Completed

Decitabine and Valproic Acid for Relapsed/Refractory Leukemia or Myelodysplastic Syndromes

What is being tested

Decitabine

+ Valproic acid

Drug

Who is being recruted

Bone Marrow Diseases+4

+ Hematologic Diseases

+ Hemic and Lymphatic Diseases

Over 2 Years

+16 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 1 & 2

Interventional

Study Start: January 2004

See protocol details

Summary

Principal SponsorM.D. Anderson Cancer Center

Last updated: January 27, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: January 23, 2004

Actual date on which the first participant was enrolled.

This study explores the combined use of Decitabine (5-aza-2'deoxycytidine) and Valproic Acid in treating patients with leukemia or myelodysplastic syndromes that have not responded well to previous treatments. The study is based on recent findings that suggest a combination of drugs that inhibit DNA methylation and those that inhibit histone deacetylase can work better together to reactivate silenced genes. Decitabine, a drug known for its DNA hypomethylator properties, has shown promise in fighting leukemia at low doses. Valproic Acid, a drug with histone acetylase activity, has been safely used for many years as an anti-seizure medication. The goal of this study is to find a more effective treatment approach for these conditions. The study aims to determine the maximum tolerated dose (MTD) of Valproic Acid when used in combination with Decitabine. Participants will receive the treatment and their response will be evaluated after completing the first cycle, which lasts 4-8 weeks. The MTD is defined as the dose level at which less than two participants experience a dose-limiting toxicity (DLT). This study is crucial in understanding the potential benefits and risks of this combined treatment approach.

Official TitlePhase I/II Study of 5-aza-2'-Deoxycytidine and Valproic Acid in Patients With Relapsed/Refractory Leukemia or Myelodysplastic Syndromes

NCT00075010

Principal SponsorM.D. Anderson Cancer Center

Last updated: January 27, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

54 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 2 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesLeukemiaMyelodysplastic SyndromesNeoplasmsNeoplasms by Histologic Type

Criteria

9 inclusion criteria required to participate

FOR PHASE I COMPONENT OF THE STUDY: Patients with refractory or relapsed: acute myelogenous leukemia (AML), acute lymphocytic leukemia (ALL), and myelodysplastic syndrome (MDS) are eligible. Patients with chronic lymphocytic leukemia (CLL) are eligible if fludarabine based therapy has failed. Patients with chronic myeloid leukemia (CML) are eligible if they have documented hematologic resistance to imatinib mesylate or have not achieved or lost any cytogenetic response to imatinib mesylate after 12 months of therapy.

Untreated patients older than 60 years of age with AML or MDS who refuse or are not eligible for frontline chemotherapy, are eligible.

Performance status of =/< 2 by the ECOG scale.

Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UTMDACC.

Show More Criteria

7 exclusion criteria prevent from participating

Nursing and pregnant females are excluded.

Patients with active and uncontrolled infections are excluded.

Patients with a known ornithine transcarbamylase disorder, history of unexplained coma or a family history of ornithine transcarbamylase disorder are excluded from this study.

Uncontrolled intercurrent illness including, but not limited to symptomatic congestive heart failure, unstable angina pectoris, pancreatitis, psychiatric illness that would limit compliance with study requirements.

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Decitabine 15 mg/m\^2 by vein over 1 hour times 10 days

Study Objectives

Primary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Suspended

M.D. Anderson Cancer Center

Houston, United StatesOpen M.D. Anderson Cancer Center in Google Maps

CompletedOne Study Center