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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01254188




Registration number
NCT01254188
Ethics application status
Date submitted
2/12/2010
Date registered
6/12/2010
Date last updated
3/03/2016

Titles & IDs
Public title
Safety and Efficacy of Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia Patients
Scientific title
Extending Molecular Responses With Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia (CML) Patients in Chronic Phase
Secondary ID [1] 0 0
CAMN107E2401
Universal Trial Number (UTN)
Trial acronym
ENESTxtnd
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic 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 - Nilotinib

Experimental: Nilotinib - 300 mg BID


Treatment: Drugs: Nilotinib
This was an open-label, single-arm, prospective, multi-center, Phase IIIb clinical study with nilotinib 300 mg bid treatment in newly diagnosed CML-CP patients not previously treated with imatinib therapy and diagnosed within 6 months of study entry. For patients insufficiently responding to nilotinib 300 mg bid, the dose may have been increased to 400 mg bid. Among patients with adverse events who had dose reduction, this study also allowed a possible re-escalation to 300 mg bid.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The Percentage of Patients Achieving MMR by 12 Months - MMR is defined as BCR-ABL ratio (%) on IS <= 0.1% (corresponds to >=3 log reduction of BCR-ABL transcripts from standardized baseline value). Clopper-Pearson method
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Time to Molecular Response at 24 Months - Estimated median time to first MMR by Kaplan-Meier method
Timepoint [1] 0 0
24 months
Secondary outcome [2] 0 0
Duration of Major Molecular Response - Kaplan-Meier estimates of duration of first MMR among patients who achieved MMR (FAS) Duration of first MMR (months) = (Minimum date of (loss of first MMR , CML-related death, progression to AP/BC during study treatment, censoring) - date of first MMR + 1) / 30.4375
Timepoint [2] 0 0
3, 6, 9, 12, 15, 18, 21, 24 Months after MMR was detected
Secondary outcome [3] 0 0
Complete Cytogenetic Response - Complete cytogenetic response (CCyR) is defined as a value of 0% Ph+ metaphases in bone marrow.
Timepoint [3] 0 0
6 months
Secondary outcome [4] 0 0
Percentage of Participants Estimated to Maintain Their First CCyR for 6, 12, 18, and 24 Months After the First CCyR Was Achieved as Determined by Kaplan Meier Estimatation. - * CCyR = 0% Ph+ metaphases based on at least 20 metaphases from bone marrow cytogenetics.
Duration of first CCyR (months) = (date of CCyR loss or censoring - date of first CCyR +1) / 30.4375
Timepoint [4] 0 0
6,12,18 and 24 months
Secondary outcome [5] 0 0
Overall Survival - OS was defined as the time between date of study entry and date of death due to any cause at any time during the study, including the follow-up period after discontinuation of treatment.
Timepoint [5] 0 0
3, 6, 9, 12, 15, 18, 21, 24 Months
Secondary outcome [6] 0 0
Kaplan-Meier Estimates of Progression-free Survival - PFS was defined as the time from the date of study entry to the date of event defined as the first documented disease progression to AP/BC or the date of death from any cause occurring on treatment.
Timepoint [6] 0 0
3,6,9,12,15,18,21,and 24 months
Secondary outcome [7] 0 0
Kaplan-Meier Estimates of Failure-free Survival - Time to event (months) = (date of event or censoring - date of study entry + 1) / 30.4375. Date of event is the earliest date of the following events during treatment : discontinuation of nilotinib for nilotinib-related adverse events, death due to any cause, progression to AP or BC, loss of PCyR, loss of CCyR, loss of CHR. Time is censored at the date of last assessment in the trial for patients without event.
Timepoint [7] 0 0
3,6,9,12,15,18,21,and 24 months

Eligibility
Key inclusion criteria
-Patients with chronic myeloid leukemia in the chronic phase diagnosed within 6 months of
study entry
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Treatment with tyrosine kinase inhibitor or other antileukemic agents or treatments
(including HSCT) for longer than 2 weeks, with exception of hydroxyurea and/or
anagrelide

- Uncontrolled congestive heart failure or hypertension

- Myocardial infarction or unstable angina pectoris within past 12 months

- Known T315I mutations

- QTcF >450 msec

- Significant arrhythmias

Other protocol-defined inclusion/exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Single group
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,SA,TAS,VIC,WA
Recruitment hospital [1] 0 0
Novartis Investigative Site - Canberra
Recruitment hospital [2] 0 0
Novartis Investigative Site - Concord NSW
Recruitment hospital [3] 0 0
Novartis Investigative Site - Gosford
Recruitment hospital [4] 0 0
Novartis Investigative Site - Kingswood
Recruitment hospital [5] 0 0
Novartis Investigative Site - Kogarah
Recruitment hospital [6] 0 0
Novartis Investigative Site - Liverpool
Recruitment hospital [7] 0 0
Novartis Investigative Site - St. Leonards
Recruitment hospital [8] 0 0
Novartis Investigative Site - Westmead
Recruitment hospital [9] 0 0
Novartis Investigative Site - Douglas
Recruitment hospital [10] 0 0
Novartis Investigative Site - Woolloongabba
Recruitment hospital [11] 0 0
Novartis Investigative Site - Adelaide
Recruitment hospital [12] 0 0
Novartis Investigative Site - Bedford Park
Recruitment hospital [13] 0 0
Novartis Investigative Site - Hobart
Recruitment hospital [14] 0 0
Novartis Investigative Site - Box Hill
Recruitment hospital [15] 0 0
Novartis Investigative Site - Clayton
Recruitment hospital [16] 0 0
Novartis Investigative Site - Fitzroy
Recruitment hospital [17] 0 0
Novartis Investigative Site - Heidelberg
Recruitment hospital [18] 0 0
Novartis Investigative Site - Parkville
Recruitment hospital [19] 0 0
Novartis Investigative Site - Wodonga
Recruitment hospital [20] 0 0
Novartis Investigative Site - Nedlands
Recruitment hospital [21] 0 0
Novartis Investigative Site - Perth
Recruitment postcode(s) [1] 0 0
2605 - Canberra
Recruitment postcode(s) [2] 0 0
2139 - Concord NSW
Recruitment postcode(s) [3] 0 0
2250 - Gosford
Recruitment postcode(s) [4] 0 0
2747 - Kingswood
Recruitment postcode(s) [5] 0 0
2217 - Kogarah
Recruitment postcode(s) [6] 0 0
2170 - Liverpool
Recruitment postcode(s) [7] 0 0
2065 - St. Leonards
Recruitment postcode(s) [8] 0 0
2145 - Westmead
Recruitment postcode(s) [9] 0 0
4810 - Douglas
Recruitment postcode(s) [10] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [11] 0 0
5000 - Adelaide
Recruitment postcode(s) [12] 0 0
5042 - Bedford Park
Recruitment postcode(s) [13] 0 0
7000 - Hobart
Recruitment postcode(s) [14] 0 0
3128 - Box Hill
Recruitment postcode(s) [15] 0 0
3168 - Clayton
Recruitment postcode(s) [16] 0 0
3065 - Fitzroy
Recruitment postcode(s) [17] 0 0
3084 - Heidelberg
Recruitment postcode(s) [18] 0 0
3050 - Parkville
Recruitment postcode(s) [19] 0 0
3690 - Wodonga
Recruitment postcode(s) [20] 0 0
6009 - Nedlands
Recruitment postcode(s) [21] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
Algeria
State/province [1] 0 0
Bouzareah
Country [2] 0 0
Algeria
State/province [2] 0 0
Oran
Country [3] 0 0
Argentina
State/province [3] 0 0
Buenos Aires
Country [4] 0 0
Argentina
State/province [4] 0 0
Entre Rios
Country [5] 0 0
Brazil
State/province [5] 0 0
BA
Country [6] 0 0
Brazil
State/province [6] 0 0
GO
Country [7] 0 0
Brazil
State/province [7] 0 0
PR
Country [8] 0 0
Brazil
State/province [8] 0 0
RS
Country [9] 0 0
Canada
State/province [9] 0 0
Alberta
Country [10] 0 0
Canada
State/province [10] 0 0
British Columbia
Country [11] 0 0
Canada
State/province [11] 0 0
New Brunswick
Country [12] 0 0
Canada
State/province [12] 0 0
Newfoundland and Labrador
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
Saskatchewan
Country [17] 0 0
Egypt
State/province [17] 0 0
Alexandria
Country [18] 0 0
Egypt
State/province [18] 0 0
Cairo
Country [19] 0 0
Egypt
State/province [19] 0 0
Mansoura
Country [20] 0 0
India
State/province [20] 0 0
Chennai
Country [21] 0 0
India
State/province [21] 0 0
Maharashtra
Country [22] 0 0
India
State/province [22] 0 0
Tamil Nadu
Country [23] 0 0
Israel
State/province [23] 0 0
Jerusalem
Country [24] 0 0
Israel
State/province [24] 0 0
Petach Tikva
Country [25] 0 0
Israel
State/province [25] 0 0
Ramat Gan
Country [26] 0 0
Lebanon
State/province [26] 0 0
Beirut
Country [27] 0 0
Lebanon
State/province [27] 0 0
Saida
Country [28] 0 0
Malaysia
State/province [28] 0 0
Pulau Pinang
Country [29] 0 0
Malaysia
State/province [29] 0 0
Selangor
Country [30] 0 0
Mexico
State/province [30] 0 0
Sonora
Country [31] 0 0
Mexico
State/province [31] 0 0
San Luis Potosi
Country [32] 0 0
Oman
State/province [32] 0 0
Muscat
Country [33] 0 0
Russian Federation
State/province [33] 0 0
Russia
Country [34] 0 0
Russian Federation
State/province [34] 0 0
Ekaterinburg
Country [35] 0 0
Russian Federation
State/province [35] 0 0
Irkutsk
Country [36] 0 0
Russian Federation
State/province [36] 0 0
Moscow
Country [37] 0 0
Russian Federation
State/province [37] 0 0
Nizhnii Novgorod
Country [38] 0 0
Russian Federation
State/province [38] 0 0
Rostov-on-Don
Country [39] 0 0
Russian Federation
State/province [39] 0 0
St Petersburg
Country [40] 0 0
Russian Federation
State/province [40] 0 0
St-Petersburg
Country [41] 0 0
Russian Federation
State/province [41] 0 0
Tula
Country [42] 0 0
Saudi Arabia
State/province [42] 0 0
Dammam
Country [43] 0 0
Saudi Arabia
State/province [43] 0 0
Jeddah
Country [44] 0 0
Saudi Arabia
State/province [44] 0 0
Riyadh
Country [45] 0 0
South Africa
State/province [45] 0 0
Gauteng
Country [46] 0 0
South Africa
State/province [46] 0 0
Bloemfontein
Country [47] 0 0
South Africa
State/province [47] 0 0
Observatory
Country [48] 0 0
South Africa
State/province [48] 0 0
Parktown
Country [49] 0 0
South Africa
State/province [49] 0 0
Pretoria
Country [50] 0 0
Taiwan
State/province [50] 0 0
Taiwan, ROC
Country [51] 0 0
Taiwan
State/province [51] 0 0
Lin-Kou
Country [52] 0 0
Taiwan
State/province [52] 0 0
Niaosong Township
Country [53] 0 0
Taiwan
State/province [53] 0 0
Taichung
Country [54] 0 0
Thailand
State/province [54] 0 0
Chiang Mai
Country [55] 0 0
Thailand
State/province [55] 0 0
Muang
Country [56] 0 0
Tunisia
State/province [56] 0 0
Tunisie
Country [57] 0 0
Tunisia
State/province [57] 0 0
Tunis
Country [58] 0 0
United Arab Emirates
State/province [58] 0 0
Dubai

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will further investigate the safety and efficacy of nilotinib in newly diagnosed
chronic myeloid leukemia patients in the chronic phase
Trial website
https://clinicaltrials.gov/show/NCT01254188
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
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

Summary results
Other publications