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Anti-metabolites

Oral Azacitidine for Myelodysplastic Syndrome

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
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 january 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Awards & highlights

Study Summary

This trial is testing a new drug to treat anemia and low blood platelet counts in people with a certain type of bone marrow disease.

Who is the study for?
Adults with low-risk myelodysplastic syndrome (MDS) who need regular red blood cell transfusions and have low platelet counts. Participants should be able to perform daily activities with minimal assistance (ECOG status 0-2), consent to study procedures, and follow pregnancy precautions. Excluded are those with certain MDS subtypes, previous specific treatments, eligibility for stem cell transplant, significant other diseases or conditions.Check my eligibility
What is being tested?
The trial is testing if oral Azacitidine combined with Best Supportive Care (BSC) is more effective than a placebo plus BSC in improving anemia and thrombocytopenia in patients with lower risk MDS. The goal is to see if this treatment can reduce the need for blood transfusions.See study design
What are the potential side effects?
Oral Azacitidine may cause gastrointestinal symptoms like nausea or vomiting, injection site reactions since it's usually given as a shot even though it's oral in this trial, fatigue, and potential lowering of blood cell counts leading to increased infection risk.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 january 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
This trial's timeline: 3 weeks for screening, Varies for treatment, and from the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 january 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days
Secondary outcome measures
Blood Transfusion
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days
Duration of RBC Transfusion Reduction for Participants Who Achieved RBC Transfusion Reduction of at Least 4 Units of RBCs for at Least 8 Weeks
+40 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%
Weight decreased
9%
Leukopenia
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%
Gastroenteritis
1%
Abdominal pain upper
1%
Escherichia sepsis
1%
Corona virus infection
1%
Prerenal failure
1%
Myocardial infarction
1%
Gastritis
1%
Epididymitis
1%
Febrile infection
1%
Pancytopenia
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

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Oral AzacitidineExperimental Treatment2 Interventions
Arm 1: Oral azacitidine tablets 300 mg daily (QD) + best supportive care (BSC) on days 1 through 21 of each 28-day treatment cycle.
Group II: PlaceboPlacebo Group2 Interventions
Arm 2: Identically matching placebo tablets plus best supportive care on days 1 to 21 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
~480

Find a Location

Who is running the clinical trial?

CelgeneLead Sponsor
643 Previous Clinical Trials
130,361 Total Patients Enrolled
Barry Skikne, MDStudy DirectorCelgene Corporation
2 Previous Clinical Trials
526 Total Patients Enrolled
Bristol-Myers SquibbStudy DirectorBristol-Myers Squibb
1,520 Previous Clinical Trials
3,371,997 Total Patients Enrolled

Media Library

Oral Azacitidine (Anti-metabolites) Clinical Trial Eligibility Overview. Trial Name: NCT01566695 — Phase 3
Myelodysplastic Syndrome Research Study Groups: Oral Azacitidine, Placebo
Myelodysplastic Syndrome Clinical Trial 2023: Oral Azacitidine Highlights & Side Effects. Trial Name: NCT01566695 — Phase 3
Oral Azacitidine (Anti-metabolites) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01566695 — Phase 3
~18 spots leftby May 2025
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