Azacitidine for Myelodysplastic Syndromes (MDS)

Phase-Based Progress Estimates
M D Anderson Cancer Center, Houston, TX
Myelodysplastic Syndromes (MDS)+12 More
Azacitidine - Drug
All Sexes
Eligible conditions

Study Summary

This study is evaluating whether a combination of two drugs may be more effective than one drug alone in treating patients with acute myeloid leukemia or myelodysplastic syndrome.

See full description

Eligible Conditions

  • Myelodysplastic Syndromes (MDS)
  • Acute Myeloid Leukemia (AML)
  • Myelodysplastic/Myeloproliferative Neoplasms
  • Myeloproliferative Neoplasms (MPNs)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Myelodysplastic Syndromes (MDS)

Study Objectives

This trial is evaluating whether Azacitidine will improve 2 primary outcomes and 7 secondary outcomes in patients with Myelodysplastic Syndromes (MDS). Measurement will happen over the course of Number of days from the date of treatment initiation to the date of documented treatment failure, relapses from CR, or death from any cause, whichever occurs first, assessed up to 5 years.

Year 5
Duration of response
Year 5
Event-free survival
Year 5
Overall survival
Up to 28 days
Minimum safe and biologically effective dose
Up to 5 years
Gene mutations in acute myeloid leukemia
Incidence of adverse events
Median duration to SCT from initiation of combination
Number of patients bridged to stem cell transplant (SCT)
Month 3
Overall response rate

Trial Safety

Safety Progress

1 of 3

Other trials for Myelodysplastic Syndromes (MDS)

Trial Design

1 Treatment Group

Treatment (BRD4 Inhibitor PLX51107, azacitidine)
1 of 1
Experimental Treatment

This trial requires 32 total participants across 1 different treatment group

This trial involves a single treatment. Azacitidine is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

Treatment (BRD4 Inhibitor PLX51107, azacitidine)Patients receive PLX51107 PO QD on days 1-21 and azacitidine SC or IV over 15 minutes on days 8-14 and 22-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 5 years for reporting.

Closest Location

M D Anderson Cancer Center - Houston, TX

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Myelodysplastic Syndromes (MDS) or one of the other 12 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Diagnosis of
AML (World Health Organization [WHO] classification definition of >= 20% blasts), or MDS intermediate-2 score or with > 10% blasts, to include: MDS, myeloproliferative neoplasm (MPN) with 10% blasts or more, or MDS/MPN 10% blasts or more
Patients who have received at least one prior therapy for AML or for MDS will be eligible. Any prior therapy for AML or MDS will be counted as a prior salvage
Intrathecal (IT) therapy for patients with controlled central nervous system (CNS) leukemia at the discretion of the principal investigator (PI). Controlled CNS leukemia is defined by the absence of active clinical signs of CNS disease and no evidence of CNS leukemia on the most recent 2 simultaneous cerebrospinal fluid (CSF) evaluations
Use of one dose of cytarabine (up to 2 g/m^2) or hydroxyurea for patients with rapidly proliferative disease is allowed before the start of study therapy and for the first four weeks on therapy. These medications will be recorded in the case-report form.
Eastern Cooperative Oncology Group (ECOG) performance status 0-2
Measured or calculated creatinine clearance >= 60 mL/min (unless considered due to leukemia)
Total bilirubin < 1.8 mg/dL (unless increase is due to hemolysis, congenital disorder, or leukemia)
Transaminases (serum glutamate pyruvate transaminase [SGPT]) < 2.5 x upper limit of normal (ULN) (unless considered due to leukemia)
Potassium levels should be within institutional normal limits (unless considered due to leukemia)

Patient Q&A Section

What causes myelodysplastic syndromes?

"Myelodysplastic syndromes occur due to abnormalities in the maturation of precursor cells. These abnormalities are thought to arise from a common genetic lesion or to be acquired as a result of environmental exposures or inherited genetic disease. Although the causes of AMPS and AML are not well defined, genes and environmental risk factors are being discussed. We propose to use the term AMPS/AML as a diagnosis for the genetic lesion(s) involved in the pathogenesis of myelodysplastic syndrome and, presumably, leukemia in a large proportion of patients." - Anonymous Online Contributor

Unverified Answer

Can myelodysplastic syndromes be cured?

"In a series of patients with a wide range of clinical and laboratory features, long-term progression-free survival was reported. The study was retrospective and not controlled in terms of the efficacy of the treatment, which was given on the basis of the degree of cytolysis only. However, the results of the treatment are promising and lead to a reevaluation of the strategy of these patients." - Anonymous Online Contributor

Unverified Answer

What are common treatments for myelodysplastic syndromes?

"Many MDSs can be cured with intensive and often complicated protocols of treatment. This is possible in the case of some MDSs with an appropriate cytogenetic abnormality, in which the chromosomal abnormalities themselves can be treated successfully as other chromosome changes. In most cases, MDSs must be treated with supportive care alone. Although more chemotherapeutic agents have been explored, most of them require at least 2 cycles of induction chemotherapy unless the person is not fit for autologous stem-cell transplantation (ASCT) or does not respond to the initial drug. MDSs of low grade that do respond to induction chemotherapy can often be cured with supportive treatment alone." - Anonymous Online Contributor

Unverified Answer

What are the signs of myelodysplastic syndromes?

"It has been shown that, in general, patients with MDS have a poorer prognosis than those without MDS. However, MDS is a heterogeneous entity and more than one sign or symptom could be present. The MDS specific presentation, including cytogenetic abnormalities, marrow morphology, immunophenotype, and presence of the V617F mutation are the principal signs of MDS. In contrast to primary myeloma, bone marrow aspirates are usually normal, thus the presence of a plasma cell dyscrasia should be considered." - Anonymous Online Contributor

Unverified Answer

What is myelodysplastic syndromes?

"MDS manifests as a multitude of different phenotypes which are distinguished from other hematologic malignancies by clinical, bone marrow dysplasia, immunophenotypical, and chromosomal abnormalities. The MDS phenotypic spectrum involves a heterogeneous population with the most severe disease being sub-clinical." - Anonymous Online Contributor

Unverified Answer

How many people get myelodysplastic syndromes a year in the United States?

"Only 60 people per year get MDS. Myeloproliferative disease is much more common than myelodysplastic disease in the United States, which suggests it must have some other explanation. While some studies have found this to be of African heritage, recent studies have not. Thus, the apparent increased risk associated with African ancestry may be due to better cancer screening in African American populations, or may be due to differences in socioeconomic status." - Anonymous Online Contributor

Unverified Answer

How quickly does myelodysplastic syndromes spread?

"Many patients with MDS get progressively less white blood cells and progressively less platelets. This deterioration can lead to a transfusion requirement or even platelet transfusion. The time it takes for this deterioration to occur varies considerably between patients. There is no doubt that a clinical trial tailored to a particular patient can improve outcomes by speeding up the process for each patient. [Power - find clinical trials tailored to your condition (MYELODYSPLASTICSYNDROMES) if you want to get your blood count back up to normal." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in azacitidine for therapeutic use?

"The latest developments are limited to a more frequent dosing regimen based on the efficacy and safety of this agent in this setting, coupled with the potential improvement in safety and tolerability. The clinical use of azacitidine remains contraindicated in patients with myelodysplastic syndromes, in patients with a WBC count above 20 x 10(9)/L at the time of diagnosis and in patients who develop a hypersensitivity reaction during the administration of an autologous peripheral blood stem cell transplant." - Anonymous Online Contributor

Unverified Answer

What is azacitidine?

"Overall survival was comparable to other chemotherapy regimens for myelodysplastic syndromes. We found that azacitidine had no increased toxicity, shorter durations of treatment, and reduced number of required hospitalization. Azacitidine was well tolerated in patients with low baseline hemoglobin. Data from a recent study support its use for patients with myelodysplasia." - Anonymous Online Contributor

Unverified Answer

Is azacitidine safe for people?

"Azacitidine is well tolerated and well tolerated in people with MD in addition to other cytopenias. It is therefore unlikely that it will cause any complications for patients with MD. We therefore recommend that azacitidine is prescribed for patients with MD." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets myelodysplastic syndromes?

"Age [is not] a reliable factor that predicts the patient's prognosis [because] there are patient's with MDS of all ages, and the risk of death of MDS depends on the patient's genetic predisposition. A close surveillance of MDS is recommended, and patients with MDS will eventually need a hematopoietic stem cell transplantation to [improves their prognosis] and is an effective and safe procedure (" - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for myelodysplastic syndromes?

"Many MDS patients do require treatment in their lifetime and are willing to participate in clinical trials. Therefore, patients with MDS should be considered even when their disease is localized." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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