Acute Graft vs Host DiseasePhase 2NCT05090384
Pediatric GVHD Low Risk Steroid Taper Trial
A Phase 2 study testing Prednisone in people with Acute Graft vs Host Disease and Allogeneic Bone Marrow Transplantation, run by John Levine at 11 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Acute Graft vs Host Disease, Allogeneic Bone Marrow Transplantation, Adverse Effects
- What is being tested?
- Prednisone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 20 Oct 2022to 25 Feb 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Newly diagnosed GVHD that meets criteria for Minnesota standard risk (see section 9.0) except isolated skin rash <25% body surface area without other manifestations.
- Ann Arbor 1 GVHD by biomarkers
- GVHD not previously treated systemically (topical therapies and non-absorbed steroids are allowed)
- Any donor type, HLA-match, conditioning regimen is acceptable
- Age 0-21 years at the time of screening
- Signed and dated written informed consent obtained from patient or legal representative and assent from pediatric patients capable of providing assent
Who cannot join
- Patients treated for GVHD with >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
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States10 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Boston Children's Hospital Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Texas Children's Hospital, Baylor College of Medicine
Houston, Texas, 77030
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Medical College of Wisconsin / Children's Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37235
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: Serial Response and Biomarker-Guided Steroid Taper for Children With GVHD
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).
Read the detailed description
Pediatric patients with Minnesota standard risk GVHD that is also Ann Arbor 1 by biomarkers will begin treatment at 0.5 mg/kg/d prednisone (or other steroid equivalent). Patients with favorable clinical responses and biomarker scores at weeks 1 and 2 will have their steroid doses tapered quickly on a weekly basis for four weeks. Patients whose GVHD does not respond or have unfavorable biomarker scores will have their steroid doses increased and be removed from study treatment. The primary endpoint is the proportion of patients whose cumulative steroid dose for the first four weeks is less than half of standard dosing.
- Being tested
- Prednisone
- Keywords
- acute Graft vs Host Disease, aGVHD, Pediatric, steroid taper, MAP, allogeneic hematopoietic stem cell transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Steroid Taper | Experimental | 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 Receives: Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Were Responders (CR, VGPR, or PR) on Day 28 With Low Cumulative Steroid Exposure
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.
Measured: study day 28
Secondary outcomes (11)
Number of Participants Who Achieved a Treatment Response by Day 28
Number 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.
Measured: study day 28
Number of Participants Who Developed Serious Infection
Number 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.
Measured: study day 90
Number of Participants Alive at 6 Months
Overall 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).
Measured: 6 months
Overall Survival at 12 Months
Overall 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).
Measured: 12 months
Number of Participants Who Had Non-Relapse Mortality (NRM) at 6 Months
Number 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.
Measured: 6 months
Cumulative Incidence of Non-Relapse Mortality (NRM) at 12 Months
Cumulative 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.
Measured: 12 months
Number of Participants Who Relapsed at 6 Months
Number of participants who relapsed of the underlying malignancy at 6 months.
Measured: 6 months
Relapse Rate at 12 Months
Relapse rate at 12 months
Relapse, including date of relapse, of the underlying malignancy will be reported.
Measured: 12 months
Cumulative Incidence of Chronic GVHD
Cumulative incidence of chronic GVHD requiring systemic steroid treatment by one year from enrollment
Measured: 12 months
Cumulative Steroid Dose at Study Day 28
Cumulative steroid dose at day 28
Steroid drug and dose is collected weekly for the first 4 weeks of study.
Measured: study day 28
Cumulative Steroid Dose at Study Day 90
Cumulative 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.
Measured: study day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2021
- Study start20 Oct 2022
- Primary completion27 Mar 2025
- Study completion25 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Acute Graft vs Host DiseaseStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- John Levine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, John Levine, Icahn School of Medicine at Mount Sinai
- Investigators
- John E Levine, MD, MS, Principal investigator, Icahn School of Medicine at Mount SinaiMuna Qayed, MD, MS, Principal investigator, Children's Healthcare of Atlanta, Emory University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05090384
- Sponsor's protocol ID
- GCO 21-0541
- Status last verified
- May 2026
- First submitted
- 12 Oct 2021
- Last updated
- 3 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Patient did not consent to this.
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Serial Response and Biomarker-Guided Steroid Taper for Children With GVHD
- ClinicalTrials.gov ID
- NCT05090384
- Acronym / sponsor ID
- GCO 21-0541
- Keywords
- acute Graft vs Host Disease, aGVHD, Pediatric, steroid taper, MAP, allogeneic hematopoietic stem cell transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- John LevineUniversity, hospital or other
- Study start
- 20 Oct 2022 (actual)
- Primary completion
- 27 Mar 2025 (actual)
- Study completion
- 25 Feb 2026 (actual)
- First posted
- 22 Oct 2021 (actual)
- Results
- Posted 8 May 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Pediatric patients with Minnesota standard risk GVHD that is also Ann Arbor 1 by biomarkers will begin treatment at 0.5 mg/kg/d prednisone (or other steroid equivalent). Patients with favorable clinical responses and biomarker scores at weeks 1 and 2 will have their steroid doses tapered quickly on a weekly basis for four weeks. Patients whose GVHD does not respond or have unfavorable biomarker scores will have their steroid doses increased and be removed from study treatment. The primary endpoint is the proportion of patients whose cumulative steroid dose for the first four weeks is less than half of standard dosing.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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
| Arm | Type | Receives |
|---|---|---|
| Steroid Taper | Experimental | Drug: PrednisoneAll 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 |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Newly diagnosed GVHD that meets criteria for Minnesota standard risk (see section 9.0) except isolated skin rash <25% body surface area without other manifestations.
- Ann Arbor 1 GVHD by biomarkers
- GVHD not previously treated systemically (topical therapies and non-absorbed steroids are allowed)
- Any donor type, HLA-match, conditioning regimen is acceptable
- Age 0-21 years at the time of screening
Key exclusion criteria (7 listed)
- Patients treated for GVHD with >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
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 |
|---|---|---|
| PrednisonePrednisone 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 | Drug | Steroid Taper |
Primary outcome
Number of Participants Who Were Responders (CR, VGPR, or PR) on Day 28 With Low Cumulative Steroid Exposure
Time frame: study day 28
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.
11 secondary outcomes
Number of Participants Who Achieved a Treatment Response by Day 28
Time frame: study day 28
Number of Participants Who Developed Serious Infection
Time frame: study day 90
Number of Participants Alive at 6 Months
Time frame: 6 months
Overall Survival at 12 Months
Time frame: 12 months
Number of Participants Who Had Non-Relapse Mortality (NRM) at 6 Months
Time frame: 6 months
Cumulative Incidence of Non-Relapse Mortality (NRM) at 12 Months
Time frame: 12 months
Number of Participants Who Relapsed at 6 Months
Time frame: 6 months
Relapse Rate at 12 Months
Time frame: 12 months
Cumulative Incidence of Chronic GVHD
Time frame: 12 months
Cumulative Steroid Dose at Study Day 28
Time frame: study day 28
Cumulative Steroid Dose at Study Day 90
Time frame: study day 90
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. Patient did not consent to this.
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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. This study is not recruiting at the moment.
John E Levine, MD, MS principal investigator
Icahn School of Medicine at Mount Sinai
Muna Qayed, MD, MS principal investigator
Children's Healthcare of Atlanta, Emory University School of Medicine
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Prednisone in people with Acute Graft vs Host Disease and Allogeneic Bone Marrow Transplantation, run by John Levine at 11 sites in 2 countries.
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 6 inclusion and 7 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: finished, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Canada. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Were Responders (CR, VGPR, or PR) on Day 28 With Low Cumulative Steroid Exposure (time frame: study day 28). 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 Jun 2026; AzTrials last refreshed its copy on 6 Nov 2026.