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Clinical Trial VICCHEM1294

Title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Subjects with Acute Myeloid Leukemia in Complete Remission

Principal Investigator(s)

Sanjay Mohan

Details

  • Protocol No. VICCHEM1294
  • Open Date: 02/25/2013
  • Staging: Phase III
  • Age Group: Adults
  • Scope: International
  • Objective: The primary objective of the study is to demonstrate if maintenance therapy with oral azacitidine improves overall survival (OS) compared with placebo in subjects with AML, 55 years of age or older, who have achieved first complete remission (CR) or complete remission with incomplete bloodcount recovery (CRi) after induction with intensive chemotherapy with or without consolidation chemotherapy.
  • Disease Sites: Leukemia
  • Therapies: Chemotherapy - cytotoxic
  • Drugs: Azacitidine (CC-486)
  • Participating Institutions: Vanderbilt University
  • National Clinical Trial ID: NCT01757535
  • Secondary Protocol No: CC-486-AML-001

Description

The purpose of this study is to determine if oral azacitidine (CC-486) is safe and effective as maintenance therapy in continuing the response patients have had with their last AML treatment and improving the quality of that response. Currently there is no standard treatment to maintain or improve patient response and patients are usually just observed or watched. Doctors may decide to follow patients closely through regular physical exams and tests, but without more treatment. Since there is possibility that the disease may come back (relapse), the goal is to search for new treatments that may reduce the risk for relapse or at least to prolong the time before disease relapse. The study drug in this study is either oral azacitidine or placebo (sometimes called a sugar pill). Azacitidine belongs to a class of anti-cancer drugs known as DNA demethylating agents. These types of drugs change how diseased cells in the blood and bone marrow grow and multiply. Vidaza-, a liquid form of azacitidine given by injections is approved in the United States (US) and the European Union (EU) for the treatment of the blood diseases myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMMoL). In the EU, Vidaza is also approved for the treatment of a specific type of AML. Overall, Vidaza is approved in over 30 countries. However, the oral tablet form of azacitidine has not been approved in any country for any disease and is an investigational drug being tested in this study. This study also includes optional biomarker testing of bone marrow aspirate and optional genetic testing of blood and bone marrow aspirate. Biomarkers are substances such as proteins or genes that tell us how the drug is working. Biomarkers in bone marrow will be measured by flow cytometry to detect residual disease. The optional genetic tests (tests involving DNA and RNA) of blood and bone marrow samples will be measured to see how the chemical makeup of bone marrow cells play a role in response to the study drug. On Days 1 of Cycles 1, 3, and 6 about 1 teaspoon [3 mL] of blood will be collected at least two hours apart between 30 minutes and 6 hours after taking the study drug in the clinic. These samples are used to assess how much the study drug is taken into the bloodstream and how long the study drug stays in the body. Patients will also be asked to complete two electronic questionnaires about their wellbeing on Day 1 of each treatment cycle and at the end of treatment.

Eligibility

Ages Eligible for Study:55 Years and older
Genders Eligible for Study:Both
Accepts Healthy Volunteers:No

Criteria

Inclusion Criteria:
1. Male or female subjects ≥ 55 years of age
2. Newly diagnosed, confirmed de novo AML or AML Secondary to prior MDS
3. First CR/CRi with induction therapy ± consolidation therapy within 90 days of achieving CR
4. Eastern Cooperative Oncology Group (ECOG) performance status•0, 1, 2, 3
Exclusion Criteria:
1. AML with inv(16), t(8;21), t(16;16), t(15;17), or t(9;22) or molecular evidence of such translocations
2. Prior bone marrow or stem cell transplantation
3. Candidate for allogeneic bone marrow or stem cell transplant
4. Have achieved CR/CRi following therapy with hypomethylating agents
5. Diagnosis of malignant disease within the previous 12 months
6. Proven Central Nervous System (CNS) leukemia