Clinical trial · Observational
Biomarker Verification in Pediatric Chronic GvHD: ABLE 2.0 / PTCTC GVH 1901 Study
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)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study will validate a previously developed pediatric prognostic biomarker algorithm aimed at improving prediction of risk for the later development of chronic graft-versus-host disease (cGvHD) in children and young adults undergoing allogeneic hematopoietic stem cell transplant. By developing an early risk stratification of patients into low-, intermediate-, and high-risk for future cGvHD development (based upon their biomarker profile, before the onset of cGvHD), pre-emptive therapies aimed at preventing the onset of cGvHD can be developed based upon an individual's biological risk profile. This study will also continue research into diagnostic biomarkers of cGvHD, and begin work into biomarker models that predict clinical response to cGvHD therapies.
Conditions
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Allogeneic Hematopoietic Stem Cell Transplantation | — | UNRESOLVED | — |
| Blood Cancer | Liquid Tumor | ALIAS | 0.90 |
| Chronic Graft-versus-Host-Disease | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Non-Malignant Hematologic and Lymphocytic Disorder | — | UNRESOLVED | — |
Interventions
Interventions (0)
Data not yet available
Design
Arms and outcomes
Arms (1)
- label
- Allogeneic HSC Transplant recipients
- description
- Five possible patient scenarios are anticipated to occur in those who underwent allogeneic HSCT: * Early event (e.g. death, non-engraftment) occurring before day 100. * No late-acute or chronic GvHD ever develops at any time point in the first year post-transplant (regardless of whether or not classical acute GvHD develops in the first 100 days after transplant). * Early-onset chronic GvHD (including overlap syndrome) occurred before day 60. * Early-onset chronic GvHD (including overlap syndrome) occurred between day 60 and day 100. * Chronic GvHD after Day 100, Late-acute GvHD (de-novo or recurrent) after day 100, or cases of overlap syndrome occurred after day 100.
Primary outcomes (10)
- measure
- Day 60 blood sample collection
- timeFrame
- Day 60 (+/- 7 days) post-transplant
- description
- Validation of prognostic and diagnostic power of cGvHD biomarkers and testing of the biomarker assay performance of Day 60 post-transplant blood sample. Using this algorithm-based assay the investigators will attempt to develop risk assignment for cGvHD and L-aGvHD at Day 60 and determine whether this time point has a similar (or improved) predictive value to the day +100 (+/-14 days). Flow Cytometry and ELISA assays will be used for biomarker measurement.
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 0 Years
- Maximum age
- 24 Years
Show eligibility criteria text
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).
References
Publications (2)
- BACKGROUNDSchultz KR, Kariminia A, Ng B, Abdossamadi S, Lauener M, Nemecek ER, Wahlstrom JT, Kitko CL, Lewis VA, Schechter T, Jacobsohn DA, Harris AC, Pulsipher MA, Bittencourt H, Choi SW, Caywood EH, Kasow KA, Bhatia M, Oshrine BR, Flower A, Chaudhury S, Coulter D, Chewning JH, Joyce M, Savasan S, Pawlowska AB, Megason GC, Mitchell D, Cheerva AC, Lawitschka A, Azadpour S, Ostroumov E, Subrt P, Halevy A, Mostafavi S, Cuvelier GDE. Immune profile differences between chronic GVHD and late acute GVHD: results of the ABLE/PBMTC 1202 studies. Blood. 2020 Apr 9;135(15):1287-1298. doi: 10.1182/blood.2019003186. PMID 32047896
- BACKGROUNDCuvelier GDE, Nemecek ER, Wahlstrom JT, Kitko CL, Lewis VA, Schechter T, Jacobsohn DA, Harris AC, Pulsipher MA, Bittencourt H, Choi SW, Caywood EH, Kasow KA, Bhatia M, Oshrine BR, Flower A, Chaudhury S, Coulter D, Chewning JH, Joyce M, Savasan S, Pawlowska AB, Megason GC, Mitchell D, Cheerva AC, Lawitschka A, West LJ, Pan B, Al Hamarneh YN, Halevy A, Schultz KR. Benefits and challenges with diagnosing chronic and late acute GVHD in children using the NIH consensus criteria. Blood. 2019 Jul 18;134(3):304-316. doi: 10.1182/blood.2019000216. Epub 2019 May 1. PMID 31043425