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5-Azacytidine With Steroids for Gastrointestinal GVHD (5-AZA FOR GVHD)

NCT06598332 · Status: WITHDRAWN · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL

Last updated 2025-09-22

No results posted yet for this study

Summary

This study aims to evaluate the safety and feasibility of administering AZA in conjunction with steroids as first line therapy for GI GVHD.

A risk for patients who have received a transplant from another donor is graft versus host disease (GVHD). This happens because of differences between the donated cells (graft) and the patient body's cells (host). The new cells from the donor might see the patients body's cells as different and attack them. GVHD can be very serious and cause death. The standard first treatment for GVHD is corticosteroids but not all patients respond and they then have to receive other treatments. In addition, when GHVD involves the gut it can damage stem cells and can cause long term gut problems such as abdominal pain bowel disturbance. In laboratory studies giving a medicine called 5 -azacytidine (AZA) has been able to protect the gut stem cells and help them recover. In this trial the investigators would like to see if AZA can do the same thing when given with steroids in patients with GVHD.

Right now, doctors and researchers don't know the best treatment for GVHD. Acute GVHD is usually treated using high-dose corticosteroids, but these don't always work well. Even if the GVHD gets better when it involves the gut there can be long term damage to gut stem cells. In the laboratory 5 azacytidine (AZA) has been able to protect gut stem cells and help them recover and the investigators would like to learn if this happens in people too.

AZA has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with leukemias. It has also been used post transplant to try and risk the chance of leukemia coming back and to try and treat GVHD but AZA has not been approved by the FDA for the treatment of acute GVHD.

Conditions

  • Graft-Versus-Host Disease(GVHD)

Interventions

DRUG

5-Azacytidine

Patients will be treated with 5 azacytidine at a dose of 32 mg/m2 SQ or IV. The investigators aim to start AZA on Day 1 of steroids but it can be started up to 72 hours after steroids are started . Patients will be premedicated with antiemetics. Patients must receive steroids at a minimum dose of prednisone 2 mg/kg/day PO (or methylprednisolone 1.6 mg/kg/day IV) divided into 1-2 daily doses as therapy for acute GVHD. For patients that weigh over 100 kg, maximal starting dose of prednisone will be 200 mg (or methylprednisolone-equivalent). Steroid Taper for responding patients (Prednisone/Methylprednisolone) Days 1-5 2 mg/kg/day (taper cannot start until 3 days after enrollment) Days 6-10 1.5 mg/kg/day Day 11-15 1.0 mg/kg/day Days 16-20 0.5 mg/kg/day Days 21-28\* 0.25 mg/kg/day Days 29-56 Gradual further taper with a goal of reaching \< 0.2 mg/kg/day of prednisone (or \< 0.16 mg/kg/day of methylprednisolone) by Day 56.

Sponsors & Collaborators

  • The Methodist Hospital Research Institute

    collaborator OTHER

  • Center for Cell and Gene Therapy, Baylor College of Medicine

    collaborator OTHER

  • Baylor College of Medicine

    lead OTHER

Principal Investigators

  • Helen Heslop, MD · Baylor College of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-31
Primary Completion
2028-05-01
Completion
2029-05-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06598332 on ClinicalTrials.gov