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

NCT02124174 | Unknown Phase 2 DMC

• 1 other identifier: 203835
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Acute Myelogenous Leukemia AML; Myelodysplastic Syndrome MDS

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Survival

  Number of participants that survive post transplant for 1 year.

  1 year

Secondary Outcomes (1)

• Disease Relapse

  Day 0 to the day of first recurrance

Study Arms (1)

Vidaza and Valproic Acid

EXPERIMENTAL

Vidaza and Valproic Acid

Drug: Vidaza and Valproic Acid

Interventions

Vidaza and Valproic Acid DRUG

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.

Also known as: Vidaza, Valproic Acid, Azacitadine

Vidaza and Valproic Acid

Eligibility Criteria

• Age: 2 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Patrick Stiff: lead

Study Sites (1)

Loyola University Cardinal Bernardin Cancer Center

Maywood, Illinois, 60153, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts; Myelodysplastic Syndromes

Interventions

Azacitidine; Valproic Acid

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Refractory; Anemia; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Pentanoic Acids; Valerates; Acids, Acyclic; Carboxylic Acids; Fatty Acids, Volatile; Fatty Acids; Lipids

Study Officials

• Patrick Stiff, MD

  Faculty

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary Lee, BSN

CONTACT

708-327-2241; mlee@luc.edu

Ceil Petrowsky, MSN

CONTACT

708-327-3306; cpetrow@luc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 24, 2014
• First Posted: April 28, 2014
• Study Start: January 1, 2012
• Primary Completion: January 1, 2022
• Study Completion: January 1, 2022
• Last Updated: April 26, 2021

Record last verified: 2021-04

Locations

US (1)