Trial Outcomes & Findings for Pediatric GVHD Low Risk Steroid Taper Trial (NCT NCT05090384)
NCT ID: NCT05090384
Last Updated: 2026-05-08
Results Overview
Number of participants who were responders. Responders are patients with low-risk GVHD (Minnesota standard risk/Ann Arbor 1) who are in CR, VGPR or PR on day 28 and whose cumulative prednisone (or other steroid equivalent) exposure during the first four weeks of treatment is ≤13.5 mg/kg and who have had no intervening additional GVHD therapy for those in CR or VGPR. Complete Response (CR): All evaluable organs (skin, liver, GI tract) stage 0. Very Good Partial Response (VGPR): Any response that approximates a CR with the exception of rash \<25% body surface area. Partial Response (PR): An improvement in one or more organ involved with GVHD symptoms without worsening in others.
COMPLETED
PHASE2
50 participants
study day 28
2026-05-08
Participant Flow
Participant milestones
| Measure |
Steroid Taper
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Overall Study
STARTED
|
50
|
|
Overall Study
COMPLETED
|
44
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Steroid Taper
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Death
|
5
|
Baseline Characteristics
Pediatric GVHD Low Risk Steroid Taper Trial
Baseline characteristics by cohort
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=41 Participants
|
|
Age, Continuous
|
11 years
n=41 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=41 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
16 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
27 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=41 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Black or African American
|
10 Participants
n=41 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=41 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=41 Participants
|
|
Diagnosis
Acute Leukemia
|
24 Participants
n=41 Participants
|
|
Diagnosis
Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN)
|
4 Participants
n=41 Participants
|
|
Diagnosis
Non-Malignant
|
22 Participants
n=41 Participants
|
|
Donor
HLA Matched Related
|
6 Participants
n=41 Participants
|
|
Donor
HLA Mismatched Related
|
12 Participants
n=41 Participants
|
|
Donor
HLA Matched Unrelated
|
12 Participants
n=41 Participants
|
|
Donor
HLA Mismatched Unrelated
|
20 Participants
n=41 Participants
|
|
Conditioning Regimen
Full
|
37 Participants
n=41 Participants
|
|
Conditioning Regimen
Reduced
|
13 Participants
n=41 Participants
|
|
Antithymocyte globulin (ATG) used
Yes
|
29 Participants
n=41 Participants
|
|
Antithymocyte globulin (ATG) used
No
|
21 Participants
n=41 Participants
|
|
Graft-Versus-Host Disease (GVHD) Prophylaxis
CNI/MTX (+/- Other)
|
14 Participants
n=41 Participants
|
|
Graft-Versus-Host Disease (GVHD) Prophylaxis
CNI/MMF (+/- Other)
|
16 Participants
n=41 Participants
|
|
Graft-Versus-Host Disease (GVHD) Prophylaxis
CNI +/- Steroids
|
3 Participants
n=41 Participants
|
|
Graft-Versus-Host Disease (GVHD) Prophylaxis
Cyclophosphamide based
|
9 Participants
n=41 Participants
|
|
Graft-Versus-Host Disease (GVHD) Prophylaxis
T-cell depletion
|
8 Participants
n=41 Participants
|
PRIMARY outcome
Timeframe: study day 28Number of participants who were responders. Responders are patients with low-risk GVHD (Minnesota standard risk/Ann Arbor 1) who are in CR, VGPR or PR on day 28 and whose cumulative prednisone (or other steroid equivalent) exposure during the first four weeks of treatment is ≤13.5 mg/kg and who have had no intervening additional GVHD therapy for those in CR or VGPR. Complete Response (CR): All evaluable organs (skin, liver, GI tract) stage 0. Very Good Partial Response (VGPR): Any response that approximates a CR with the exception of rash \<25% body surface area. Partial Response (PR): An improvement in one or more organ involved with GVHD symptoms without worsening in others.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Number of Participants Who Were Responders (CR, VGPR, or PR) on Day 28 With Low Cumulative Steroid Exposure
|
41 Participants
|
SECONDARY outcome
Timeframe: study day 28Number of participants who achieve a treatment response by day 28 of treatment. Treatment responses are defined as complete response (CR), very good partial response (VGPR), or partial response (PR). For a response to be scored as CR, VGPR, or PR on day 28, the patient must be in response on day 28 and have had no intervening systemic therapy for acute GVHD other than steroids.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Number of Participants Who Achieved a Treatment Response by Day 28
|
43 Participants
|
SECONDARY outcome
Timeframe: study day 90Number of patients who develop serious infections (viral, bacterial, fungal, parasitic) Serious infections are defined using the standardized criteria widely used for clinical trials at academic BMT centers, such as life-threatening fungal infections or hemorrhagic cystitis from BK viral infection and include clinically significant CMV infections that require anti-viral treatment regardless of end-organ damage, given the toxicity of such treatments. Serious infections include any viral, bacterial, fungal or parasitic infections that requires systemic treatment.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Number of Participants Who Developed Serious Infection
|
15 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOverall survival assessed by number of participants alive at 6 months OS is defined as the time from the first dose of study treatment to the date of death (whatever the cause).
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Number of Participants Alive at 6 Months
|
47 Participants
|
SECONDARY outcome
Timeframe: 12 monthsOverall survival at 12 months OS is defined as the time from the first dose of study treatment to the date of death (whatever the cause).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 monthsNumber of Participants who had NRM at 6 months Survival will be tracked during the study, any deaths will be collected. Any death that occurs after hematopoietic stem cell transplantation (HCT) not attributable to relapse of the underlying disease will be considered a non-relapse death.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Number of Participants Who Had Non-Relapse Mortality (NRM) at 6 Months
|
3 Participants
|
SECONDARY outcome
Timeframe: 12 monthsCumulative incidence of NRM at 12 months Survival will be tracked during the study, any deaths will be collected. Any death that occurs after HCT not attributable to relapse of the underlying disease will be considered a non-relapse death.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 monthsNumber of participants who relapsed of the underlying malignancy at 6 months.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Number of Participants Who Relapsed at 6 Months
|
2 Participants
|
SECONDARY outcome
Timeframe: 12 monthsRelapse rate at 12 months Relapse, including date of relapse, of the underlying malignancy will be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 monthsCumulative incidence of chronic GVHD requiring systemic steroid treatment by one year from enrollment
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: study day 28Cumulative steroid dose at day 28 Steroid drug and dose is collected weekly for the first 4 weeks of study.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Cumulative Steroid Dose at Study Day 28
|
11.9 mg/kg
Standard Error 1.4
|
SECONDARY outcome
Timeframe: study day 90Cumulative steroid dose at day 90 Steroid drug and dose is collected weekly for the first 4 weeks of study, and bi-weekly through study day 90.
Outcome measures
| Measure |
Steroid Taper
n=50 Participants
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Cumulative Steroid Dose at Study Day 90
|
14.2 mg/kg
Standard Error 3.7
|
Adverse Events
Steroid Taper
Serious adverse events
| Measure |
Steroid Taper
n=50 participants at risk
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Infections and Infestations
Lung Infection
|
2.0%
1/50 • 1 year
|
|
Metabolism and Nutrition Disorders
Hyponatremia
|
2.0%
1/50 • 1 year
|
|
Gastrointestinal Disorders
Vomiting
|
2.0%
1/50 • 1 year
|
|
Infections and Infestations
Infections and Infestations - Other
|
4.0%
2/50 • 1 year
|
|
Metabolism and Nutrition Disorders
Metabolism and Nutrition Disorders - Other
|
2.0%
1/50 • 1 year
|
|
Nervous System Disorders
Reversible Posterior Leukoencephalopathy Syndrome
|
2.0%
1/50 • 1 year
|
|
Infections and Infestations
Sepsis
|
4.0%
2/50 • 1 year
|
|
Respiratory, Thoracic and Mediastinal Disorders
Respiratory, Thoracic and Mediastinal Disorders - Other
|
6.0%
3/50 • 1 year
|
|
Blood and Lymphatic System Disorders
Febrile Neutropenia
|
2.0%
1/50 • 1 year
|
|
Infections and Infestations
Enterocolitis Infectious
|
2.0%
1/50 • 1 year
|
|
Infections and Infestations
Viremia
|
2.0%
1/50 • 1 year
|
|
Blood and Lymphatic System Disorders
Blood and Lymphatic System Disorders - Other
|
2.0%
1/50 • 1 year
|
|
Vascular Disorders
Vascular Disorders - Other
|
2.0%
1/50 • 1 year
|
|
Metabolism and Nutrition Disorders
Dehydration
|
2.0%
1/50 • 1 year
|
|
Infections and Infestations
Upper Respiratory Infection
|
2.0%
1/50 • 1 year
|
|
Skin and Subcutaneous Tissue Disorders
Skin and Subcutaneous Tissue Disorders - Other
|
2.0%
1/50 • 1 year
|
|
Cardiac Disorders
Cardiac Disroders - Other
|
2.0%
1/50 • 1 year
|
|
Investigations
Alanine Aminotransferase Increased
|
2.0%
1/50 • 1 year
|
|
Vascular Disorders
Hypotension
|
4.0%
2/50 • 1 year
|
|
General Disorders
Fever
|
6.0%
3/50 • 1 year
|
|
Infections and Infestations
Bacteremia
|
2.0%
1/50 • 1 year
|
|
Respiratory, Thoracic and Mediastinal Disorders
Respiratory Failure
|
6.0%
3/50 • 1 year
|
|
Immune System Disorders
Immune System Disorders - Other
|
4.0%
2/50 • 1 year
|
Other adverse events
| Measure |
Steroid Taper
n=50 participants at risk
All enrolled patients start on the same dose of steroids for treatment of GVHD, blood samples are taken at week 1 and 2 post study start and biomarkers plus clinical response determines how steroid treatment is continued
Prednisone: Prednisone starting dose of 0.5 mg/kg; for patients who respond clinically and continue to have low biomarkers will be tapered rapidly; those that are not clinically responding or whose biomarkers increase will be treated per their treating physicians plan or by standard of care
|
|---|---|
|
Blood and Lymphatic System Disorders
Anemia
|
2.0%
1/50 • 1 year
|
|
Investigations
Alanine Aminotransferase Increased
|
4.0%
2/50 • 1 year
|
|
Investigations
Platelet Count Decreased
|
8.0%
4/50 • 1 year
|
|
Investigations
Lymphocyte Count Decreased
|
10.0%
5/50 • 1 year
|
|
Investigations
White Blood Cell Decreased
|
2.0%
1/50 • 1 year
|
|
Investigations
Aspartate Aminotransferase Increased
|
2.0%
1/50 • 1 year
|
|
Investigations
Neutrophil Count Decreased
|
2.0%
1/50 • 1 year
|
|
Metabolism and nutrition disorders
Hyponatremia
|
2.0%
1/50 • 1 year
|
|
Infections and infestations
Infections and Infestations - Other
|
2.0%
1/50 • 1 year
|
|
Investigations
Blood Bilirubin Increased
|
2.0%
1/50 • 1 year
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
2.0%
1/50 • 1 year
|
|
Metabolism and nutrition disorders
Hypokalemia
|
2.0%
1/50 • 1 year
|
Additional Information
Dr. John E. Levine
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place