Treatment Response and Biomarker-Guided Steroid Taper for Children with GVHD

Active:	Yes
Cancer Type:	Unknown Primary	NCT ID:	NCT05090384
Trial Phases:	Phase II	Protocol IDs:	21-0541 (primary) NCI-2022-05853
Eligibility:	0 - 21 Years, Male and Female	Study Type:	Supportive care
Study Sponsor:	Icahn School of Medicine at Mount Sinai
NCI Full Details:	http://clinicaltrials.gov/show/NCT05090384

Summary

This phase II trial studies the treatment response for patients with acute graft-versus-host disease (GVHD). GVHD occurs when donor immune cells attack the healthy tissue of a bone marrow or stem cell transplant patient. The standard treatment for GVHD is to lower the activity of the donor cells by using steroid medications such as prednisone. But steroid treatment may cause many complications and 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. Researchers are doing this study to find out how many subjects respond well to lower steroid dosing based on a blood test (GVHD biomarker) and if they develop fewer complications.

Objectives

PRIMARY OBJECTIVE:
I. To improve the proportion of pediatric patients whose low -risk graft-versus-host GVHD is successfully treated with low cumulative doses of steroids from 14% to 42%.

SECONDARY OBJECTIVES:
I. To determine the overall response rate at day 28 of treatment.
II. To calculate the duration of response.
III. To calculate the incidence of serious infections (overall, viral, bacterial, fungal, parasitic) by day 90 of treatment.
IV. To determine the cumulative steroid exposure through days 28 and 90 of treatment.
V. To determine the incidence of 1 year non-relapse mortality (NRM), relapse, overall survival, and chronic GVHD.
VI. To determine the incidence of steroid-refractory GVHD in patients by day 90 of treatment.

EXPLORATORY OBJECTIVES:
I. To measure quality of life (physical functioning and neuropsychiatric well-being).
II. To calculate linear growth velocity in study patients.
III. To calculate the Mount Sinai Acute GVHD International Consortium ([MAGIC] algorithm probability (MAP) at day 28 of treatment.

OUTLINE:
Patients receive prednisone (or equivalent) orally (PO) or intravenously (IV) for 4 weeks in the absence of disease progression or unacceptable toxicity.

After completion of study, patients are followed-up at 42, 56, 90, 180 and 365 days .

Treatment Sites in Georgia

Winship Cancer Institute of Emory University
1365 Clifton Road NE
Building C
Atlanta, GA 30322
404-778-5180
winshipcancer.emory.edu

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