Vidaza and Valproic Acid Post Allogeneic Transplant for High Risk AML and MDS

April 23, 2021 updated by: Patrick Stiff

Maintenance Therapy With Azacitidine and Valproic Acid After Allogeneic Stem Cell Transplant in Patients With High-Risk Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)(Version 1_06 Jan 2012)

Phase II trial combining azacitidine with valproic acid as maintenance therapy post allogeneic stem cell transplantation in patients with high-risk MDS/AML. We hypothesize that adding valproic acid to azacitidine will improve outcomes via both direct anti-tumor and immunologically mediated antitumor response with alloreactive donor lymphocytes, having an additive effect and extending 1 year survival in patient with high-risk AML/MDS after hematopoietic stem cell transplant. Based on aforementioned data from the US Department of Health and Human Services, standard 1 year survival for AML after stem cell transplant is near 40%. We hypothesize that valproic acid and azacitidine will prolong survival, with a 1 year survival goal of 60%. In addition to assessing for 1 year survival, we will have secondary objectives of assessing progression-free survival, relapse, and toxicity. The primary toxicity endpoint from this will be cytopenias and infections.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

To assess the combination of valproic acid and azacitidine in preventing relapse in patients with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant. The primary objective of this study will be determining the 1 year overall survival from combining valproic acid (VPA) with 5-azacytidine (5-aza).

To assess the effect that adding valproic acid to azacitidine will have in patient with high-risk Acute Myeloid Leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic stem cell transplant on the following endpoints

Study Type

Interventional

Enrollment (Anticipated)

50

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Mary Lee, BSN
  • Phone Number: 708-327-2241
  • Email: mlee@luc.edu

Study Contact Backup

  • Name: Ceil Petrowsky, MSN
  • Phone Number: 708-327-3306
  • Email: cpetrow@luc.edu

Study Locations

  • United States
    • Illinois
      • Maywood, Illinois, United States, 60153
        • Recruiting
        • Loyola University Cardinal Bernardin Cancer Center
        • Contact:
          • Mary Lee, BSN
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 89 years (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. All allograft patients > 2 years of age.

  2. Patients will have one of the following malignancies:

    a. Patients with refractory or relapsed: acute myelogenous leukemia (AML) (including inv16, t(8;21) or t(15;17)) or high risk myelodysplastic syndrome (MDS) (defined as bone marrow blasts > or = 5%) are eligible. Patients may be in remission at the time of entry.

  3. Patients with adequate organ function and performance status criteria measured by:

    1. Karnofsky score greater than or equal to 70% or Performance status of < or = 2 by the Eastern Cooperative Oncology Group (ECOG) scale
    2. Adequate liver function (bilirubin of < 2mg/dL, serum glutamate pyruvate transaminase < 3 * ULN) and renal function (creatinine < 2mg/dL)
  4. Signed informed consent indicating that patients are aware of the investigational nature of this study in accordance with the regulations of Loyola University Medical Center
  5. Patients must have undergone allogeneic stem cell transplant within 40-60 days before starting treatment and be self-sufficient in caloric intake along with no active graft vs. host disease

Exclusion Criteria:

  1. Nursing and pregnant females are excluded.
  2. Active and uncontrolled infections will cause patients to be excluded.
  3. Patients already receiving valproic acid or receiving other anticonvulsants will be excluded.
  4. Low risk AML in complete remission 1, will not be candidates for this study.
  5. Patients with an absolute neutrophil count less than 1500 will be excluded
  6. Patients with platelets less than 50,000 will be excluded
  7. Children less than 2 years of age will be excluded due to increased hepatotoxicity from valproic acid in this age group

Study Plan

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

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Vidaza and Valproic Acid
Drug: Vidaza and Valproic Acid

Days 1-5: 5-Azacytidine 40 mg/m^2 daily Days 1-5: +Valproic acid 15 mg/kg daily Days 6-28: Valproic acid 15 mg/kg daily

*treatments will be repeated on the same days of each cycle for up to 4 total cycles. Each cycle will consist of 28 days.

Other Names:
  • Vidaza
  • Valproic Acid
  • Azacitadine

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Survival
Time Frame: 1 year
Number of participants that survive post transplant for 1 year.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease Relapse
Time Frame: Day 0 to the day of first recurrance
The time to relapse is from Day 0 to the day of first hematologic, cytogenetic, or radiological evidence of recurrent disease.
Day 0 to the day of first recurrance

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Patrick Stiff

Investigators

  • Principal Investigator: Patrick Stiff, MD, Faculty

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2012

Primary Completion (ANTICIPATED)

January 1, 2022

Study Completion (ANTICIPATED)

January 1, 2022

Study Registration Dates

First Submitted

April 24, 2014

First Submitted That Met QC Criteria

April 24, 2014

First Posted (ESTIMATE)

April 28, 2014

Study Record Updates

Last Update Posted (ACTUAL)

April 26, 2021

Last Update Submitted That Met QC Criteria

April 23, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 203835

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Myelodysplastic Syndrome MDS

Clinical Trials on Vidaza and Valproic Acid

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Kyrgyzstan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe