Phase 2
N=50
Pediatric GVHD Low Risk Steroid Taper Trial
Acute Graft vs Host Disease · Allogeneic Bone Marrow Transplantation · Adverse Effects
Bottom Line
View on ClinicalTrials.gov: NCT05090384 ↗Enrolled (actual)
50
Serious AEs
40.0%
Results posted
May 2026
Primary outcome: Primary: Number of Participants Who Were Responders (CR, VGPR, or PR) on Day 28 With Low Cumulative Steroid Exposure — 41 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Prednisone (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- John Levine
- Primary completion
- Mar 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Were Responders (CR, VGPR, or PR) on Day 28 With Low Cumulative Steroid Exposure |
41 | — |
| SECONDARY Number of Participants Who Achieved a Treatment Response by Day 28 |
43 | — |
| SECONDARY Number of Participants Who Developed Serious Infection |
15 | — |
| SECONDARY Number of Participants Alive at 6 Months |
47 | — |
| SECONDARY Overall Survival at 12 Months |
— | — |
| SECONDARY Number of Participants Who Had Non-Relapse Mortality (NRM) at 6 Months |
3 | — |
| SECONDARY Cumulative Incidence of Non-Relapse Mortality (NRM) at 12 Months |
— | — |
| SECONDARY Number of Participants Who Relapsed at 6 Months |
2 | — |
| SECONDARY Relapse Rate at 12 Months |
— | — |
| SECONDARY Cumulative Incidence of Chronic GVHD |
— | — |
| SECONDARY Cumulative Steroid Dose at Study Day 28 |
11.9 | — |
| SECONDARY Cumulative Steroid Dose at Study Day 90 |
14.2 | — |
Summary
The standard treatment for acute graft-vs-host disease (GVHD) is to suppress the activity of the donor immune cells using steroid medications such as prednisone. Although most GVHD, especially in children, responds well to treatment, sometimes (around 1/3 of the time) there is either no response to steroids or the response does not last. In those cases, the GVHD can become dangerous and even life-threatening. Unfortunately, doctors cannot predict who will have a good response to treatment based on symptom severity or initial response to steroids. As a result, nearly all children who develop GVHD are treated with long courses of high dose steroids even though that means many patients receive more treatment than they probably need. Steroid treatment can cause short-term complications like infections, high blood sugar, high blood pressure, muscle weakness, depression, anxiety, and problems sleeping and long-term complications like bone damage, cataracts in the eyes, and decreased growth. The risk of these complications increases with higher doses of steroids and longer treatment. It is important to find ways to decrease the steroid treatment in patients who do not need long courses.
The doctors conducting this research have developed a blood test (GVHD biomarkers) that predicts whether a patient will respond well to steroids. The study team found that children who have low GVHD biomarkers at the start of treatment and for the first two weeks of treatment have a very high response rate to steroids. In this study, the study team will monitor GVHD symptoms and biomarkers during treatment and taper steroids quickly in patients who have GVHD that is expected to respond very well to treatment. The study team will assess how many patients respond well to lower steroid dosing and what steroid complications develop. The study team will also use surveys to obtain the patient's own assessment of their quality of life (down to age 5 years).
Eligibility Criteria
Inclusion Criteria
- Newly diagnosed GVHD that meets criteria for Minnesota standard risk (see section 9.0) except isolated skin rash 0.5 mg/kg/day prednisone for any duration or any steroid treatment for GVHD for more than 2 days prior to screening.
- Patients receiving corticosteroids >0.1 mg/kg prednisone (or other steroid equivalent) for any indication within 7 days before the onset of acute GVHD except for adrenal insufficiency, premedication for transfusions/IV medications, or intermittent use for symptom control such as nausea/vomiting
- Relapsed, progressing, or persistent malignancy or other condition (e.g., known declining donor chimerism) requiring withdrawal of systemic immune suppression or donor leukocyte infusion (DLI)
- Patients with uncontrolled infection (i.e., progressive symptoms related to infection despite treatment, persistently positive microbiological cultures despite treatment, viral reactivations unresponsive to treatment, or any other evidence of severe infection)
- A clinical presentation resembling de novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment
- Patients who are pregnant
- Patients requiring mechanical ventilation or cardiac pressor support
Data sourced from ClinicalTrials.gov (NCT05090384). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.