NCT00887068

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

Completed Phase 3 Results posted Last updated 14 January 2020

What this trial tests

Phase 3 trial testing Azacitidine in Leukemia in 187 participants. Completed in 20 August 2018.

Timeline

21 April 2009

Primary endpoint
20 August 2018

20 August 2018

Quick facts

Lead sponsor	M.D. Anderson Cancer Center
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	187
Start date	21 April 2009
Primary completion	20 August 2018
Estimated completion	20 August 2018
Sites	1 location across United States

Drugs / interventions tested

• Azacitidine (azacitidine) — full drug profile →

Conditions studied

• Leukemia — all drugs for Leukemia →
• AML — all drugs for AML →
• MDS — all drugs for MDS →

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

Adults 18 to 75, any sex, with Leukemia or AML. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Relapse-free Survival (RFS) Primary · 3 years

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

Group	Value	95% CI
AZA Group	2.1	± .43
Standard of Care Group	1.3	± .43

Overall Survival (OS) Secondary · 3 years

Group	Value	95% CI
AZA Group	2.5	± .85
Standard of Care Group	2.6	± .85

Adverse events — posted to ClinicalTrials.gov

Time frame: From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

AZA Group

Serious: 0/87 (0%)

Deaths: 0/87

Standard of Care Group

Serious: 0/94 (0%)

Deaths: 1/94

Other adverse events (7 terms — click to expand)

Reaction	System	AZA Group	Standard of Care Group
Thrombocytopenia	Blood and lymphatic system disorders	—	—
Poor Graft function	Blood and lymphatic system disorders	—	—
Infection	Infections and infestations	—	—
Gastrointestetinal	Gastrointestinal disorders	—	—
Hepatic	Hepatobiliary disorders	—	—
Pulmonary	Respiratory, thoracic and mediastinal disorders	—	—
Skin	Skin and subcutaneous tissue disorders	—	—

Data from ClinicalTrials.gov NCT00887068 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting epigenetic regulators for cancer therapy: mechanisms and advances in clinical trials.
   Cheng Y, He C, Wang M, Ma X, et al · · 2019 · cited 760× · PMID 31871779 · DOI 10.1038/s41392-019-0095-0
2. Targeting epigenetic regulators to overcome drug resistance in cancers.
   Wang N, Ma T, Yu B, Yu B. · · 2023 · cited 213× · PMID 36797239 · DOI 10.1038/s41392-023-01341-7
3. A phase 3 randomized study of 5-azacitidine maintenance vs observation after transplant in high-risk AML and MDS patients.
   Oran B, de Lima M, Garcia-Manero G, Thall PF, et al · · 2020 · cited 155× · PMID 33170934 · DOI 10.1182/bloodadvances.2020002544
4. Proceedings from the National Cancer Institute's Second International Workshop on the Biology, Prevention, and Treatment of Relapse After Hematopoietic Stem Cell Transplantation: part III. Prevention and treatment of relapse after allogeneic transplantation.
   de Lima M, Porter DL, Battiwalla M, Bishop MR, et al · · 2014 · cited 115× · PMID 24018392 · DOI 10.1016/j.bbmt.2013.08.012
5. Relapse of Acute Myeloid Leukemia after Allogeneic Stem Cell Transplantation: Prevention, Detection, and Treatment.
   Rautenberg C, Germing U, Haas R, Kobbe G, et al · · 2019 · cited 86× · PMID 30626126 · DOI 10.3390/ijms20010228
6. Epigenetic regulation in hematopoiesis and its implications in the targeted therapy of hematologic malignancies.
   Zhao A, Zhou H, Yang J, Li M, et al · · 2023 · cited 81× · PMID 36797244 · DOI 10.1038/s41392-023-01342-6
7. Optimizing survival outcomes with post-remission therapy in acute myeloid leukemia.
   Medeiros BC, Chan SM, Daver NG, Jonas BA, et al · · 2019 · cited 62× · PMID 30945331 · DOI 10.1002/ajh.25484
8. The paradigm of drug resistance in cancer: an epigenetic perspective.
   Adhikari S, Bhattacharya A, Adhikary S, Singh V, et al · · 2022 · cited 52× · PMID 35438143 · DOI 10.1042/bsr20211812