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

Primary Purpose

Leukemia, AML, MDS

Status

Completed

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

Azacitidine

About this trial

This is an interventional treatment trial for Leukemia focused on measuring 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

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

University of Texas MD Anderson Cancer Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Azacitidine

No Azacitidine

Arm Description

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.

Standard treatment post allogeneic transplant is supportive care only.

Outcomes

Primary Outcome Measures

Relapse-free Survival (RFS)

The time that a participant survives without relapse of the disease.

Secondary Outcome Measures

Overall Survival (OS)

Full Information

NCT ID

NCT00887068

First Posted

April 22, 2009

Last Updated

January 6, 2020

Sponsor

M.D. Anderson Cancer Center

Collaborators

Celgene

1. Study Identification

Unique Protocol Identification Number

NCT00887068

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2020

Overall Recruitment Status

Completed

Study Start Date

April 21, 2009 (Actual)

Primary Completion Date

August 20, 2018 (Actual)

Study Completion Date

August 20, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

M.D. Anderson Cancer Center

Collaborators

Celgene

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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.

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.

6. Conditions and Keywords

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

Leukemia, AML, MDS

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

187 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Azacitidine

Arm Type

Experimental

Arm Description

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.

Arm Title

No Azacitidine

Arm Type

No Intervention

Arm Description

Standard treatment post allogeneic transplant is supportive care only.

Intervention Type

Drug

Intervention Name(s)

Azacitidine

Other Intervention Name(s)

5-Azacitidine, 5-aza, Vidaza, 5-AZC, AZA-CR, Ladakamycin, NSC-102816

Intervention Description

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.

Primary Outcome Measure Information:

Title

Relapse-free Survival (RFS)

Description

The time that a participant survives without relapse of the disease.

Time Frame

3 years

Secondary Outcome Measure Information:

Title

Overall Survival (OS)

Time Frame

3 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Richard E. Champlin, MD, BS

Organizational Affiliation

M.D. Anderson Cancer Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Texas MD Anderson Cancer Center

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

33170934

Citation

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.

Results Reference

derived

Links:

URL

http://www.mdanderson.org

Description

University of Texas MD Anderson Cancer Center Website

Learn more about this trial

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

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