their transplant and followed for up to 12 months to monitor the development of cGvHD. Blood samples will be collected at specific time points to analyze biomarkers and improve risk stratification for future cGvHD development. The study also seeks to explore diagnostic biomarkers and models predicting responses to cGvHD therapies.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 0 to 24.99 years who are undergoing allogeneic hematopoietic stem cell transplantation.

Not a fit: Patients who have had a second or greater allogeneic transplant or weigh 7 kg or less may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved risk assessment and preemptive therapies for children at risk of developing cGvHD.

How similar studies have performed: Other studies have shown promise in using biomarkers for predicting cGvHD, making this approach both relevant and potentially impactful.

Eligibility criteria

Show full inclusion / exclusion criteria
INCLUSION CRITERIA:

1. Any indication for allogeneic hematopoietic stem cell transplant (malignant or non-malignant)
2. Age 0 - 24.99 years at the time of transplant (on day 0)
3. Any conditioning regimen (including myeloablative or reduced-toxicity/reduced-intensity)
4. Any graft source (bone marrow, peripheral blood, cord blood)
5. Any graft-versus-host disease prophylaxis strategy, including serotherapy such as ATG or alemtuzumab
6. Haploidentical transplants, including post-transplant cyclophosphamide and alpha-beta TCR depletion, are allowed

EXCLUSION CRITERIA:

1. Second or greater allogeneic transplant
2. Weight 7 kg or less
3. Pure CD34+ selected haploidentical stem cell transplant (not including CD34 enrichment used in alpha-beta TCR depleted haploidentical transplants, which is allowed)
4. Inability of a center to follow a patient for the development of late-acute and chronic GVHD until 1-year post-transplant (referral sites who transplant patients from outside institutions should not enroll participants if sending back to the referring site early, such that long-term follow up, blood, and data collection cannot be assured).

Where this trial is running

San Francisco, California and 15 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Chronic Graft-versus-Host-DiseaseLeukemiaAllogeneic Hematopoietic Stem Cell TransplantationBlood CancerNon-Malignant Hematologic and Lymphocytic DisordercGvHDHSCTL-aGvHD
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.