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Anti-metabolites

5-Azacytidine + Steroids for Graft-versus-Host Disease

Phase < 1

Waitlist Available

Led By Helen Heslop, MD

Research Sponsored by Baylor College of Medicine

Eligibility Criteria Checklist

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

Must have

Patients 12 years of age or older at time of enrollment

Patients experiencing their initial presentation of stage 1 or greater acute LGI GVHD requiring systemic therapy after allogeneic transplant for any malignant or non-malignant indication using any graft/donor source or conditioning intensity

Must not have

Patients on renal replacement therapy

Significant active cardiac disease within the previous 6 months including NYHA class 4 CHF, Unstable angina, Myocardial infarction

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 30 days after last dose of aza

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to test the safety and effectiveness of using a medication called AZA along with steroids as the first treatment for gastrointestinal graft versus host disease (GVHD). GVHD is a serious condition where

Who is the study for?

This trial is for individuals aged 12 or older who are experiencing their first episode of acute lower gastrointestinal GVHD after an allogeneic transplant. They must not have had more than 72 hours of steroid treatment for GVHD, except topical steroids. A biopsy is encouraged but not mandatory.

What is being tested?

The study tests the safety and effectiveness of combining a drug called AZA with steroids as the initial treatment for GI GVHD. Researchers want to see if AZA can protect gut stem cells and aid recovery in patients suffering from this condition.

What are the potential side effects?

While specific side effects aren't listed here, AZA may cause blood disorders, nausea, vomiting, and increased risk of infection based on its use in other conditions like leukemia.

Eligibility Criteria

Inclusion Criteria

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

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I am 12 years old or older.

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I have a new case of acute lower GI graft-versus-host disease needing treatment after a transplant.

Exclusion Criteria

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

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I am on dialysis.

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I haven't had serious heart problems like heart failure, unstable chest pain, or a heart attack in the last 6 months.

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I have liver problems, including fluid in the abdomen or confusion due to liver disease.

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I need extra oxygen to keep my blood oxygen level above 90%.

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My cancer has returned or is not responding to treatment, and I need to stop immune suppression therapy.

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I do not have any infections, or if I do, they are under control with no signs of getting worse.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 30 days after first dose of aza

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~30 days after first dose of aza

This trial's timeline: 3 weeks for screening, Varies for treatment, and 30 days after first dose of aza for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Feasibility rate

Treatment-related adverse event (tAE) rate

Secondary study objectives

GVHD response rate

Overall survival (OS)

Progression-free survival (PFS)

+1 more

Side effects data

From 2008 Phase 2 trial • 11 Patients • NCT00382590

33%

Anemia

33%

Death

17%

Infection

17%

chest pain

17%

edema

17%

fatigue

17%

depression

17%

Elevated liver function test

17%

Pneumonia

17%

Congestive Heart Failure

17%

Right periocular hemorrhage

17%

Left ocular hemorrhage

17%

Atrail fibrillation

17%

Thrombocytopenia

17%

Arrythmia

17%

Confusion

100%

80%

60%

40%

20%

Study treatment Arm

Ara-C

5-Aza + VPA

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: TreatmentExperimental Treatment1 Intervention

Patients will receive one cycle of AZA through the vein or as a shot under the skin daily for 5 days. This will start at the same time or within 3 days of starting standard treatment for gut GVHD with steroids.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

5-Azacytidine

2015

Completed Phase 2

~270

0 / 8Eligibility criteria met