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NCT01720225

Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk

Completed Phase 2 Results posted Last updated 24 December 2020
What this trial tests

Phase 2 trial testing Decitabine in Leukemia in 113 participants. Completed in 8 January 2020.

Timeline
6 November 2012
Primary endpoint
8 January 2020
8 January 2020

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment113
Start date6 November 2012
Primary completion8 January 2020
Estimated completion8 January 2020
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

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

Who can join

18 and older, any sex, with Leukemia. 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.

Participants With a Response Primary · 56 days

Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with \<5% bone marrow blasts, a peripheral blood granulocyte count \> (1.0 x 10\^9/L, and platelet count \> 100 x 10\^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. Marrow CR is blasts \</= 5% and decreased by \>/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10\^9/L untransfused (if lower than that pre-therapy; or hemoglobin in

GroupValue95% CI
Decitabine49
Azacitidine19
Number of Participants Who Became Transfusion Independent Secondary · 8 weeks

Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.

GroupValue95% CI
Decitabine12
Azacitidine3

Adverse events — posted to ClinicalTrials.gov

Time frame: 3 years, 3 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Decitabine
Serious: 32/73 (44%)
Deaths: 7/73
Azacitidine
Serious: 14/40 (35%)
Deaths: 2/40

Serious adverse events (56 terms)

ReactionSystemDecitabineAzacitidine
Neutropenic FeverBlood and lymphatic system disorders
PneumoniaInfections and infestations
SepsisInfections and infestations
Lung InfectionInfections and infestations
Respiratory FailureRespiratory, thoracic and mediastinal disorders
FeverGeneral disorders
Heart FailureCardiac disorders
Urinary Tract InfectionInfections and infestations
Acute Kidney InjuryRenal and urinary disorders
Acute Respiratory Distress SyndromeRespiratory, thoracic and mediastinal disorders
Altered Mental StatusNervous system disorders
AppendicitisInfections and infestations
AscitesGastrointestinal disorders
Back PainGeneral disorders
Bronchopulmonary HemorrhageRespiratory, thoracic and mediastinal disorders
CellulitisInfections and infestations
Chest PainGeneral disorders
CholecystectomySurgical and medical procedures
DiverticulitisGastrointestinal disorders
Ear InfectionInfections and infestations
Edema LimbsGeneral disorders
FallInjury, poisoning and procedural complications
FatigueGeneral disorders
Food PoisoningInjury, poisoning and procedural complications
GastroenteritisGastrointestinal disorders
Other adverse events (4 terms — click to expand)

ReactionSystemDecitabineAzacitidine
NauseaGastrointestinal disorders
FatigueGeneral disorders
ConstipationGastrointestinal disorders
DiarrheaGastrointestinal disorders

Most-reported serious reactions: Neutropenic Fever, Pneumonia, Sepsis, Lung Infection, Respiratory Failure, Fever, Heart Failure, Urinary Tract Infection.

Data from ClinicalTrials.gov NCT01720225 adverse events section.

Sponsor's own description

The goal of this clinical research study is to compare how two different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule can help to control MDS. The safety of the drugs will also be studied. Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause cancer cells to die. Azacitidine is designed to block certain proteins in cancer cells whose job is to stop the function of the tumor-fighting proteins. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better. This could cause the cancer cells to die.

Publications & conference data

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

  1. Randomized phase 2 study of low-dose decitabine vs low-dose azacitidine in lower-risk MDS and MDS/MPN.
    Jabbour E, Short NJ, Montalban-Bravo G, Huang X, et al · · 2017 · cited 128× · PMID 28774880 · DOI 10.1182/blood-2017-06-788497
  2. 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
  3. Therapy for lower-risk MDS.
    Carraway HE, Saygin C. · · 2020 · cited 42× · PMID 33275714 · DOI 10.1182/hematology.2020000127
  4. Low-Dose Decitabine versus Low-Dose Azacitidine in Lower-Risk MDS.
    Sasaki K, Jabbour E, Montalban-Bravo G, Darbaniyan F, et al · · 2022 · cited 23× · PMID 38319837 · DOI 10.1056/evidoa2200034
  5. Epimutational profile of hematologic malignancies as attractive target for new epigenetic therapies.
    Fratta E, Montico B, Rizzo A, Colizzi F, et al · · 2016 · cited 17× · PMID 27329599 · DOI 10.18632/oncotarget.10033
  6. Clinical Management of Anemia in Patients with Myelodysplastic Syndromes: An Update on Emerging Therapeutic Options.
    Lewis R, Bewersdorf JP, Zeidan AM. · · 2021 · cited 11× · PMID 33531837 · DOI 10.2147/cmar.s240600
  7. Treatment of Anemia in Lower-Risk Myelodysplastic Syndrome.
    Battaglia MR, Cannova J, Madero-Marroquin R, Patel AA. · · 2024 · cited 5× · PMID 38814537 · DOI 10.1007/s11864-024-01217-0
  8. Next-generation therapy for lower-risk MDS.
    Sébert M. · · 2023 · cited 5× · PMID 38066862 · DOI 10.1182/hematology.2023000520

Verify or expand the search:

Other trials of Decitabine

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Other recruiting trials for Leukemia

Currently open trials in the same condition.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

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