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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01757535




Registration number
NCT01757535
Ethics application status
Date submitted
21/11/2012
Date registered
31/12/2012
Date last updated
28/08/2024

Titles & IDs
Public title
Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission
Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia in Complete Remission
Secondary ID [1] 0 0
2012-003457-28
Secondary ID [2] 0 0
CC-486-AML-001
Universal Trial Number (UTN)
Trial acronym
QUAZAR AML-001
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Leukemia, Myeloid, Acute 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Cancer 0 0 0 0
Any cancer

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Oral Azacitidine
Treatment: Drugs - Placebo

Experimental: Oral Azacitidine - 300 mg oral azacitidine on days 1 to 14 of each 28-day treatment cycle.

Placebo comparator: Placebo - Identically matching placebo tablets on days 1 to 14 of each 28-day treatment cycle.


Treatment: Drugs: Oral Azacitidine
300 mg oral azacitidine on days 1 to 14 of each 28-day treatment cycle.

Treatment: Drugs: Placebo
Identically matching placebo tablets on days 1 to 14 of each 28-day treatment cycle.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Kaplan-Meier (K-M) Estimate for Overall Survival (OS)
Timepoint [1] 0 0
Day 1 (randomization) up to data cut off date of 15 July 2019; median follow-up for OS estimated by the reverse K-M method was 41.2 months for all participants.
Secondary outcome [1] 0 0
Kaplan-Meier Estimate of Relapse Free Survival (RFS)
Timepoint [1] 0 0
From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months
Secondary outcome [2] 0 0
Kaplan-Meier Estimate of Time to Relapse
Timepoint [2] 0 0
Day 1 (randomization) to the date of the data cut off date of 15 July 2019; approximately 74 months
Secondary outcome [3] 0 0
Kaplan-Meier Estimates of Time to Discontinuation From Treatment
Timepoint [3] 0 0
From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months
Secondary outcome [4] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Timepoint [4] 0 0
Day 1 (randomization) to the data cut off date of 15 July 2019; the median treatment duration was 11.6 months (range: 0.5 to 74.3 months) for the oral aza arm and 5.7 months (range: 0.7 to 68.5 months) for the placebo arm.
Secondary outcome [5] 0 0
Mean Change in the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale V 4.0) Score From Baseline
Timepoint [5] 0 0
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
Secondary outcome [6] 0 0
Mean Change in the European Quality of Life-Five Dimensions-Three Levels (EQ-5D-3L) Score From Baseline
Timepoint [6] 0 0
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
Secondary outcome [7] 0 0
Percentage of Participants Experiencing a Clinically Meaningful Change (Improvement, No Change and Deterioration) in the FACIT-Fatigue Scale From Baseline
Timepoint [7] 0 0
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
Secondary outcome [8] 0 0
Percentage of Participants Experiencing a Clinically Meaningful Change (Improvement, No Change and Deterioration) in the EQ-5D-3L Scale From Baseline
Timepoint [8] 0 0
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
Secondary outcome [9] 0 0
Time to Definitive Clinically Meaningful Deterioration for = 2 Consecutive Visits as Measured Using the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale V 4.0)
Timepoint [9] 0 0
From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months
Secondary outcome [10] 0 0
Time to Definitive Clinically Meaningful Deterioration for = 2 Consecutive Visits as Measured Using the EQ-5D HRQoL Scale
Timepoint [10] 0 0
From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months
Secondary outcome [11] 0 0
Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Person Year
Timepoint [11] 0 0
Day 1 (randomization) to the date of the data cut off date of 15 July 2019; approximately 74 months
Secondary outcome [12] 0 0
Healthcare Resource Utilization (HRU): Number of Days Hospitalized Per Person-Year
Timepoint [12] 0 0
Day 1 (randomization) to the date of the data cut off date of 15 July 2019; approximately 74 months

Eligibility
Key inclusion criteria
Key

1. Male or female participants = 55 years of age
2. Newly diagnosed, histologically confirmed de novo acute myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or CMML (Chronic myelomonocytic leukemia)
3. First complete remission (CR)/ complete remission with incomplete blood count recovery (CRi) with induction therapy with intensive chemotherapy with or without consolidation therapy within 4 months (+/- 7 days of achieving CR or CRi)
4. Eastern Cooperative Oncology Group (ECOG) performance status - 0, 1, 2, 3

Key Inclusion Criteria in the Extended Phase of the study:

At the Investigator's discretion and with approval of the sponsor, participants meeting all of the following eligibility criteria are eligible to enter the extension phase:

1. All participants randomized into the oral azacitidine or placebo arm and are continuing in either the treatment phase or follow-up phase of the CC-486-AML-001 study;

* Participants randomized to oral azacitidine treatment arm and continuing in the treatment phase demonstrating clinical benefit as assessed by the investigator are eligible to receive oral azacitidine in the extension phase (EP);
* Participants randomized into placebo arm of the study will not receive oral azacitidine in the EP, but will be followed for survival in the EP;
* Participants currently in the follow-up phase will continue to be followed for survival in the EP;
2. Participants who have signed the informed consent for the EP of the study;
3. Participants who do not meet any of the criteria for study discontinuation

Key
Minimum age
55 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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
2. Prior bone marrow or stem cell transplantation
3. Have achieved CR/CRi following therapy with hypomethylating agents
4. Diagnosis of malignant disease within the previous 12 months
5. Proven central nervous system (CNS) leukemia

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/04/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/06/2024
Sample size
Target
Accrual to date
Final
472
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA
Recruitment hospital [1] 0 0
Local Institution - 510 - Wollongong
Recruitment hospital [2] 0 0
Local Institution - 509 - South Brisbane
Recruitment hospital [3] 0 0
Local Institution - 508 - Adelaide
Recruitment hospital [4] 0 0
Local Institution - 511 - Bedford Park
Recruitment hospital [5] 0 0
Local Institution - 504 - Woodville South
Recruitment hospital [6] 0 0
Local Institution - 503 - Heidelberg
Recruitment hospital [7] 0 0
Local Institution - 502 - Hobart
Recruitment hospital [8] 0 0
Local Institution - 507 - Liverpool
Recruitment hospital [9] 0 0
Local Institution - 500 - Melbourne
Recruitment hospital [10] 0 0
Local Institution - 505 - Perth
Recruitment hospital [11] 0 0
Local Institution - 512 - Perth
Recruitment hospital [12] 0 0
Local Institution - 506 - St Leonards
Recruitment hospital [13] 0 0
Local Institution - 501 - Woolloongabba
Recruitment postcode(s) [1] 0 0
2500 - Wollongong
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
SA 5000 - Adelaide
Recruitment postcode(s) [4] 0 0
5042 - Bedford Park
Recruitment postcode(s) [5] 0 0
5011 - Woodville South
Recruitment postcode(s) [6] 0 0
3084 - Heidelberg
Recruitment postcode(s) [7] 0 0
7000 - Hobart
Recruitment postcode(s) [8] 0 0
2170 - Liverpool
Recruitment postcode(s) [9] 0 0
3004 - Melbourne
Recruitment postcode(s) [10] 0 0
6000 - Perth
Recruitment postcode(s) [11] 0 0
2065 - St Leonards
Recruitment postcode(s) [12] 0 0
4102 - Woolloongabba
Recruitment outside Australia
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Sevilla
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Valencia
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Taiwan
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Beitou District, Taipei City
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Taiwan
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Ankara
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Istanbul
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Turkey
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Samsun
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United Kingdom
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Nottinghamshire
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Boston
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Brighton East Sussex
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Canterbury Kent
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London
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United Kingdom
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Maidstone
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Manchester
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United Kingdom
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Romford, Essex

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Celgene
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study enrolled 472 participants, aged 55 or older, with a diagnosis of de novo acute myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or chronic myelomonocytic leukemia (CMML), and who have achieved first complete remission (CR)/ complete remission with incomplete blood count recovery (CRi) following induction with or without consolidation chemotherapy.

The study is amended to include an extension phase (EP). The EP allows participants who are currently receiving oral azacitidine and who are demonstrating clinical benefit as assessed by the investigator, to continue receiving oral azacitidine after unblinding by sponsor until the participant meets the criteria for study discontinuation or until oral azacitidine becomes commercially available and reimbursed. In addition, all participants in the placebo arm and participants who had been discontinued from the treatment phase (irrespective of randomization arm) and continuing in the follow-up phase will be followed for survival in the EP.
Trial website
https://clinicaltrials.gov/study/NCT01757535
Trial related presentations / publications
Roboz GJ, Montesinos P, Selleslag D, Wei A, Jang JH, Falantes J, Voso MT, Sayar H, Porkka K, Marlton P, Almeida A, Mohan S, Ravandi F, Garcia-Manero G, Skikne B, Kantarjian H. Design of the randomized, Phase III, QUAZAR AML Maintenance trial of CC-486 (oral azacitidine) maintenance therapy in acute myeloid leukemia. Future Oncol. 2016 Feb;12(3):293-302. doi: 10.2217/fon.15.326. Epub 2016 Jan 19.
Dohner H, Wei AH, Roboz GJ, Montesinos P, Thol FR, Ravandi F, Dombret H, Porkka K, Sandhu I, Skikne B, See WL, Ugidos M, Risueno A, Chan ET, Thakurta A, Beach CL, Lopes de Menezes D. Prognostic impact of NPM1 and FLT3 mutations in patients with AML in first remission treated with oral azacitidine. Blood. 2022 Oct 13;140(15):1674-1685. doi: 10.1182/blood.2022016293.
Zhu J, Wu Q, Wang J, Niu T. Cost-effectiveness analysis of azacitidine maintenance therapy in patients with acute myeloid leukemia. Expert Rev Hematol. 2022 Apr;15(4):375-382. doi: 10.1080/17474086.2022.2061456. Epub 2022 May 11.
Roboz GJ, Ravandi F, Wei AH, Dombret H, Thol F, Voso MT, Schuh AC, Porkka K, La Torre I, Skikne B, Zhong J, Beach CL, Risueno A, Menezes DL, Ossenkoppele G, Dohner H. Oral azacitidine prolongs survival of patients with AML in remission independently of measurable residual disease status. Blood. 2022 Apr 7;139(14):2145-2155. doi: 10.1182/blood.2021013404.
Ravandi F, Roboz GJ, Wei AH, Dohner H, Pocock C, Selleslag D, Montesinos P, Sayar H, Musso M, Figuera-Alvarez A, Safah H, Tse W, Sohn SK, Hiwase D, Chevassut T, Pierdomenico F, La Torre I, Skikne B, Bailey R, Zhong J, Beach CL, Dombret H. Management of adverse events in patients with acute myeloid leukemia in remission receiving oral azacitidine: experience from the phase 3 randomized QUAZAR AML-001 trial. J Hematol Oncol. 2021 Aug 28;14(1):133. doi: 10.1186/s13045-021-01142-x.
Wei AH, Dohner H, Pocock C, Montesinos P, Afanasyev B, Dombret H, Ravandi F, Sayar H, Jang JH, Porkka K, Selleslag D, Sandhu I, Turgut M, Giai V, Ofran Y, Kizil Cakar M, Botelho de Sousa A, Rybka J, Frairia C, Borin L, Beltrami G, Cermak J, Ossenkoppele GJ, La Torre I, Skikne B, Kumar K, Dong Q, Beach CL, Roboz GJ; QUAZAR AML-001 Trial Investigators. Oral Azacitidine Maintenance Therapy for Acute Myeloid Leukemia in First Remission. N Engl J Med. 2020 Dec 24;383(26):2526-2537. doi: 10.1056/NEJMoa2004444.
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Bristol-Myers Squibb
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Bristol-Myers Squibb
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Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01757535