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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01074047




Registration number
NCT01074047
Ethics application status
Date submitted
16/02/2010
Date registered
24/02/2010
Date last updated
29/08/2017

Titles & IDs
Public title
Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)
Scientific title
A Phase 3, Multicenter, Randomized, Open-Label, Study of Azacitidine (Vidaza®) Versus Conventional Care Regimens for the Treatment of Older Subjects With Newly Diagnosed Acute Myeloid Leukemia
Secondary ID [1] 0 0
AZA-AML-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Myeloid Leukemia 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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Azacitidine
Treatment: Drugs - Conventional Care Regimen

Experimental: Azacitidine - Azacitidine daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity

Active comparator: Conventional Care Regimen - Conventional Care Regimen


Treatment: Drugs: Azacitidine
75 mg/m\^2 subcutaneous (SC) daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity

Treatment: Drugs: Conventional Care Regimen
Physician pre-selects prior to randomization from one of the following:

* Intensive chemotherapy (cytarabine 100-200 mg/m\^2 continuous intravenous infusion for 7 days + anthracycline IV x 3 days) + Best Supportive Care; induction with up to 2 consolidation cycles
* Low-dose cytarabine 20 mg subcutaneous (SC) twice a day (BID) for 10 days, for 28 day cycles + BSC; until disease progression or unacceptable toxicity
* Best Supportive Care only; until study end
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Kaplan-Meier Estimates for Overall Survival
Timepoint [1] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [1] 0 0
One-year Overall Survival Rate
Timepoint [1] 0 0
From Day 1 (randomization) to 40 months
Secondary outcome [2] 0 0
Event-free Survival (EFS)
Timepoint [2] 0 0
Day 1 (randomization) to date of treatment failure, progressive disease, relapse after Complete Remission (CR) or Complete remission with incomplete blood count recovery (CRi), death from any cause. Day 1 (randomization) to 40 months
Secondary outcome [3] 0 0
Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi)
Timepoint [3] 0 0
Day 1 of first documented CR or CRi to the date of relapse, death from any cause, or lost to follow-up. Day 1 (randomization) to 40 months
Secondary outcome [4] 0 0
Percentage of Participants Who Achieved a Morphologic CR + CRi as Determined by the Independent Review Committee (IRC) Based on International Working Group (IWG) Response Criteria for Acute Myeloid Leukemia (AML)
Timepoint [4] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [5] 0 0
Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates
Timepoint [5] 0 0
Day 1 (randomization) to 40 months; date of the first documented CR or CRi until date of first documented relapse.
Secondary outcome [6] 0 0
Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC.
Timepoint [6] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [7] 0 0
Number of Participants With Adverse Events (AEs)
Timepoint [7] 0 0
Day 1 (randomization) up to last visit completed; final data cut off of 28 Feb 2017
Secondary outcome [8] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain
Timepoint [8] 0 0
Baseline to Cycle 3; at approximately 3 months
Secondary outcome [9] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain
Timepoint [9] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [10] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain
Timepoint [10] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [11] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain
Timepoint [11] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [12] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain
Timepoint [12] 0 0
Baseline to End of Study; at approximately 11-12 months
Secondary outcome [13] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
Timepoint [13] 0 0
Baseline to Cycle 3, at approximately 3 months
Secondary outcome [14] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
Timepoint [14] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [15] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
Timepoint [15] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [16] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
Timepoint [16] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [17] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
Timepoint [17] 0 0
Baseline to end of study, at approximately 11-12 months
Secondary outcome [18] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
Timepoint [18] 0 0
Baseline to Cycle 3, at approximately 3 months
Secondary outcome [19] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
Timepoint [19] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [20] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
Timepoint [20] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [21] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
Timepoint [21] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [22] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
Timepoint [22] 0 0
Baseline to end of study, at approximately 11-12 months
Secondary outcome [23] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
Timepoint [23] 0 0
Baseline to Cycle 3, at approximately 3 months
Secondary outcome [24] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
Timepoint [24] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [25] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
Timepoint [25] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [26] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
Timepoint [26] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [27] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
Timepoint [27] 0 0
Baseline to end of study, at approximately 11-12 months
Secondary outcome [28] 0 0
Healthcare Resource Utilization (HRU): Number of Inpatient Hospitalizations
Timepoint [28] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [29] 0 0
Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Per Year
Timepoint [29] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [30] 0 0
HRU: Number of Participants Receiving Transfusions
Timepoint [30] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [31] 0 0
HRU: Rate of Transfusions Per Patient Year
Timepoint [31] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [32] 0 0
Number of Participants in the Extension Phase With Treatment Emergent Adverse Events (TEAEs)
Timepoint [32] 0 0
From the date of informed consent for the Extension Phase through to the date of last dose of study drug + 28 days up to last visit completed 24 July 2016; maximum duration of exposure to Azacitidine was 871 days

Eligibility
Key inclusion criteria
* Diagnosis of one of the following

* Newly diagnosed de novo acute myeloid leukemia (AML)
* AML secondary to myelodysplastic syndromes (MDS)
* AML secondary to exposure to leukemogenic therapy or agents with primary malignancy in remission for at least 2 years
* Bone marrow blasts >30%
* Age = 65 years
* Easter Cooperative Oncology Group (ECOG) 0-2
Minimum age
65 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Previous cytotoxic or biologic treatment for AML (except hydroxyurea)
* Previous treatment with azacitidine, decitabine or cytarabine
* Prior use of targeted therapy agents (e.g., FLT3 inhibitors, other kinase inhibitors)
* AML French American British subtype (FAB M3)
* AML associated with inv(16), t(8;21), t(16;16), t(15:17), or t(9;22) karyotypes
* Prior bone marrow or stem cell transplantation
* Candidate for allogeneic bone marrow or stem cell transplant
* Diagnosis of malignant disease within the previous 12 months (excluding base cell carcinoma, "in-situ" carcinoma of the cervix or breast or other local malignancy excised or irradiated with a high probability of cure)
* Malignant hepatic tumors
* Uncontrolled systemic infection
* Active viral infection with Human Immunodeficiency Virus (HIV) or Hepatitis type B or C
* Use of any experimental drug or therapy within 28 days prior to Day 1

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
Open (masking not used)
Who is / are masked / blinded?



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
1/06/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/07/2016
Sample size
Target
Accrual to date
Final
488
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
Recruitment hospital [1] 0 0
Prince of Wales Hospital - Randwick
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [3] 0 0
Peter MacCallum Cancer Centre - East Melbourne
Recruitment hospital [4] 0 0
Western Hospital - Footscray
Recruitment hospital [5] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [6] 0 0
St Vincent's Hospital - Fitzroy
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
3002 - East Melbourne
Recruitment postcode(s) [4] 0 0
3011 - Footscray
Recruitment postcode(s) [5] 0 0
3050 - Melbourne
Recruitment postcode(s) [6] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Austria
State/province [3] 0 0
Upper Austria
Country [4] 0 0
Austria
State/province [4] 0 0
Vienna
Country [5] 0 0
Austria
State/province [5] 0 0
Salzburg
Country [6] 0 0
Belgium
State/province [6] 0 0
Hainaut
Country [7] 0 0
Belgium
State/province [7] 0 0
Namur
Country [8] 0 0
Belgium
State/province [8] 0 0
Oost-vlaanderen
Country [9] 0 0
Belgium
State/province [9] 0 0
West-vlaanderen
Country [10] 0 0
Belgium
State/province [10] 0 0
La Louvière
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
Manitoba
Country [13] 0 0
Canada
State/province [13] 0 0
Nova Scotia
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
Canada
State/province [15] 0 0
Quebec
Country [16] 0 0
Canada
State/province [16] 0 0
Calgary
Country [17] 0 0
China
State/province [17] 0 0
Beijing
Country [18] 0 0
China
State/province [18] 0 0
Jiangsu
Country [19] 0 0
China
State/province [19] 0 0
Shanghai
Country [20] 0 0
China
State/province [20] 0 0
Sichuan
Country [21] 0 0
China
State/province [21] 0 0
Tianjin
Country [22] 0 0
Czechia
State/province [22] 0 0
Jihormoravsky Kraj
Country [23] 0 0
Czechia
State/province [23] 0 0
Olomoucký Kraj
Country [24] 0 0
Czechia
State/province [24] 0 0
Praha
Country [25] 0 0
France
State/province [25] 0 0
Alsace
Country [26] 0 0
France
State/province [26] 0 0
ILE-DE-France
Country [27] 0 0
France
State/province [27] 0 0
Ile-de-france
Country [28] 0 0
France
State/province [28] 0 0
Limousin Lorraine
Country [29] 0 0
France
State/province [29] 0 0
Midi-pyrénées
Country [30] 0 0
France
State/province [30] 0 0
Nice
Country [31] 0 0
France
State/province [31] 0 0
Pays de La Loire
Country [32] 0 0
France
State/province [32] 0 0
Picardie
Country [33] 0 0
France
State/province [33] 0 0
Provence Alpes Cote D'azur
Country [34] 0 0
France
State/province [34] 0 0
Aquitaine
Country [35] 0 0
France
State/province [35] 0 0
Lyon Cedex 03
Country [36] 0 0
Germany
State/province [36] 0 0
Baden-wuerttemberg
Country [37] 0 0
Germany
State/province [37] 0 0
Mecklenburg-vorpommern
Country [38] 0 0
Germany
State/province [38] 0 0
Nordrhein-westfalen
Country [39] 0 0
Germany
State/province [39] 0 0
Nordrhein-Westfallen
Country [40] 0 0
Germany
State/province [40] 0 0
Sachsen
Country [41] 0 0
Germany
State/province [41] 0 0
Thueringen
Country [42] 0 0
Israel
State/province [42] 0 0
Beersheva
Country [43] 0 0
Israel
State/province [43] 0 0
Beer Yaakov
Country [44] 0 0
Israel
State/province [44] 0 0
Jerusalem
Country [45] 0 0
Israel
State/province [45] 0 0
Petach Tikva
Country [46] 0 0
Israel
State/province [46] 0 0
Tel Aviv
Country [47] 0 0
Israel
State/province [47] 0 0
Tel Hashomer
Country [48] 0 0
Italy
State/province [48] 0 0
Potenza
Country [49] 0 0
Italy
State/province [49] 0 0
Turin
Country [50] 0 0
Italy
State/province [50] 0 0
Alessandria
Country [51] 0 0
Italy
State/province [51] 0 0
Ancona
Country [52] 0 0
Italy
State/province [52] 0 0
Bari
Country [53] 0 0
Italy
State/province [53] 0 0
Bologna
Country [54] 0 0
Italy
State/province [54] 0 0
Firenze
Country [55] 0 0
Italy
State/province [55] 0 0
Reggio Calabria
Country [56] 0 0
Italy
State/province [56] 0 0
Roma
Country [57] 0 0
Italy
State/province [57] 0 0
Udine
Country [58] 0 0
Italy
State/province [58] 0 0
Varese
Country [59] 0 0
Korea, Republic of
State/province [59] 0 0
Seoul
Country [60] 0 0
Korea, Republic of
State/province [60] 0 0
Daegu
Country [61] 0 0
Netherlands
State/province [61] 0 0
Groningen
Country [62] 0 0
Poland
State/province [62] 0 0
Dolnoslaskie
Country [63] 0 0
Poland
State/province [63] 0 0
Lodzkie
Country [64] 0 0
Poland
State/province [64] 0 0
Mazowieckie
Country [65] 0 0
Poland
State/province [65] 0 0
Slaskie
Country [66] 0 0
Russian Federation
State/province [66] 0 0
Ekaterinburg
Country [67] 0 0
Russian Federation
State/province [67] 0 0
Moscow
Country [68] 0 0
Russian Federation
State/province [68] 0 0
Nizhniy Novgorod
Country [69] 0 0
Russian Federation
State/province [69] 0 0
Saint Petersburg
Country [70] 0 0
Russian Federation
State/province [70] 0 0
Saratov
Country [71] 0 0
Spain
State/province [71] 0 0
Asturias
Country [72] 0 0
Spain
State/province [72] 0 0
Baleares
Country [73] 0 0
Spain
State/province [73] 0 0
Barcelona
Country [74] 0 0
Spain
State/province [74] 0 0
Madrid
Country [75] 0 0
Spain
State/province [75] 0 0
Salamanca
Country [76] 0 0
Spain
State/province [76] 0 0
Sevilla
Country [77] 0 0
Spain
State/province [77] 0 0
Valencia
Country [78] 0 0
Taiwan
State/province [78] 0 0
Kaohsiung
Country [79] 0 0
Taiwan
State/province [79] 0 0
Taipei
Country [80] 0 0
United Kingdom
State/province [80] 0 0
Surrey
Country [81] 0 0
United Kingdom
State/province [81] 0 0
Bournemouth
Country [82] 0 0
United Kingdom
State/province [82] 0 0
London
Country [83] 0 0
United Kingdom
State/province [83] 0 0
Manchester
Country [84] 0 0
United Kingdom
State/province [84] 0 0
Oxford
Country [85] 0 0
United Kingdom
State/province [85] 0 0
Wolverhampton

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
C L Beach, PharmD
Address 0 0
Celgene Corporation
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01074047