Study of efficacy and safety of nilotinib in pediatric CML patients

A multi-center, open label, non-controlled phase II study to evaluate efficacy and safety of oral nilotinib in pediatric patients with newly diagnosed Ph+ chronic myelogenous leukemia (CML) in chronic phase (CP) or with Ph+ CML in CP or accelerated phase (AP) resistant or intolerant to either imatinib or dasatinib

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080222210

Enrollment

Registered

2013-09-04

Start date

2013-08-20

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Male or female pediatric patients (1 to <18 years of age) with newly diagnosed Ph+ CML-CP (at least 15 patients) or with Ph+ CML in CP (at least 15 patients) or AP (no minimum number specified) resistant or intolerant to imatinib or dasatinib.

Interventions

investigational material(s) Generic name etc : AMN107 (Nilotinib) INN of investigational material : Nilotinib Therapeutic category code : 429 Other antitumor agents Dosage and Administration for Inves

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Male or female patients from 1 year of age to less than 18 years of age at study entry 2. Patients must have the diagnosis of newly diagnosed Ph+ CML-CP or Ph+CML-CP or AP resistant or intolerant to either imatinib or dasatinib 3. Performance status: Karnofsky >= 50% for patients > 10 years of age, and Lansky >= 50 for patients = LLN (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of study medication 6. Written informed consent must be obtained prior to any screening procedures

Exclusion criteria

Exclusion criteria: -Patients actively receiving therapy with strong CYP3A4 inhibitors or inducers and the treatment cannot be either discontinued or switched to a different medication at least 14 days prior to starting study drug. -Patients who are currently receiving treatment with any medications that have a known risk or possible risk to prolong the QT interval and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug. -Acute or chronic liver, pancreatic or severe renal disease considered unrelated to CML. -History of pancreatitis within 12 months of starting study drug or past medical history of chronic pancreatitis. -In case of Stem Cell Transplant (SCT) or Rescue without total body irradiation (TBI): Evidence of either active graft vs. host disease or less than 3 months since SCT. -Patients who have a known hypersensitivity to the active ingredient or any of the excipients including lactose.

Design outcomes

Primary

Measure	Time frame
efficacy -	—

Secondary

Measure	Time frame
safety -	—

Countries

Asia except Japan, Europe, Japan, North America, Oceania, South America

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026