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Corticosteroid

Steroid Tapering for Pediatric Graft-versus-Host Disease

Phase 2

Recruiting

Led By John E Levine, MD, MS

Research Sponsored by John Levine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Ann Arbor 1 GVHD by biomarkers

Performance score (Lansky/Karnofsky) ≥70%

Must not have

Significant liver disease evidenced by direct bilirubin >2 mg/dl or ALT or AST >5 times the upper limit of normal

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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a blood test to help predict which kids with GVHD will respond well to steroids, so that they can be tapered off quickly.

Who is the study for?

This trial is for children and young adults (0-21 years) with a specific immune reaction called GVHD after bone marrow transplant. They must have certain biomarkers, not been treated with systemic steroids for GVHD before, and be in good physical condition. Pregnant individuals or those with severe liver disease, uncontrolled infections, or serious organ dysfunction cannot participate.

What is being tested?

The trial tests if tapering steroid medication quickly can effectively treat GVHD in patients who are likely to respond well based on their biomarker levels. It aims to reduce the duration of high-dose steroid treatment to minimize side effects while monitoring symptoms and quality of life.

What are the potential side effects?

Steroid treatments like prednisone may cause infections, high blood sugar, high blood pressure, muscle weakness, mood changes including depression and anxiety, sleep issues in the short term; long-term effects include bone damage, eye cataracts and stunted growth.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My condition is diagnosed with Grade 1 GVHD based on biomarkers.

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I am mostly active and can care for myself.

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I am 21 years old or younger.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My liver is not functioning well, shown by high bilirubin or liver enzyme levels.

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I do not have an infection that is getting worse despite treatment.

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My cancer is getting worse or not improving, needing a change in my immune therapy.

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I do not need dialysis, mechanical breathing support, or high oxygen.

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I have symptoms similar to chronic GVHD or overlap syndrome at enrollment.

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My kidney function is low, with a filtration rate under 30 ml/min.

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I have been treated for GVHD with steroids for more than 3 days before joining.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Proportion of CR, VGPR, or PR on day 28 with low cumulative steroid exposure

Secondary study objectives

Cumulative incidence of Non-Relapse Mortality (NRM) at 12 months

Cumulative incidence of Non-Relapse Mortality (NRM) at 6 months

Graft-vs-Host Disease

+8 more

Side effects data

From 2016 Phase 3 trial • 854 Patients • NCT00003389

98%

Anemia

93%

Leukocytes decreased

90%

Lymphopenia

84%

Neutrophils decreased

78%

Neuropathy-sensory

75%

Alopecia

74%

Fatigue

67%

Nausea

60%

Hyperglycemia

52%

Constipation

46%

Hypoalbuminemia

40%

Myalgia

34%

Stomatitis

33%

Insomnia

32%

Vomiting

27%

Platelets decreased

26%

Alkaline phosphatase increased

26%

Aspartate aminotransferase increased

23%

Dyspnea

20%

Dyspepsia

19%

Dysphagia

19%

Headache

16%

Anorexia

16%

Arthralgia

15%

Neuropathy-motor

15%

Abdominal pain

14%

Infection w/o neutropenia

14%

Cough

14%

Fever

13%

Rash/desquamation

13%

Diarrhea w/o prior colostomy

12%

Bone pain

11%

Weight gain

11%

Taste disturbance

11%

Anxiety/agitation

10%

Sweating

10%

Radiation dermatitis

Rigors/chills

Dizziness/lightheadedness

Injection site reaction

Dysphagia-esophageal radiation

Hypoglycemia

Blood bilirubin increased

Chest pain

Pain-other

Phlebitis

Creatinine increased

Edema

Pruritus

Hot flashes

Infection w/ grade 3 or 4 neutropenia

Weight loss

Muscle weakness

Depression

Mouth dryness

Transfusion: pRBCs

Pneumonitis/pulmonary infiltrates

Thrombosis/embolism

Febrile neutropenia

Irregular menses

Nail changes

Allergic rhinitis

Allergic reaction

Infection w/ unknown ANC

Syncope

Sinus tachycardia

Dehydration

Neuropathic pain

100%

80%

60%

40%

20%

Study treatment Arm

Arm B (Stanford V)

Arm A (ABVD)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Steroid TaperExperimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Prednisone

2014

Completed Phase 4

~2500

0 / 10Eligibility criteria met