Vorinostat for Myelodysplastic Syndromes

Waitlist Available · 18+ · All Sexes · Bronx, NY

This study is evaluating the side effects and best dose of vorinostat and azacitidine and to see how well they work in treating patients with myelodysplastic syndromes or acute myeloid leukemia.

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About the trial for Myelodysplastic Syndromes

Eligible Conditions
Myelodysplastic Syndromes · Leukemia, Megakaryoblastic, Acute · Leukemia, Myelomonocytic, Chronic · Anemia, Refractory · Syndrome · Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome · Leukemia, Myelomonocytic, Juvenile · Leukemia, Myeloid · Leukemia, Myeloid, Acute · Preleukemia · Leukemia, Erythroblastic, Acute · Leukemia · Anemia, Refractory, with Excess of Blasts · Myelodysplastic Syndrome With Ring Sideroblasts · Refractory Anemia · Acute Erythroid Leukemia · Myelodysplastic Syndrome With Excess Blasts · Chronic Myelomonocytic Leukemia (CMML) · Recurrent Acute Myeloid Leukemia, Adult · Myelodysplastic Syndromes (MDS)

Treatment Groups

This trial involves 2 different treatments. Vorinostat 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 & 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Pharmacological Study
Laboratory Biomarker Analysis
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved
FDA approved


This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Myelodysplastic Syndromes or one of the other 19 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
, with blast count >10% (but not including blasts in the peripheral blood) at diagnosis To be eligible for the phase I portion of the study, patients must have a diagnosis of either MDS according to French-American-British (FAB) and International Prognostic Scoring System (IPSS) criteria, or a diagnosis of AML according to FAB or World Health Organization (WHO) criteria, with blast count >10% (but not including blasts in the peripheral blood) at diagnosis. show original
Refractory anemia (RA) is a type of anemia where the blood does not contain enough red blood cells show original
Refractory anemia with ring sideroblasts (RARS), defined as refractory anemia above, but also including the presence of ringed sideroblasts comprising >= 15% of all nucleated cells in the bone marrow
Refractory anemia with excess blasts (RAEB) is a condition in which there are too many immature white blood cells (blasts) in the bone marrow and/or blood show original
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 1 month post-treatment
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 1 month post-treatment.
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Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Vorinostat will improve 3 primary outcomes and 5 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of Time from first treatment day until objective or symptomatic progression, assessed up to 8 years.

Progression-free survival
Assessed by Kaplan-Meier survival analysis and 95% confidence intervals will be calculated using Greenwood's formulae.
Overall survival
Assessed by Kaplan-Meier survival analysis and 95% confidence intervals will be calculated using Greenwood's formulae.
Frequency of leukemic transformation
Time to response
Time to leukemic transformation
Objective overall response proportion (complete response [CR] + CR with incomplete blood count + partial response) (Phase II)
Ninety-five percent confidence intervals will be estimated for the response proportion in all three treatment groups via binomial proportions.
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Patient Q & A Section

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

Can myelodysplastic syndromes be cured?

Although myelodysplastic syndromes cannot be cured, some of these rare diseases could be prevented, if patients can be screened for the mutations in the genes encoding the enzyme dihydrofolate reductase, or if the patients at risk from the disorder can be identified before they develop significant symptoms (as is the case for several rare myelodysplastic syndromes).

Anonymous Patient Answer

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

about 100,000 people are diagnosed with an MDS in the US each year. The number of people with MDS fluctuates significantly over time and is estimated at approximately 100,000 annually, a level of overdiagnosis not documented to date. These data suggest that the current number of cases of MDS and MDS-associated aneuploidy may be greater than previously thought.

Anonymous Patient Answer

What causes myelodysplastic syndromes?

The diagnosis is the result of specific pathological and cytogenetic findings and the finding of the presence of other conditions (i.e. congenital, infection or other chronic inflammatory disease) does not modify the diagnosis. Patients who have a history of myelodysplastic syndromes present in children are predominantly found to have refractory anaemias. However, in adults with myelodysplastic syndromes, they are predominantly found to have acute myeloid leukaemias in the absence of signs of infection or congenital disorder. A clear correlation was found between the number of abnormalities identified and the disease aggressiveness.

Anonymous Patient Answer

What are the signs of myelodysplastic syndromes?

The symptoms of myelodysplastic syndromes involve the bone marrow in the central nervous system, skin, skeletal, or visceral tissues. The symptoms can include fatigue, malaise, myelosuppression, and thrombocytopenia.

Anonymous Patient Answer

What is myelodysplastic syndromes?

MDS is a heterogeneous group of blood syndromes with diverse underlying pathophysiological mechanisms. It is characterised by ineffective and rapidly undergoing clonal cell development, with consequent accumulation of monoclonal population, resulting in monosomy for the chromosome of interest. MDS is often associated with other hematological and non-hematological abnormalities. If MDS is diagnosed at a younger age, its prognosis is good, and treatment is often initiated to tackle the hematological, immunological and/or metabolic disturbances associated with MDS.

Anonymous Patient Answer

What are common treatments for myelodysplastic syndromes?

Patients with the high-risk MDS subgroup have a poor prognosis. Common treatments include blood marrow transplantation and erythroblast differentiation therapy. New targeted drugs that can target abnormal proteins such as Bcr-abl are in clinical trials.

Anonymous Patient Answer

What does vorinostat usually treat?

Vorinostat is seldom used for treatment of hematological malignancies, despite widespread clinical evidence supporting the hypothesis that HDAC1 and 2 inhibition can overcome a number of impediments to effective treatments of AML. Thus, other HDAC inhibitors may be more suitable for this indication.

Anonymous Patient Answer

Has vorinostat proven to be more effective than a placebo?

Findings from a recent study, patients were randomized to vemurafenib or a placebo. Vorinostat did not appear to improve PFS, and a substantial percentage of patients discontinued vemurafenib early in the trial. Thus, vorinostat did not appear to be more effective than a placebo in the first, single-agent treatment of BRAF(V600E) MDS.

Anonymous Patient Answer

Have there been any new discoveries for treating myelodysplastic syndromes?

There have been few new developments regarding the use of cytotoxic chemotherapy and biologics for treatment of MDS over the past 12 years. Many of the patients with MDS have very low blood counts and require constant monitoring to diagnose and treat abnormalities like infections. It is still unclear how these treatments may work for MDS. Further studies need to be conducted in order to learn more about the treatment of MDS.

Anonymous Patient Answer

What are the chances of developing myelodysplastic syndromes?

The overall risk for MDS is approximately 30/100,000 person-years with the male being impacted more than the female. The risk for MDS after radiation radiation therapy has been found to be a hazard of about 1 in 10 000. However, not all patients who are previously irradiated will develop a secondary myelodysplastic syndrome. However, with age, it has been found that the chances of developing secondary myelodysplastic syndromes are higher for patients that are exposed to a higher dose of radiation.

Anonymous Patient Answer

How quickly does myelodysplastic syndromes spread?

In our setting myelodysplasia as well as MDS have a very fast progression and may be an early indication for an allogeneic stem cell transplant.

Anonymous Patient Answer

What is the latest research for myelodysplastic syndromes?

Significant research results were summarized for the year 2012. While several new therapeutic approaches to MDS had been established, no drug regimen has yet been approved by the FDA. Ongoing clinical trials and therapeutic developments will be needed to further decrease the number of patients affected by this disease.

Anonymous Patient Answer
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