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Nucleoside Metabolic Inhibitor

Vorinostat + Azacitidine for Myelodysplastic Syndrome / Acute Myeloid Leukemia

Phase 1 & 2

Waitlist Available

Led By Lewis R Silverman

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Additional criteria for AML patients include stable disease, no recent use of corticosteroids, interferon, retinoids, hematopoietic growth factors, azacitidine, decitabine, vorinostat, or investigational agents

Patients must have a diagnosis of either MDS according to FAB and IPSS criteria, or a diagnosis of AML according to FAB or WHO criteria

Must not have

Patients with recent chemotherapy or radiotherapy, evidence of another malignancy within the past 3 years, or receiving other investigational agents

Patients with CNS involvement, allergic reactions to study drugs, pregnancy, serious medical or psychiatric illnesses, uncontrolled congestive heart failure, high myeloblast percentage, prior treatment with specific growth factors, positive HIV serology, active infections, or advanced hepatic tumors

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 month post-treatment

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying vorinostat and azacitidine to treat patients with myelodysplastic syndromes or acute myeloid leukemia.

Who is the study for?

This trial is for adults with certain types of blood disorders, including various forms of leukemia and myelodysplastic syndromes. Participants should have specific disease characteristics, not be on certain medications recently, and must have a life expectancy over 2 months. They need to be in relatively good health otherwise (ECOG <=2), with normal organ function tests. Pregnant women or those who've had recent cancer treatments are excluded.

What is being tested?

The study is testing the combination of two drugs: Vorinostat and Azacitidine. It aims to find the safest doses and see how effective they are against different blood disorders like acute myeloid leukemia and myelodysplastic syndromes by blocking enzymes that allow abnormal cells to grow or by directly killing them.

What are the potential side effects?

Potential side effects include allergic reactions similar to other compounds related to Vorinostat or Azacitidine, as well as risks associated with chemotherapy such as fatigue, nausea, infection risk increase due to low white blood cell counts, bleeding from low platelets, liver issues indicated by elevated bilirubin levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have AML and haven't used certain medications recently.

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I have been diagnosed with MDS or AML according to specific medical criteria.

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My AML is new or evolved from MDS and is not likely to respond well to treatment.

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I have a type of anemia related to my bone marrow disorder.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't had chemotherapy, radiotherapy, or been diagnosed with another cancer in the last 3 years.

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I do not have brain involvement, allergies to study drugs, serious illnesses, heart failure, high myeloblasts, HIV, infections, or liver tumors.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 month post-treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 month post-treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 month post-treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Distribution of toxicities in the 12th treatment arm (Phase II)

Incidence of toxicities of vorinostat in combination with azacitidine graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version (v)5.0 (Phase I)

Objective overall response proportion (complete response [CR] + CR with incomplete blood count + partial response) (Phase II)

Secondary study objectives

Overall survival

Progression-free survival

Side effects data

From 2011 Phase 3 trial • 661 Patients • NCT00128102

57%

Nausea

47%

Fatigue

43%

Diarrhoea

40%

Vomiting

40%

Decreased appetite

29%

Dyspnoea

24%

Constipation

20%

Weight decreased

18%

Tumour pain

18%

Cough

15%

Pleural mesothelioma malignant advanced

14%

Anaemia

12%

Pyrexia

Insomnia

Dry mouth

Blood creatinine increased

Abdominal pain

Back pain

Dysgeusia

Oedema peripheral

Dizziness

Thrombocytopenia

Headache

C-reactive protein increased

Dehydration

Musculoskeletal pain

Malaise

Pneumonia

Anxiety

Rash

Atrial fibrillation

Accidental overdose

Pleural effusion

Bladder cancer

Overdose

Sepsis

Pneumothorax

Confusional state

General physical health deterioration

Non-cardiac chest pain

Pericarditis

Disseminated intravascular coagulation

Death

Ascites

Dysphagia

Pulmonary embolism

Deep vein thrombosis

100%

80%

60%

40%

20%

Study treatment Arm

Vorinostat

Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (azacitidine, vorinostat)Experimental Treatment4 Interventions

Patients receive azacitidine SC QD on days 1-7 and vorinostat PO 2-3 times daily on days 3-5, 3-9, or 3-16. Treatment repeats every 28 days for at least 4 courses in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vorinostat

2014

Completed Phase 3

~1600

Azacitidine

2012

Completed Phase 3

~1440