Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129)

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ClinicalTrials.gov Identifier: NCT00887068

Recruitment Status : Completed

First Posted : April 23, 2009

Results First Posted : January 14, 2020

Last Update Posted : January 14, 2020

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborator:

Celgene

Information provided by (Responsible Party):

M.D. Anderson Cancer Center

Study Description

Brief Summary:

The goal of this clinical research study is to learn if Vidaza (azacitidine) will help to control the disease in patients with AML, CMML, or MDS after an allogeneic (donor) stem cell transplant. The safety of this drug will also be studied.

Condition or disease	Intervention/treatment	Phase
Leukemia AML MDS	Drug: Azacitidine	Phase 3

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Detailed Description:

The Study Drug:

Azacitidine is designed to block certain genes in cancer cells whose job is to stop the function of the tumor-fighting genes. By blocking the "bad" genes, the tumor-fighting genes may be able to work better.

Study Groups:

If you are found to be eligible to take part in this study, you will be randomly assigned (as in a flip of a coin) to 1 of 2 groups.

If you are in Group 1, you will receive azacitidine.
If you are in Group 2, you will not receive azacitidine.

Study Drug Administration:

If you are in Group 1, you will receive azacitidine through a needle under your skin on Days 1-5 of each cycle.

Each cycle is 28 days long.

Your dose of azacitidine may be lowered or stopped if certain side effects develop.

Study Visits:

About 2 or 3 days before each cycle and, if your doctor thinks it is needed, on Day 3 of each cycle and 1 time during Weeks 2 and 3 of each cycle, blood (about 4 teaspoons each time) will be drawn for routine tests.

At 3, 6, and 12 months after the stem cell transplant:

You will have a complete medical history and physical exam.
Blood (about 4 teaspoons each time) will be drawn for routine tests.
You will have a bone marrow aspiration to check the status of the disease.

You may come back for study visits more often if the doctor thinks it is needed.

While on study, you will need to stay in Houston for about 3 months after the transplant (this is standard after stem cell transplants).

Length of Study:

You will be on study treatment for up to 1 year (up to 12 cycles of azacitidine). You will be taken off study early if you experience intolerable side effects or the disease gets worse.

End-of-Treatment Visit:

After you complete the planned treatment with azacitidine, you will have an end-of-treatment visit:

You will have a complete medical history and physical exam.
Blood (about 4 teaspoons) will be drawn for routine tests.
You will have a bone marrow aspiration to check the status of the disease.

This is an investigational study. Azacitidine is FDA approved and is commercially available for the treatment of myelodysplastic syndrome.

Up to 246 patients will take part in this study. All will be enrolled at MD Anderson.

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	187 participants
Allocation:	Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Randomized Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129)
Actual Study Start Date :	April 21, 2009
Actual Primary Completion Date :	August 20, 2018
Actual Study Completion Date :	August 20, 2018

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Familial acute myeloid leukemia with mutated CEBPA Cytogenetically normal acute myeloid leukemia Core binding factor acute myeloid leukemia

MedlinePlus related topics: Acute Myeloid Leukemia Leukemia Myelodysplastic Syndromes

Drug Information available for: Azacitidine

Genetic and Rare Diseases Information Center resources: Myelodysplastic Syndromes Myeloid Leukemia Acute Myeloid Leukemia Acute Non Lymphoblastic Leukemia Acute Graft Versus Host Disease

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: Azacitidine Azacitidine 32 mg/m^2 given through a needle under the skin for five consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles.	Drug: Azacitidine 32 mg/m^2 given through a needle under the skin for five consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Other Names: 5-Azacitidine 5-aza Vidaza 5-AZC AZA-CR Ladakamycin NSC-102816
No Intervention: No Azacitidine Standard treatment post allogeneic transplant is supportive care only.

Outcome Measures

Primary Outcome Measures :

Relapse-free Survival (RFS) [ Time Frame: 3 years ]
The time that a participant survives without relapse of the disease.

Secondary Outcome Measures :

Overall Survival (OS) [ Time Frame: 3 years ]

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:	18 Years to 75 Years (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients with a diagnosis of AML (World Health Organization classification: >=20% blasts in the bone marrow and / or peripheral blood) or MDS (International Prognostic Scoring System intermediate-1 or higher) that at the time of allogeneic transplantation were in: - Induction Failure, relapsed disease or second or greater remission; patients in first complete remission that required more than 1 cycle of treatment to achieve the remission, or that have AML evolving from MDS, or that had the following abnormalities: FLT3 mutation, deletion of chromosome 5 or 7, MLL gene rearrangement, or more than or equal to 3 cytogenetics abnormalities. Patients with de novo or therapy-related MDS, CMML, or AML are also eligible, regardless of cytogenetics or molecular rearrangements.
Biphenotypic Leukemia that at the time of allogeneic transplantation was in induction failure, relapsed disease, first, second or greater remission.
Patients must be in complete remission post transplant.
Patient may be enrolled 40 to 100 days after transplant.
Age 18 to 75 years old.
Serum creatinine < 1.8 mg/dL or creatinine clearance greater or equal than 40 cc/min as defined by the Cockcroft-Gault Equation*. a. Males(mL/min):(140-age)*IBW(kg) / 72*(serum creatinine(mg/dl)) b. Females(mL/min):0.85*(140-age)*IBW(kg) / 72*(serum creatinine(mg/dl)).
Serum direct bilirubin < 1.5 mg/dL (unless Gilbert's syndrome).
SGPT </= 200 IU/ml unless related to patient's malignancy.
Be able to understand and sign informed consent.

Exclusion Criteria:

Active uncontrolled infection.
Presence of uncontrolled graft-versus-host disease.
Patients that underwent allogeneic transplantation as a treatment of graft failure.
Pregnancy or breast-feeding (women of childbearing potential, any female who has experienced menarche and who has not undergone surgical sterilization or is not post-menopausal with a positive serum pregnancy test.
Known or suspected hypersensitivity to azacitidine or mannitol.
Patients with advanced malignant hepatic tumors.

Contacts and Locations

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 ClinicalTrials.gov identifier (NCT number): NCT00887068

Locations

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United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030

Sponsors and Collaborators

M.D. Anderson Cancer Center

Celgene

Investigators

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Principal Investigator:	Richard E. Champlin, MD, BS	M.D. Anderson Cancer Center

Study Documents (Full-Text)

Documents provided by M.D. Anderson Cancer Center:

Study Protocol and Statistical Analysis Plan [PDF] November 10, 2015

More Information

Additional Information:

University of Texas MD Anderson Cancer Center Website This link exits the ClinicalTrials.gov site

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Oran B, de Lima M, Garcia-Manero G, Thall PF, Lin R, Popat U, Alousi AM, Hosing C, Giralt S, Rondon G, Woodworth G, Champlin RE. A phase 3 randomized study of 5-azacitidine maintenance vs observation after transplant in high-risk AML and MDS patients. Blood Adv. 2020 Nov 10;4(21):5580-5588. doi: 10.1182/bloodadvances.2020002544. Erratum In: Blood Adv. 2021 Mar 23;5(6):1755-1756.

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Responsible Party:	M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:	NCT00887068
Other Study ID Numbers:	2008-0503 NCI-2012-01259 ( Registry Identifier: NCI CTRP )
First Posted:	April 23, 2009 Key Record Dates
Results First Posted:	January 14, 2020
Last Update Posted:	January 14, 2020
Last Verified:	January 2020

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Studies a U.S. FDA-regulated Drug Product:	Yes
Studies a U.S. FDA-regulated Device Product:	No

Keywords provided by M.D. Anderson Cancer Center:


Leukemia Acute myelogenous leukemia AML Myelodysplastic syndrome MDS Remission Allogeneic stem cell transplant Allotx	Azacitidine 5-Azacitidine 5-aza Vidaza 5-AZC AZA-CR Ladakamycin NSC-102816

Additional relevant MeSH terms:

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Leukemia Preleukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Myelodysplastic Syndromes Neoplasms by Histologic Type Neoplasms Bone Marrow Diseases	Hematologic Diseases Precancerous Conditions Azacitidine Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Enzyme Inhibitors

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