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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02124174
Recruitment Status : Recruiting
First Posted : April 28, 2014
Last Update Posted : July 2, 2018
Information provided by (Responsible Party):
Patrick Stiff, Loyola University

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Acute Myelogenous Leukemia AML Myelodysplastic Syndrome MDS Drug: Vidaza and Valproic Acid Phase 2

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)
Study Start Date : January 2012
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2021

Arm Intervention/treatment
Experimental: Vidaza and Valproic Acid
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

Primary Outcome Measures :
  1. Survival [ Time Frame: 1 year ]
    Number of participants that survive post transplant for 1 year.

Secondary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 89 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02124174

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Contact: Mary Lee, BSN 708-327-2241
Contact: Ceil Petrowsky, MSN 708-327-3306

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United States, Illinois
Loyola University Cardinal Bernardin Cancer Center Recruiting
Maywood, Illinois, United States, 60153
Contact: Mary Lee, BSN         
Contact: Ceil Petrowsky, MSN         
Principal Investigator: Patrick Stiff, MD         
Sponsors and Collaborators
Patrick Stiff
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Principal Investigator: Patrick Stiff, MD Faculty

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Responsible Party: Patrick Stiff, Professor, Loyola University Identifier: NCT02124174     History of Changes
Other Study ID Numbers: 203835
First Posted: April 28, 2014    Key Record Dates
Last Update Posted: July 2, 2018
Last Verified: June 2018

Keywords provided by Patrick Stiff, Loyola University:
acute myelogenous leukemia (AML)
myelodysplastic syndrome (MDS)

Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Valproic Acid
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Antimanic Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs