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Oral Azacitidine for Acute Myeloid Leukemia (QUAZAR AML-001 Trial)

Phase 3
Waitlist Available
Research Sponsored by Celgene
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Eastern Cooperative Oncology Group (ECOG) performance status - 0, 1, 2, 3
Male or female participants ≥ 55 years of age
Must not have
AML with inversion (inv)(16), translocation = t(8;21), t(16;16), t(15;17), or t(9;22) or molecular evidence of such translocations
Have achieved CR/CRi following therapy with hypomethylating agents
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to cycle 2, day 1 (c2d1), c3d1, c4d1, c5d1, c6d1, c7d1, c8d1, c9d1c3, c10d1, c11d1, c12d1, c13d1, c14d1, c15d1, c15d1, c16d1, c17d1, c18d1, c19d1, c20d1, c21d1, c22d1, c23d1, c24d1, c25d1 and continued on day 1 at each cycle through c33d1
Awards & highlights
Pivotal Trial

Summary

This trial is studying oral azacitidine to see how well it works in treating patients with acute myeloid leukemia or myelodysplastic syndromes.

Who is the study for?
This trial is for people aged 55 or older with a type of blood cancer called acute myeloid leukemia (AML), who have achieved remission after initial chemotherapy. They must be able to perform daily activities on their own or with some help (ECOG status 0-3). Those with certain genetic mutations in their leukemia, recent other cancers, CNS leukemia, or previous bone marrow transplants can't join.
What is being tested?
The study tests if taking oral Azacitidine helps maintain remission in AML patients compared to a placebo. Participants are randomly assigned to either the drug or placebo group and may continue treatment if beneficial. The extension phase tracks survival rates even after unblinding when the drug becomes commercially available.
What are the potential side effects?
Oral Azacitidine might cause nausea, vomiting, diarrhea, constipation, fatigue and weakness. There's also a risk of lowered blood cell counts leading to increased infection risk, bleeding problems or anemia. Side effects vary from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can care for myself but may not be able to do heavy physical work.
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I am 55 years old or older.
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I have been newly diagnosed with AML or AML that developed from a previous blood disorder.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My AML has specific genetic changes.
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I achieved complete remission after treatment with drugs that affect DNA.
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My leukemia has spread to my brain or spinal cord.
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I have had a bone marrow or stem cell transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to cycle 2, day 1 (c2d1), c3d1, c4d1, c5d1, c6d1, c7d1, c8d1, c9d1c3, c10d1, c11d1, c12d1, c13d1, c14d1, c15d1, c15d1, c16d1, c17d1, c18d1, c19d1, c20d1, c21d1, c22d1, c23d1, c24d1, c25d1 and continued on day 1 at each cycle through c33d1
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to cycle 2, day 1 (c2d1), c3d1, c4d1, c5d1, c6d1, c7d1, c8d1, c9d1c3, c10d1, c11d1, c12d1, c13d1, c14d1, c15d1, c15d1, c16d1, c17d1, c18d1, c19d1, c20d1, c21d1, c22d1, c23d1, c24d1, c25d1 and continued on day 1 at each cycle through c33d1 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Kaplan-Meier (K-M) Estimate for Overall Survival (OS)
Secondary study objectives
Healthcare Resource Utilization (HRU): Number of Days Hospitalized Per Person-Year
Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Person Year
Kaplan-Meier Estimate of Relapse Free Survival (RFS)
+9 more

Side effects data

From 2023 Phase 3 trial • 216 Patients • NCT01566695
76%
Nausea
68%
Diarrhoea
63%
Vomiting
49%
Neutropenia
47%
Constipation
28%
Pyrexia
27%
Thrombocytopenia
27%
Febrile neutropenia
27%
Oedema peripheral
26%
Epistaxis
25%
Decreased appetite
23%
Asthenia
21%
Fatigue
20%
Petechiae
18%
Anaemia
15%
Cough
14%
Contusion
13%
Abdominal pain
12%
Dyspnoea
12%
Back pain
11%
Urinary tract infection
11%
Hypokalaemia
9%
Leukopenia
9%
Weight decreased
9%
Insomnia
9%
Pneumonia
9%
Mouth haemorrhage
9%
Hypomagnesaemia
9%
Haematoma
8%
Anxiety
8%
Alanine aminotransferase increased
8%
Arthralgia
7%
Sepsis
7%
Dizziness
7%
Gingival bleeding
7%
Upper respiratory tract infection
7%
Pain in extremity
6%
Depression
6%
Confusional state
6%
Septic shock
6%
Gastrooesophageal reflux disease
6%
Cellulitis
6%
Oral herpes
6%
Serum ferritin increased
6%
Hyperglycaemia
6%
Iron overload
6%
Ecchymosis
6%
Hypotension
5%
Neutropenic sepsis
4%
Fall
3%
Lung infection
3%
General physical health deterioration
3%
Cardiac failure congestive
2%
Tachyarrhythmia
2%
Bone marrow failure
2%
Cardiac failure
2%
Multiple organ dysfunction syndrome
2%
Cholecystitis
2%
Hyperbilirubinaemia
2%
Atypical pneumonia
2%
Bronchopulmonary aspergillosis
2%
Subdural haematoma
2%
Haemorrhage intracranial
2%
Acute kidney injury
2%
Renal failure
1%
Gastritis
1%
Febrile infection
1%
Epididymitis
1%
Gastroenteritis
1%
Prerenal failure
1%
Pancytopenia
1%
Myocardial infarction
1%
Corona virus infection
1%
Escherichia sepsis
1%
Abdominal pain upper
1%
Renal colic
1%
Chronic kidney disease
1%
Lethargy
1%
Groin abscess
1%
Lower respiratory tract infection
1%
Device related infection
1%
Influenza
1%
Klebsiella infection
1%
Haemolytic anaemia
1%
Haemorrhagic anaemia
1%
Acute myocardial infarction
1%
Angina unstable
1%
Atrial fibrillation
1%
Gastrointestinal haemorrhage
1%
Intestinal obstruction
1%
Intestinal perforation
1%
Neutropenic colitis
1%
Oesophageal achalasia
1%
Oral mucosal blistering
1%
Rectal haemorrhage
1%
Gait disturbance
1%
Hypothermia
1%
Abscess limb
1%
Arteriovenous fistula site infection
1%
Klebsiella sepsis
1%
Meningitis
1%
Meningitis bacterial
1%
Myringitis
1%
Pneumonia fungal
1%
Pneumonia pneumococcal
1%
Pseudomonal sepsis
1%
Pulmonary mycosis
1%
Respiratory tract infection
1%
Skin infection
1%
Staphylococcal infection
1%
Urinary tract infection bacterial
1%
Viral sepsis
1%
Periorbital haematoma
1%
Febrile nonhaemolytic transfusion reaction
1%
Head injury
1%
Hip fracture
1%
Subdural haemorrhage
1%
Upper limb fracture
1%
Dehydration
1%
Diabetes mellitus inadequate control
1%
Diabetic metabolic decompensation
1%
Hyperkalaemia
1%
Hypoglycaemia
1%
Muscular weakness
1%
Polychondritis
1%
Acute myeloid leukaemia
1%
Bone neoplasm
1%
Bowen's disease
1%
Colon adenoma
1%
Mantle cell lymphoma recurrent
1%
Spinal cord neoplasm
1%
Central nervous system lesion
1%
Transient ischaemic attack
1%
Epilepsy
1%
Generalised tonic-clonic seizure
1%
Acute respiratory distress syndrome
1%
Pleural effusion
1%
Pleurisy
1%
Pneumonia aspiration
1%
Pulmonary embolism
1%
Respiratory failure
1%
Hypersensitivity vasculitis
1%
Rash
1%
Rash generalised
1%
Shock haemorrhagic
1%
Cardiogenic shock
1%
Intra-abdominal haemorrhage
1%
Status epilepticus
1%
Syncope
1%
Urinary retention
1%
Prostatitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Oral Azacitidine Plus Best Supportive Care
Placebo Plus Best Supportive Care

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Oral AzacitidineExperimental Treatment1 Intervention
300 mg oral azacitidine on days 1 to 14 of each 28-day treatment cycle.
Group II: PlaceboPlacebo Group1 Intervention
Identically matching placebo tablets on days 1 to 14 of each 28-day treatment cycle.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Oral Azacitidine
2013
Completed Phase 3
~950

Find a Location

Who is running the clinical trial?

CelgeneLead Sponsor
645 Previous Clinical Trials
129,712 Total Patients Enrolled
Barry Skikne, MDStudy DirectorCelgene Corporation
2 Previous Clinical Trials
270 Total Patients Enrolled
Bristol-Myers SquibbStudy DirectorBristol-Myers Squibb
1,579 Previous Clinical Trials
3,387,149 Total Patients Enrolled

Media Library

Acute Myeloid Leukemia Research Study Groups: Oral Azacitidine, Placebo
Acute Myeloid Leukemia Clinical Trial 2023: Oral Azacitidine Highlights & Side Effects. Trial Name: NCT01757535 — Phase 3
~38 spots leftby Dec 2025
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