Leukemia, Myeloid, AcutePhase 3QUAZAR AML-001NCT01757535
Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission
A Phase 3 study testing Oral Azacitidine in people with Leukemia, Myeloid, Acute, run by Celgene at 223 sites in 23 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- Oral Azacitidine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 472 participants
- Where
- 223 sitesin 23 countries
- Study dates
- 24 Apr 2013to 18 Jun 2024
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female participants ≥ 55 years of age
- Newly diagnosed, histologically confirmed de novo acute myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or CMML (Chronic myelomonocytic leukemia)
- 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)
- Eastern Cooperative Oncology Group (ECOG) performance status - 0, 1, 2, 3
- 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:
- 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;
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- 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;
- Participants who have signed the informed consent for the EP of the study;
- Participants who do not meet any of the criteria for study discontinuation
Who cannot join
- 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
- Prior bone marrow or stem cell transplantation
- Have achieved CR/CRi following therapy with hypomethylating agents
- Diagnosis of malignant disease within the previous 12 months
- Proven central nervous system (CNS) leukemia
Trial locations and contacts
223 sites in 23 countries.
223 sites in 23 countries
United States60 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Providence St Joseph Medical Center Cancer Center
Burbank, California, 91505
Northwestern University Medical Center
Chicago, Illinois, 60611
Local Institution - 016
Cleveland, Ohio, 44106
Local Institution - 041
Dallas, Texas, 75390-9068
Rocky Mountain Cancer Center
Denver, Colorado, 80218-1210
City Of Hope
Duarte, California, 91010-301
Duke University Medical Center
Durham, North Carolina, 27705
Local Institution - 034
Fort Sam Houston, Texas, 78234
University of California San Francisco Fresno Campus
Fresno, California, 93701
Local Institution - 046
Gainesville, Florida, 32610
Greenville Hospital System
Greenville, South Carolina, 29605
John Theurer Cancer Center at Hackensack University Medical Center
Hackensack, New Jersey, 07601
Local Institution - 001
Houston, Texas, 77030
Local Institution - 013
Indianapolis, Indiana, 46202-528
Franciscan St. Francis Health
Indianapolis, Indiana, 46237
Local Institution - 023
Kansas City, Missouri, 64128
Lancaster General Hospital
Lancaster, Pennsylvania, 17604
University of Southern California Norris Cancer Center
Los Angeles, California, 90033
Local Institution - 006
Los Angeles, California, 90095-6956
Local Institution - 049
Louisville, Kentucky, 40202
Local Institution - 058
Louisville, Kentucky, 40207
Loyola University Chicago
Maywood, Illinois, 60153
Mount Sinai Comprehensive Cancer Center
Miami Beach, Florida, 33140
Froedtert Hospital BMT Medical College of Wisconsin
Milwaukee, Wisconsin, 53226-3522
Winthrop University Hospital
Mineola, New York, 11501-3893
Local Institution - 011
Nashville, Tennessee, 37203
Local Institution - 007
Nashville, Tennessee, 37232-5505
Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Local Institution - 047
New Orleans, Louisiana, 70112
Ochsner Medical Center - Jefferson Highway
New Orleans, Louisiana, 70121-2483
Local Institution - 009
New York, New York, 10029
Columbia University Irving Medical Center
New York, New York, 10032
Local Institution - 002
New York, New York, 10065
Cancer Care and Hematology Specialists of Chicagoland, P.C. - Niles, IL
Niles, Illinois, 60714
University of Oklahoma Peggy and Charles Stephenson Cancer Center
Oklahoma City, Oklahoma, 73104
Local Institution - 057
Omaha, Nebraska, 68198-7680
Local Institution - 050
Orange, California, 92868
Local Institution - 044
Orlando, Florida, 32806
Arizona Oncology Associates, P.C.
Phoenix, Arizona, 85016
UPMC Cancer Pavillion
Pittsburgh, Pennsylvania, 15232
Kaiser Permanente Northwest Oncology Hematology
Portland, Oregon, 97227
Local Institution - 035
Richmond, Virginia, 23298-0037
Local Institution - 037
Rochester, Minnesota, 55905
Local Institution - 004
Rochester, New York, 14642
Cancer Care Centers of South Texas - Loop
San Antonio, Texas, 78217
Local Institution - 039
San Antonio, Texas, 78229
Sharp Memorial Hospital
San Diego, California, 92123
Swedish Cancer Inst
Seattle, Washington, 98104
Local Institution - 010
Southington, Connecticut, 06489
Washington University School Of Medicine
St Louis, Missouri, 63110
Stanford Cancer Center
Stanford, California, 94305
Local Institution - 014
Valhalla, New York, 10595
George Washington University Cancer Center
Washington D.C., District of Columbia, 20037
Local Institution - 003
Westwood, Kansas, 66205
Innovative Clinical Research Institute
Whittier, California, 90603
Local Institution - 025
Winston-Salem, North Carolina, 27157
Local Institution - 015
Worcester, Massachusetts, 01655
Yakima Valley Memorial Hospital/ North Star Lodge
Yakima, Washington, 98902
Italy26 sites
Local Institution - 701
Alessandria, 15121
Local Institution - 721
Bari, 70124
Local Institution - 720
Bologna, 40138
Local Institution - 710
Cagliari, O9126
Local Institution - 702
Cremona, 26100
Local Institution - 708
Florence, 50129
Local Institution - 712
Genova, 16132
Local Institution - 716
Lecce, 73100
Local Institution - 706
Milan, 20122
Local Institution - 726
Milan, 20162
Local Institution - 704
Monza, 20900
Local Institution - 717
Naples, 80131
Local Institution - 725
Naples, 80131
Local Institution - 705
Orbassano (TO), 10043
Local Institution - 703
Palermo, 90146
Local Institution - 719
Palermo, 90146
Local Institution - 724
Pesaro, 31122
Local Institution - 700
Reggio Calabria, 89100
Local Institution - 709
Roma, 00161
Local Institution - 714
Roma, 00168
Local Institution - 723
Roma, 00189
Local Institution - 722
Rome, 133
Local Institution - 715
Torino, 10126
Local Institution - 718
Torino, 10126
Local Institution - 711
Udine, 33100
Local Institution - 707
Varese, 21100
France17 sites
Local Institution - 456
Amiens, 80054
Local Institution - 465
Argenteuil, 95100
Local Institution - 457
Bobigny, 93009
Local Institution - 462
Boulognes Sur Mer, 62200
Local Institution - 460
Clamart, 92141
Local Institution - 452
Créteil, 94010
Local Institution - 458
Le Chesnay, 78157
Local Institution - 453
Lille, 59037
Local Institution - 461
Limoges, 87042
Local Institution - 450
Lyon, 69437
Local Institution - 463
Paris, 75015
Local Institution - 454
Paris, 75475
Local Institution - 800
Paris, 75475
Local Institution - 464
Pontoise, 95301
Local Institution - 455
Rouen, 76038
Local Institution - 459
Saint-Cloud, 92210
Local Institution - 451
Villejuif, 94805
Germany17 sites
Local Institution - 413
Berlin, 12203
Local Institution - 410
Bonn, 53127
Local Institution - 400
Dresden, Saxony, 01307
Local Institution - 406
Düsseldorf, 40225
Local Institution - 415
Erlangen, 91054
Local Institution - 404
Frankfurt am Main, 65929
Local Institution - 412
Goch, 47574
Local Institution - 405
Hanover, 30625
Local Institution - 408
Heilbronn, 74078
Local Institution - 414
Jena, 07740
Local Institution - 403
Keil, 24105
Local Institution - 402
Mannheim, 68167
Local Institution - 409
München, 80804
Local Institution - 411
München, 81675
Local Institution - 407
Oldenburg, 26133
Local Institution - 416
Schweiler, 52249
Local Institution - 401
Ulm, 89081
Spain14 sites
Local Institution - 867
A Coruña, 15006
Local Institution - 869
Badalona (Barcelona), 8916
Local Institution - 871
Barcelona, 08025
Local Institution - 870
Barcelona, 08035
Local Institution - 873
Cáceres, 10003
Local Institution - 863
Córdoba, 14004
Local Institution - 868
Madrid, 28006
Local Institution - 866
Madrid, 28007
Local Institution - 865
Madrid, 28040
Local Institution - 864
Oviedo, 33006
Local Institution - 872
Palma de Mallorca, Balearic Islands, 07198
Local Institution - 861
Salamanca, 37007
Local Institution - 862
Seville, 41013
Local Institution - 860
Valencia, 46026
Australia13 sites
Local Institution - 508
Adelaide, South Australia, SA 5000
Local Institution - 511
Bedford Park, South Australia, 5042
Local Institution - 503
Heidelberg, 3084
Local Institution - 502
Hobart, 7000
Local Institution - 507
Liverpool, 2170
Local Institution - 500
Melbourne, 3004
Local Institution - 505
Perth, 6000
Local Institution - 512
Perth, 6000
Local Institution - 509
South Brisbane, Queensland, 4101
Local Institution - 506
St Leonards, 2065
Local Institution - 510
Wollongong, New South Wales, 2500
Local Institution - 504
Woodville South, South Australia, 5011
Local Institution - 501
Woolloongabba, 4102
United Kingdom10 sites
Local Institution - 907
Boston, PE21 9QS
Local Institution - 903
Brighton East Sussex, BN2 5BE
Local Institution - 902
Canterbury Kent, CT1 3NG
Local Institution - 905
London, NW1 2PG
Local Institution - 901
London, SE5 9RS
Local Institution - 908
London, W12 0HS
Local Institution - 909
Maidstone, ME16 9QQ
Local Institution - 900
Manchester, M20 4BX
Local Institution - 904
Nottingham, Nottinghamshire, NG5 1PB
Local Institution - 906
Romford, Essex, RM7 0AG
Canada8 sites
Local Institution - 605
Edmonton, Alberta, T6G 2B7
Local Institution - 604
Halifax, Nova Scotia, B3H 2Y9
Local Institution - 608
Montreal, Quebec, H2W 1S6
Local Institution - 602
Montreal, Quebec, H4J 1C5
Local Institution - 601
Saint John, New Brunswick, E2L 4L2
Local Institution - 603
St. John's, Newfoundland and Labrador, A1B3V6
Local Institution - 607
Toronto, Ontario, M5G 2M9
Local Institution - 600
Winnipeg, Manitoba, R3E OV9
South Korea6 sites
Local Institution - 535
Busan, 49241
Local Institution - 533
Daegu, 700-721
Local Institution - 536
Seoul, 120-752
Local Institution - 530
Seoul, 135-710
Local Institution - 531
Seoul, 137-701
Local Institution - 532
Seoul, 138-736
Austria5 sites
Local Institution - 271
Graz, 73013
Local Institution - 270
Salzburg, 5020
Local Institution - 274
Vienna, 1130
Local Institution - 272
Vienna, 1140
Local Institution - 273
Vienna, 1190
Brazil5 sites
Local Institution - 233
Curitiba, Paraná, 81520-060
Local Institution - 231
Porto Alegre, Rio Grande do Sul, 90035-903
Local Institution - 232
Rio de Janeiro, 20230-130
Local Institution - 234
São Paulo, 01308-050
Local Institution - 230
São Paulo, 05651-901
Poland5 sites
Local Institution - 824
Bydgoszcz, 85-168
Local Institution - 820
Gdansk, 80-211
Local Institution - 822
Lodz, 93-510
Local Institution - 821
Warsaw, 02-776
Local Institution - 823
Wroclaw, 50-367
Portugal5 sites
Local Institution - 841
Coimbra, 4200-072
Local Institution - 840
Lisbon, 1099-023
Local Institution - 843
Lisbon, 1150-314
Local Institution - 842
Porto, 4200-072
Local Institution - 844
Porto, 4200
Taiwan5 sites
Local Institution - 599
Beitou District, Taipei City, 11217
Local Institution - 595
Niaosong District Kaohsiung City, 83301
Local Institution - 596
Taichung, Northern Dist., 404
Local Institution - 597
Tainan, Taiana, 704
Local Institution - 598
Taipei, Zhongzheng Dist., 10002
Israel4 sites
Local Institution - 381
Beersheba, 84101
Local Institution - 380
Haifa, 35254
Local Institution - 383
Jerusalem, 91120
Local Institution - 382
Petah Tikva, 49100
Russia4 sites
Local Institution - 971
Moscow, 125101
Local Institution - 970
Nizhny Novgorod, 603005
Local Institution - 972
Saint Petersburg, 196022
Local Institution - 973
Saint Petersburg, 197341
Turkey (Türkiye)4 sites
Local Institution - 653
Ankara, 06100
Local Institution - 650
Ankara, 06200
Local Institution - 651
Istanbul, 34662
Local Institution - 652
Samsun, 55139
Belgium3 sites
Local Institution - 300
Bruges, 8000
Local Institution - 301
Charleroi, 6000
Local Institution - 302
Mons, 7000
Czechia3 sites
Local Institution - 320
Brno, South Moravian, 625 00
Local Institution - 321
Prague, 128 08
Local Institution - 322
Prague, 128 20
Finland3 sites
Local Institution - 361
Helsinki, 290
Local Institution - 362
Tampere, 33521
Local Institution - 360
Turku, 20521
Mexico3 sites
Local Institution - 252
Huixquilucan de Degollado, 52763
Local Institution - 251
México, 14080
Local Institution - 250
Monterrey, 64460
Ireland2 sites
Local Institution - 950
Dublin, 24
Local Institution - 951
Galway, ST46QG
About this study
Official 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
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.
Read the detailed description
This is an international, multicenter, placebo-controlled, Phase 3 study with a double-blind, randomized, parallel-group design in subjects with de novo AML or AML secondary to prior diagnosis of myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) aged ≥ 55 years, who are in first CR/CRi following induction therapy with or without consolidation chemotherapy. The study consists of 3 phases; the pre-randomization phase (screening phase), the treatment phase, and the follow-up phase.
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 they meet 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.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- Oral Azacitidine, Placebo
- Keywords
- Maintenance therapy, AML, Acute Myeloid Leukemia, Oral Azacitidine, Best supportive care, Complete remission
- Medical subject headings
- Leukemia, Myeloid, Acute, Pathologic Complete Response
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oral AzacitidineDrug
Also called CC-486; Onureg®
300 mg oral azacitidine on days 1 to 14 of each 28-day treatment cycle.
PlaceboDrug
Identically matching placebo tablets on days 1 to 14 of each 28-day treatment cycle.
| Study group | Role | What they receive |
|---|---|---|
| Oral Azacitidine | Experimental | 300 mg oral azacitidine on days 1 to 14 of each 28-day treatment cycle. Receives: Drug: Oral Azacitidine |
| Placebo | Placebo comparator | Identically matching placebo tablets on days 1 to 14 of each 28-day treatment cycle. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Kaplan-Meier (K-M) Estimate for Overall Survival (OS)
Overall survival was defined as time from randomization to death from any cause; participants surviving at the end of the follow-up period, or who withdraw consent, or who were lost to follow up were censored at the date last known alive.
Measured: 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 outcomes (9)
Kaplan-Meier Estimate of Relapse Free Survival (RFS)
RFS was defined as the time from the date of randomization to the date of documented relapse or death from any cause, whichever occurred first. Participants who were still alive without documented relapse, or who were lost to follow-up or withdrew consent without documented relapse, were censored at the date of their last bone marrow assessment, prior to receiving any other therapy for AML. Documented relapse was defined as the earliest date of the following: • ≥ 5% bone marrow blasts (myeloblasts) from Central Pathology report, or • appearance of > 0% blasts in the peripheral blood with a later bone marrow confirmation (bone marrow blast [myeloblasts] ≥ 5%) within 100 days, or • at least 2 peripheral blasts ≥ 5% within 30 days.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Kaplan-Meier Estimate of Time to Relapse
Time to relapse was defined as the interval (in months) from the date of randomization to the date of documented relapse. Estimates of relapse rate were based on the cumulative incidence function from a competing risk analysis with death as a competing risk for relapse from complete remission (CR)/ complete remission with incomplete blood count recovery (CRi). Documented relapse was defined as, the earliest date of the following: • ≥ 5% bone marrow blasts (myeloblasts) from Central Pathology report, or • appearance of > 0% blasts in the peripheral blood with a later bone marrow confirmation (bone marrow blast [myeloblasts] ≥ 5%) within 100 days, or • at least 2 peripheral blasts ≥ 5% within 30 days.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Kaplan-Meier Estimates of Time to Discontinuation From Treatment
Time to discontinuation from treatment was assessed and defined as the interval from the date of randomization to the date of discontinuation from study drug. Participants who were receiving treatment at the time of study closure were censored at the date of last visit. Estimates of relapse rate were based on the cumulative incidence function from a competing risk analysis with death as a competing risk for relapse from CR/ CRi.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
TEAEs include AEs that started between first dose date and 28 days after the last dose of study drug. A serious adverse event (SAE) is: • Death • Life-threatening event • Inpatient hospitalization or prolongation of existing hospitalization • Persistent or significant disability or incapacity • Congenital anomaly or birth defect • Other important medical event The severity of AEs were assessed by the investigator according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0: Grade 1 (Mild): asymptomatic/mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 (Moderate): minimal, local or noninvasive intervention indicated; limiting age-appropriate activities of daily living. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care Grade 4: Life-threatening; urgent intervention indicated. Grade 5: Death due to AE.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Mean Change in the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale V 4.0) Score From Baseline
The functional assessment of chronic illness therapy (FACIT-Fatigue Scale V 4.0) is a subscale of the FACIT-F and has been validated in the oncology setting. The FACIT-Fatigue Scale is a short, 13-item, self-administered tool that measures the level of fatigue in an individual during usually daily activities over the past week. The level of fatigue is measured on a 5-point Likert scale (0 = not at all; 4 = very much. The scores range from 0 to 52, with higher scores indicating less fatigue. If there were missing items, but the participant answered at least 50% of the items, then subscores were prorated.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Mean Change in the European Quality of Life-Five Dimensions-Three Levels (EQ-5D-3L) Score From Baseline
The EQ-5D-3L is a self-administered questionnaire consisting of 5 questions, pertaining to specific health dimensions (ie, mobility, self-care, pain, usual activities, and anxiety/depression) and a health status scale. Each question has 3 levels of severity, corresponding to no problems, moderate problems and severe problems. Canadian population sample weights were used to derive health utility scores. A higher utility score represents a better health state. A clinically meaningful improvement or worsening was defined as at least 0.08 points of improvement or 0.10 points of worsening from baseline, respectively, for the EQ-5D-3L Health Utility Index. The instrument is scored using the United Kingdom (UK) index ranges from -0.594 to 1, where 0 equates to death and 1 equates to full health; -0.594 is considered 'worse than death'.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Time to Definitive Clinically Meaningful Deterioration for ≥ 2 Consecutive Visits as Measured Using the EQ-5D HRQoL Scale
Clinically meaningful deterioration was defined at least 0.10 point of deterioration from baseline for at least 2 consecutive visits for the EQ-5D Health Utility Index. The EQ-5D-3L is a self-administered questionnaire consisting of 5 questions, pertaining to specific health dimensions (ie, mobility, self-care, pain, usual activities, and anxiety/depression) and a health status scale. Each question has 3 levels of severity, corresponding to no problems, moderate problems and severe problems. Canadian population sample weights were used to derive health utility scores. A higher utility score represents a better health state. A clinically meaningful improvement or worsening was defined as at least 0.08 points of improvement or 0.10 points of worsening from baseline, respectively, for the EQ-5D-3L Health Utility Index. The EQ-5D-3L is scored using the UK index ranges from -0.594 to 1, where 0 equates to death and 1 equates to full health; -0.594 is considered 'worse than death'.
Measured: From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months
Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Person Year
HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Healthcare Resource Utilization (HRU): Number of Days Hospitalized Per Person-Year
HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.
Measured: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2012 (estimated)
- Study start24 Apr 2013
- Primary completion15 Jul 2019
- Study completion18 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by CelgeneThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 472 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01757535
- Sponsor's protocol ID
- CC-486-AML-001
- Other IDs
- 2012-003457-28
- Acronym
- QUAZAR AML-001
- Status last verified
- Jun 2025
- First submitted
- 21 Nov 2012
- Last updated
- 8 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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.
PubMed 26785287Ravandi F, Dohner H, Wei AH, Montesinos P, Pfeilstocker M, Papayannidis C, Lai Y, Wang K, See WL, de Menezes DL, Petrlik E, Prebet T, Roboz GJ. Survival outcomes in patients with acute myeloid leukaemia who received subsequent therapy for relapse in QUAZAR AML-001. Br J Haematol. 2024 Mar;204(3):877-886. doi: 10.1111/bjh.19202. Epub 2023 Nov 12.
PubMed 37952982Wei AH, Roboz GJ, Dombret H, Dohner H, Schuh AC, Montesinos P, Selleslag D, Bondarenko SN, Prebet T, Lai Y, Skikne B, Beach CL, Ravandi F. Survival outcomes with oral azacitidine maintenance in patients with acute myeloid leukemia in remission by receipt of initial chemotherapy: subgroup analyses from the phase III QUAZAR AML-001 trial. Haematologica. 2023 Oct 1;108(10):2820-2825. doi: 10.3324/haematol.2022.282296. No abstract available.
PubMed 36951156Dohner 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.
PubMed 35960871Zhu 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.
PubMed 35437111Roboz 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.
PubMed 34995344Ravandi 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.
PubMed 34454540Wei 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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