Phase II Study for Safety and Efficacy Evaluation of Imatinib Mesylate in Children With Chronic Myeloid Leukemia (CML) Philadelphia Chromosome-positive (Ph+)

The purpose of this study is to evaluate the hematological, cytogenetic and molecular response to continuous-use of Imatinib in children with CML Ph+.

Study Overview

• Status: Unknown
• Conditions: Chronic Myeloid Leukemia (CML) With Philadelphia Chromosome-positive (Ph+)
• Intervention / Treatment: Drug: Imatinib Mesylate

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• Brazil
  • SP
    • Sao Paulo, SP, Brazil, 08270-070
      • Hospital Santa Marcelina

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Diagnose: suspected CML (hematology and/or myelogram and/or immunophenotyping and/or Leukocyte alkaline phosphatase [LAP]) to be confirmed, after, by cytogenetic and/or molecular biology. OBS: only CML Ph+ newly-diagnose in chronic or accelerate phase; resistant CML Ph+ to Interferon α (INF-α), Hydroxyurea and/or low-dose ARA-C in chronic or accelerate phase; CML Ph+ with cytogenetic relapse after BMT, that didn't use Imatinib previously, in chronic or accelerate phase.
2. Female patients of childbearing age, should have pregnancy test (blood βhCG) performed before treatment initiation. Effective contraception must be used. Pregnant women won't be included.
3. Karnofsky and Lansky scale: ≥40.
4. Life expectation > 8 weeks.
5. Laboratory: renal function (serum creatinine ≤ 1,5 x ULN and/or Clearance ≥70 ml/min/1,73m2), hepatic function (total bilirubin ≤ 1,5 x ULN, TGP < 3 x ULN and albumin > 2 g/dl.
6. CNS toxicity ≤ II
7. Cardiac function: normal ejection fraction.
8. Signed ICF by child legal responsible.

Exclusion Criteria:

1. Patient receiving any other tyrosine kinase inhibitor (TKI).
2. Pregnant patient or breastfeeding.
3. Patient considered incapable to follow purposed treatment.
4. Patients with molecular relapsed.

5. Medications:

   • Colony stimulating: it cannot be administered at least 1 week before treatment.
   • Anticonvulsants: Imatinib is metabolized by P-450 enzyme, thereby subject cannot receive drug that activates the P-450 system. The anticonvulsants allowed are valproic acid and benzodiazepines.
   • Anticoagulants: The use of warfarin (Marevan) is not allowed. If anticoagulant is needed, low-molecular-weight heparin (LMWH) can be used. Avoid anticoagulants with platelets < 50000.
   • INF-Α 48h before D1.
   • Hydroxyurea 24h before D1.
   • ARA-C doses >100 mg/m2 for 5-7 days, 14 days before D1.
   • Anthracyclines, Mitoxantrone or Etoposide 21 days before D1.
   • Any other chemotherapeutic agent 28 days before D1.
   • Hematopoietic Cell Transplantation (HCT) 6 weeks before D1.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Imatinib Mesylate	Drug: Imatinib Mesylate; Patient will receive Imatinib Mesylate, continuous-use, 260 mg/m2/day dose, maximum allowed 400 mg, for 24 months.; Other Names: Glivec, MI

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Evaluate the complete cytogenetic response with continuous-use of Imatinib.	Up to 12 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Evaluate the response to continuous-use of Imatinib and the toxicity and tolerability in children with CML Ph+.	Up to 24 months

Collaborators and Investigators

• Sponsor: Renato Melaragno
• Investigators: Study Chair: Alejandro M Arancibia, MD, Casa de Saude Santa Marcelina

Study record dates

Study Major Dates

• Study Start: February 1, 2011
• Primary Completion (ANTICIPATED): June 1, 2013
• Study Completion (ANTICIPATED): December 1, 2013

Study Registration Dates

• First Submitted: October 14, 2010
• First Submitted That Met QC Criteria: October 14, 2010
• First Posted (ESTIMATE): October 15, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): March 26, 2013
• Last Update Submitted That Met QC Criteria: March 25, 2013
• Last Verified: March 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Chromosome Aberrations; Translocation, Genetic; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Philadelphia Chromosome; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Imatinib Mesylate
• Other Study ID Numbers: CSTI571ABR23T