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A Multicenter Phase 3, Open-Label Study of Bosutinib Versus Imatinib in Adult Patients With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia

Phase 3
Completed
Conditions
Leukemia, Myelogenous, Chronic, Breakpoint Cluster Region-Abelson Proto-oncogene (BCR-ABL) Positive
Interventions
Registration Number
NCT02130557
Lead Sponsor
Pfizer
Brief Summary

Phase 3, 2-arm, randomized, open label trial. Patients will be randomized to receive bosutinib or imatinib for the duration of the study.

Detailed Description

The study will be open for enrollment until the planned number of approximately 500 Philadelphia Chromosome Positive (Ph+) patients have been randomized (approximately 250 Ph+ patients in each treatment arm; a total of approximately 530 Ph+ and Ph- patients). All patients will be treated and/or followed for approximately 5 years (240 weeks) after randomization until the study has closed. Patients who discontinue study therapy early due to disease progression or intolerance to study medication will continue to be followed yearly for survival for up to approximately 5 years (240 weeks) after randomization.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
536
Inclusion Criteria
  1. Molecular diagnosis of CP CML of ≤ 6 months (from initial diagnosis).
  2. Adequate hepatic, renal and pancreatic function.
  3. Age ≥ 18 years.
Exclusion Criteria
  1. Any prior medical treatment for CML, including tyrosine kinase inhibitors (TKIs), with the exception of hydroxyurea and/or anagrelide treatment, which are permitted for up to 6 months prior to study entry (signature of ICF) if suitably approved for use in the subject's region.
  2. Any past or current Central Nervous System (CNS) involvement, including leptomeningeal leukemia.
  3. Extramedullary disease only.
  4. Major surgery or radiotherapy within 14 days of randomization.
  5. History of clinically significant or uncontrolled cardiac disease.
  6. Known seropositivity to human immunodeficiency virus (HIV), current acute or chronic hepatitis B (hepatitis B surface-antigen positive), hepatitis C, cirrhosis or evidence of decompensated liver disease. Patients with resolved Hepatitis B can be included.
  7. Recent or ongoing clinically significant GI disorder, e.g. Crohn's Disease, Ulcerative Colitis, or prior total or partial gastrectomy.
  8. History of another malignancy within 5 years with the exception of basal cell carcinoma or cervical carcinoma in situ or stage 1 or 2 cancer that is considered adequately treated and currently in complete remission for at least l2 months.
  9. Current, or recent (within 30 days, or 5 half-lives of investigational product) participation in other clinical trials of investigational agents and/or containing interventional procedures deemed contrary to the objectives and conduct of this trial.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
ImatinibImatinibImatinib, 400 mg, oral administration once a day
BosutinibBosutinibBosutinib, 400 mg, oral administration once a day
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With Major Molecular Response (MMR) at Month 12Month 12

MMR was defined as a ratio of breakpoint cluster region to abelson (BCR-ABL/ABL) less than or equal to (\<=) 0.1 percent (%) on the international scale (IS) (greater than or equal to \[\>=\] 3 log reduction from standardized baseline in ratio of BCR-ABL to ABL transcripts \[\>=3000 ABL required\]) by quantitative reverse transcriptase polymerase chain reaction (RT-qPCR). The percentage of participants with MMR at Month 12 are reported.

Secondary Outcome Measures
NameTimeMethod
Kaplan-Meier Estimate of Probability of Retaining Complete Cytogenetic Response (CCyR) at Month 48Month 48

The Kaplan-Meier curve was generated based on the first date of CCyR until the date of the confirmed loss of CCyR or censoring, objectively documented, for responders only. Confirmed loss of CCyR was the presence of at least one Ph+ metaphase confirmed by a second assessment at least 28 days later. Treatment discontinuation due to suboptimal response/treatment failure, PD or death due to PD within 28 days of last dose were considered confirmed loss of CCyR. PD was defined as disease progression to AP or BP CML.

Percentage of Participants With Complete Cytogenetic Response (CCyR) Up to Month 12Up to Month 12

Complete Cytogenetic Response (CCyR) was based on the prevalence of Ph+ metaphases among cells in metaphase on a bone marrow (BM) aspirate. CCyR was achieved when there was 0% Ph+ metaphases among cells in a BM sample when at least 20 metaphases from a BM sample were analyzed, or MMR if no BM was available. The percentage of participants with CCyR for up to Month 12 are reported.

Kaplan-Meier Estimate of Probability of Retaining Major Molecular Response (MMR) at Month 48Month 48

The Kaplan-Meier curve was generated based on the first date of MMR until the date of the confirmed loss of MMR or censoring, objectively documented, for responders only. Confirmed loss of MMR was BCR-ABL/ABL IS ratio \>0.1% in association with a \>=5-fold increase in BCR-ABL/ABL IS ratio from the lowest value achieved up to that time-point confirmed by a second assessment at least 28 days later. Treatment discontinuation due to suboptimal response/treatment failure, progressive disease (PD) or death due to PD within 28 days of last dose were considered confirmed loss of MMR. PD was defined as disease progression to accelerated phase (AP) or blast phase (BP) CML.

Cumulative Incidence of Event Free Survival (EFS) EventsUp to Month 60

EFS was defined as time from randomization to death due to any cause, transformation to AP or BP at any time, confirmed loss of complete hematologic response (CHR), confirmed loss of CCyR or censoring. Loss of CHR was defined as a hematologic assessment of non-CHR (chronic phase, AP, or BP) confirmed by 2 assessments at least 4 weeks apart. Loss of CHR was defined as appearance of any of the following: WBC count that rises to \>20.0\*10\^9/L, platelet count rises to \>=600\*10\^9/L, appearance of palpable spleen or other extramedullary involvement proven by biopsy, appearance of 5% myelocytes in peripheral blood, appearance of blasts or promyelocytes in peripheral blood. Loss of CCyR was defined as at least 1 Ph+ metaphase from analysis of \<100 metaphases confirmed by follow up cytogenetic analysis after 1 month. Cumulative incidence of EFS was defined as percentage of participants with EFS event at Month 60 and was adjusted for competing risk of treatment discontinuation without event.

Overall Survival (OS) RateUp to Month 60

OS was defined as the time (in months) from randomization to the occurrence of death due to any cause or censoring. Kaplan-meier analysis was used for determination of OS. Percentage of participants who were alive were estimated in this outcome measure.

Percentage of Participants With Major Molecular Response (MMR) Up to Month 18Up to Month 18

MMR was defined as a ratio of BCR-ABL/ABL \<=0.1% on the international scale (\>=3 log reduction from standardized baseline in ratio of BCR-ABL to ABL transcripts \[\>=3000 ABL required\]) by quantitative RT-qPCR. The percentage of participants with MMR for up to Month 18 are reported.

Trial Locations

Locations (182)

Pacific Cancer Medical Center, Inc.

🇺🇸

Anaheim, California, United States

Emory University Hospital

🇺🇸

Atlanta, Georgia, United States

The Emory Clinic

🇺🇸

Atlanta, Georgia, United States

Winship Cancer Institute, Emory University

🇺🇸

Atlanta, Georgia, United States

Kaiser Permanente Hawaii

🇺🇸

Honolulu, Hawaii, United States

Saint Alphonsus Regional Medical Center, Cancer Care Center

🇺🇸

Boise, Idaho, United States

Saint Alphonsus Regional Medical Center

🇺🇸

Boise, Idaho, United States

Saint Alphonsus Caldwell Cancer Care Center

🇺🇸

Caldwell, Idaho, United States

Saint Alphonsus Medical Center Nampa

🇺🇸

Nampa, Idaho, United States

University of Illinois Cancer Center

🇺🇸

Chicago, Illinois, United States

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Pacific Cancer Medical Center, Inc.
🇺🇸Anaheim, California, United States

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