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Safety and Efficacy of Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia Patients (ENESTxtnd)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01254188
Recruitment Status : Completed
First Posted : December 6, 2010
Results First Posted : March 3, 2016
Last Update Posted : March 3, 2016
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
This study will further investigate the safety and efficacy of nilotinib in newly diagnosed chronic myeloid leukemia patients in the chronic phase

Condition or disease Intervention/treatment Phase
Chronic Myeloid Leukemia Drug: Nilotinib Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 421 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Extending Molecular Responses With Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia (CML) Patients in Chronic Phase
Study Start Date : April 2011
Actual Primary Completion Date : November 2014
Actual Study Completion Date : November 2014

Arm Intervention/treatment
Experimental: Nilotinib
300 mg BID
Drug: 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.
Other Name: AMN107

Primary Outcome Measures :
  1. The Percentage of Patients Achieving MMR by 12 Months [ Time Frame: 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

Secondary Outcome Measures :
  1. Time to Molecular Response at 24 Months [ Time Frame: 24 months ]
    Estimated median time to first MMR by Kaplan-Meier method

  2. Duration of Major Molecular Response [ Time Frame: 3, 6, 9, 12, 15, 18, 21, 24 Months after MMR was detected ]
    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

  3. Complete Cytogenetic Response [ Time Frame: 6 months ]
    Complete cytogenetic response (CCyR) is defined as a value of 0% Ph+ metaphases in bone marrow.

  4. 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. [ Time Frame: 6,12,18 and 24 months ]

    * 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

  5. Overall Survival [ Time Frame: 3, 6, 9, 12, 15, 18, 21, 24 Months ]
    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.

  6. Kaplan-Meier Estimates of Progression-free Survival [ Time Frame: 3,6,9,12,15,18,21,and 24 months ]
    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.

  7. Kaplan-Meier Estimates of Failure-free Survival [ Time Frame: 3,6,9,12,15,18,21,and 24 months ]
    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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

-Patients with chronic myeloid leukemia in the chronic phase diagnosed within 6 months of study entry

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01254188

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Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01254188    
Obsolete Identifiers: NCT01580059
Other Study ID Numbers: CAMN107E2401
First Posted: December 6, 2010    Key Record Dates
Results First Posted: March 3, 2016
Last Update Posted: March 3, 2016
Last Verified: February 2016
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Chronic myeloid leukemia,
Philadelphia chromosome
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Neoplasms by Histologic Type
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases