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Study of Imatinib Discontinuation in Chronic Myeloid Leukemia with Deep Molecular Response

Phase 2
Completed
Conditions
Leukemia, Chronic Myeloid
Interventions
Other: imatinib discontinuation
Registration Number
NCT02852486
Lead Sponsor
University of Campinas, Brazil
Brief Summary

The purpose of this study is to evaluate treatment-free remission after imatinib discontinuation in patients with chronic myeloid leukemia with deep molecular response. Before discontinuation, patients will receive pioglitazone associated with imatinib during 3 months.

Detailed Description

Treatment of chronic myeloid leucemia (CML) with tyrosine kinase inhibitors (TKIs) changed dramatically the prognosis of CML, with high rates of cytogenetic and molecular remission and increase of overall and progression-free survival. However, the long-term treatment of CML has a high cost to the health system, due to the price of these drugs and the need for continued use. In addition, chronic adverse effects may compromise the quality of life of patients. Discontinuation trials of TKIs have been developed in order to identify groups of patients who may benefit from treatment discontinuation if they have obtained deeper molecular responses.The primary objective of this study is to evaluate treatment free remission (TFR) after imatinib discontinuation in patients treated for more than 3 years with imatinib and with deep molecular response stable for two years (defined in the present study as a molecular response of 4.5 log reduction in breakpoint cluster region (BCR)-Abelson murine leukemia viral oncogene homolog 1(ABL) transcripts levels according to the international scale (MR 4.5; BCR-ABL/ABL ratio \< or = 0.0032%). Patients with these criteria will receive pioglitazone for 3 months concomitant with imatinib, prior to discontinuation. After imatinib discontinuation, patients will be evaluated by molecular assessment of BCR-ABL transcripts levels by quantitative real time polymerase chain reaction (RQ-PCR) monthly during the first year, every 2 months in the second year and then every 3 months. The criteria for restarting treatment will be the loss of major molecular response (MMR), documented by a single RQ-PCR test \> 0.1%, or confirmed loss of 4 log reduction molecular response (MR4.0), by 2 consecutive RQ-PCR tests \> 0.01%.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
31
Inclusion Criteria
  • CML in chronic phase
  • treatment with imatinib for 3 or more years
  • MR4.5 (RQ-PCR< ou =0.0032%) confirmed by 4 RQ-PCR tests for BCR-ABL in the last 2 years (2 tests within the last 6 months)
  • Eastern Cooperative Oncology Group Performance Status (ECOG) 0-2
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 reference level
  • Bilirubins ≤ 1.5 reference level
  • Contraceptive precautions for women
Exclusion Criteria
  • Patients less than 18 years
  • Severe organ disfunction (liver or kidney)
  • Severe cardiovascular disease: grade I-IV from New York Heart Association (NYHA) or acute myocardial infarction in the last six months, symptomatic arrhythmias
  • Fluid retention grade 3 or 4
  • Osteoporosis in treatment
  • Patients with previous CML in accelerated or blast phase or blast or Philadelphia positive (Ph+) acute lymphoid leukemia (ALL)
  • BCR-ABL mutations related to resistance
  • Previous allogeneic bone marrow transplantation

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
PioglitazonePioglitazone 30 Mg Oral TabletPioglitazone will be given 30 mg/day, orally, for 3 months, before imatinib discontinuation
Pioglitazoneimatinib discontinuationPioglitazone will be given 30 mg/day, orally, for 3 months, before imatinib discontinuation
Primary Outcome Measures
NameTimeMethod
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 months

assessment of number of participants with treatment-related adverse events as assessed by CTCAE v4.0 during the 3 months of treatment with imatinib and pioglitazone

Treatment-free remission after imatinib discontinuationThrough study completion (five years)

Treatment-free remission time after imatinib discontinuation in patients with CML treated with pioglitazone for 3 months before imatinib discontinuation. Calculated from the date of imatinib discontinuation until imatinib reintroduction

Secondary Outcome Measures
NameTimeMethod
Time from imatinib discontinuation until loss of MMRThrough study completion (five years)

Time measured from the date of imatinib discontinuation until the date of loss of MMR, in days

proportion of patients with MMR, MR4.0, MR4.53, 6 and 12 months

Proportion of patients with MMR, MR4.0 and MR4.5 at 3, 6 and 12 months after imatinib discontinuation

Rate of loss of complete cytogenetic responseThrough study completion (five years)

% of patients who lost complete cytogenetic response

Time to reach MMR after restarting imatinibThrough study completion (five years)

Time measured from the date of the reestart of imatinib until the achievement of MMR, days

Overall survival after imatinib discontinuationThrough study completion (five years)

Overall survival is calculated from the date of imatinib discontinuation until the date of death by any cause

Progression-free survival after imatinib discontinuationThrough study completion (five years)

PFS is calculated from the date of imatinib discontinuatio until the date of progression of the disease

Event-free survival after imatinib discontinuationThrough study completion (five years)

Event-free is calculated from the date of imatinib discontinuation until the date of loss of complete hematologic response, disease progression or death

Molecular relapse free survivalThrough study completion (five years)

Calculated from the date of imatinib discontinuation until de loss of major molecular response

Trial Locations

Locations (1)

Centro de Hematologia e Hemoterapia - Universidade Estadual de Campinas

🇧🇷

Campinas, SP, Brazil

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