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

Primary Purpose

Acute Myelogenous Leukemia AML, Myelodysplastic Syndrome MDS

Status

Unknown status

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Vidaza and Valproic Acid

About this trial

This is an interventional treatment trial for Acute Myelogenous Leukemia AML focused on measuring acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS)

Eligibility Criteria

2 Years - 89 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

All allograft patients > 2 years of age.
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.
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)
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
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:

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

Sites / Locations

Loyola University Cardinal Bernardin Cancer CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Vidaza and Valproic Acid

Arm Description

Vidaza and Valproic Acid

Outcomes

Primary Outcome Measures

Survival

Number of participants that survive post transplant for 1 year.

Secondary Outcome Measures

Disease Relapse

The time to relapse is from Day 0 to the day of first hematologic, cytogenetic, or radiological evidence of recurrent disease.

Full Information

NCT ID

NCT02124174

First Posted

April 24, 2014

Last Updated

April 23, 2021

Sponsor

Patrick Stiff

1. Study Identification

Unique Protocol Identification Number

NCT02124174

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

April 2021

Overall Recruitment Status

Unknown status

Study Start Date

January 2012 (undefined)

Primary Completion Date

January 2022 (Anticipated)

Study Completion Date

January 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Patrick Stiff

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acute Myelogenous Leukemia AML, Myelodysplastic Syndrome MDS

Keywords

acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Vidaza and Valproic Acid

Arm Type

Experimental

Arm Description

Vidaza and Valproic Acid

Intervention Type

Drug

Intervention Name(s)

Vidaza and Valproic Acid

Other Intervention Name(s)

Vidaza, Valproic Acid, Azacitadine

Intervention Description

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.

Primary Outcome Measure Information:

Title

Survival

Description

Number of participants that survive post transplant for 1 year.

Time Frame

1 year

Secondary Outcome Measure Information:

Title

Disease Relapse

Description

The time to relapse is from Day 0 to the day of first hematologic, cytogenetic, or radiological evidence of recurrent disease.

Time Frame

Day 0 to the day of first recurrance

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

89 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: All allograft patients > 2 years of age. 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. Patients with adequate organ function and performance status criteria measured by: Karnofsky score greater than or equal to 70% or Performance status of < or = 2 by the Eastern Cooperative Oncology Group (ECOG) scale Adequate liver function (bilirubin of < 2mg/dL, serum glutamate pyruvate transaminase < 3 * ULN) and renal function (creatinine < 2mg/dL) 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 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: Nursing and pregnant females are excluded. Active and uncontrolled infections will cause patients to be excluded. Patients already receiving valproic acid or receiving other anticonvulsants will be excluded. Low risk AML in complete remission 1, will not be candidates for this study. Patients with an absolute neutrophil count less than 1500 will be excluded Patients with platelets less than 50,000 will be excluded Children less than 2 years of age will be excluded due to increased hepatotoxicity from valproic acid in this age group

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Mary Lee, BSN

Phone

708-327-2241

mlee@luc.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Ceil Petrowsky, MSN

Phone

708-327-3306

cpetrow@luc.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Patrick Stiff, MD

Organizational Affiliation

Faculty

Official's Role

Principal Investigator

Facility Information:

Facility Name

Loyola University Cardinal Bernardin Cancer Center

City

Maywood

State/Province

Illinois

ZIP/Postal Code

60153

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Mary Lee, BSN

First Name & Middle Initial & Last Name & Degree

Ceil Petrowsky, MSN

cpetrow@luc.edu

First Name & Middle Initial & Last Name & Degree

Patrick Stiff, MD

12. IPD Sharing Statement

Learn more about this trial

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

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