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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 : August 20, 2019
Information provided by (Responsible Party):
Patrick Stiff, Loyola University

Tracking Information
First Submitted Date  ICMJE April 24, 2014
First Posted Date  ICMJE April 28, 2014
Last Update Posted Date August 20, 2019
Study Start Date  ICMJE January 2012
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 24, 2014)
Survival [ Time Frame: 1 year ]
Number of participants that survive post transplant for 1 year.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2014)
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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Vidaza and Valproic Acid Post Allogeneic Transplant for High Risk AML and MDS
Official Title  ICMJE 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)
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Acute Myelogenous Leukemia AML
  • Myelodysplastic Syndrome MDS
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Vidaza and Valproic Acid
Vidaza and Valproic Acid
Intervention: Drug: Vidaza and Valproic Acid
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 24, 2014)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2021
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 89 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Mary Lee, BSN 708-327-2241
Contact: Ceil Petrowsky, MSN 708-327-3306
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT02124174
Other Study ID Numbers  ICMJE 203835
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Patrick Stiff, Loyola University
Study Sponsor  ICMJE Patrick Stiff
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Patrick Stiff, MD Faculty
PRS Account Loyola University
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP