Open label, phase II study to evaluate efficacy and safety of oral nilotinib in Philadelphia positive (Ph+) chronic myelogenous leukemia (CML) pediatric 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

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-000200-41-IT

Enrollment

Registered

2013-05-27

Start date

2013-07-22

Completion date

Unknown

Last updated

2014-03-10

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

Conditions

newly diagnosed Ph+ chronic myelogenous leukemia (CML) in chronic phase (CP) or Ph+ CML in CP or accelerated phase (AP) resistant or intolerant to either imatinib or dasatinib- bone marrow disease MedDRA version: 14.1 Level: LLT Classification code 10054352 Term: Chronic phase chronic myeloid leukemia System Organ Class: 100000004864

Interventions

Trade Name: Tasigna Product Name: nilotinib Product Code: AMN107 Pharmaceutical Form: Capsule, hard INN or Proposed INN: NILOTINIB CAS Number: 641571-10-0 Current Sponsor code: AMN107 Other descriptiv

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients eligible for inclusion in this study have to meet all of the following criteria; additional inclusion criteria may apply as per protocol: 1. Newly diagnosed and untreated Ph+ CML CP or Ph+ CML CP or AP resistant or intolerant to either imatinib or dasatinib 2. Karnofsky or Lansky = 50 3. Adequate renal, hepatic and pancreatic function 4. Potassium, magnesium, phosphorus and total calcium values = LLN (lower limit of normal) 5. Written informed consent Additional inclusion criteria are defined in the protocol. Are the trial subjects under 18? yes Number of subjects for this age range: 70 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: Patients eligible for this study must not meet any of the following criteria: 1. Treatment with strong CYP3A4 inhibitors or inducers 2. Use or planned use of any medications that have a known risk or possible risk to prolong the QT interval 3. Acute or chronic liver, pancreatic or severe renal disease 4. History of pancreatitis or chronic pancreatitis. 5. Impaired cardiac function 6. No evidence of active graft vs host and <3mo since Stem Cell Transplant 7. Total body irradiation (TBI) or craniospinal radiation therapy <6months 8. Hypersensitivity to the active ingredient or any of the excipients including lactose Additional exclusion criteria are defined in the protocol.

Design outcomes

Primary

Measure	Time frame
Main Objective: 1. To assess efficacy of nilotinib in pediatric patients with Ph+ CML CP resistant or intolerant to either imatinib or dasatinib 2. To assess efficacy of nilotinib in pediatric patients with Ph+ CML AP resistant or intolerant to either imatinib or dasatinib 3. To assess efficacy of nilotinib in pediatric patients with newly diagnosed Ph+ CML CP;Secondary Objective: 1. To further characterize efficacy and PK profile of nilotinib in pediatric patients with Ph+ CML 2. To further characterize safety and tolerability of nilotinib in pediatric patients with Ph+ CML 3. To identify emerging signs of resistance to nilotinib 4. To describe acceptability of the study drug formulation;Primary end point(s): 1. Rate of Major Molecular Responder (MMR) by BCRABL RQ-PCR analysis from peripheral blood by 12 months 2. Rate of Major Cytogenetic Responder (MCyR) measured by the percentage of Ph+ metaphases in bone marrow by 12 months 3. Rate of Confirmed Hematological Responder (CHR) measured by complete blood count by 3 months;Timepoint(s) of evaluation of this end point: 1. 12 months 2. 12 months 3. 3 months	—

Measure

Time frame

Main Objective: 1. To assess efficacy of nilotinib in pediatric patients with Ph+ CML CP resistant or intolerant to either imatinib or dasatinib 2. To assess efficacy of nilotinib in pediatric patients with Ph+ CML AP resistant or intolerant to either imatinib or dasatinib 3. To assess efficacy of nilotinib in pediatric patients with newly diagnosed Ph+ CML CP;Secondary Objective: 1. To further characterize efficacy and PK profile of nilotinib in pediatric patients with Ph+ CML 2. To further characterize safety and tolerability of nilotinib in pediatric patients with Ph+ CML 3. To identify emerging signs of resistance to nilotinib 4. To describe acceptability of the study drug formulation;Primary end point(s): 1. Rate of Major Molecular Responder (MMR) by BCRABL RQ-PCR analysis from peripheral blood by 12 months 2. Rate of Major Cytogenetic Responder (MCyR) measured by the percentage of Ph+ metaphases in bone marrow by 12 months 3. Rate of Confirmed Hematological Responder (CHR) measured by complete blood count by 3 months;Timepoint(s) of evaluation of this end point: 1. 12 months 2. 12 months 3. 3 months

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Secondary

Measure	Time frame
Secondary end point(s): 1. Time to response of primary endpoints 2. Duration of response 3. Time to disease progression 4. Overall Survival (OS) 5. Rate of major cytogenetic response (MCyR) and confirmed cytogenetic response (CCR) in all patients for timepoints not already analyzed in primary endpoints 6. BCR-ABL transcript levels determined with standard protocols in peripheral blood and bone marrow for all time points with available data 7. Rate of MMR and CHR in all patients for timepoints not already analyzed in primary endpoints;Timepoint(s) of evaluation of this end point: 1. 1, 3, 6, 9, 12 months 2. up to 24 months 3. up to 24 months 4. up to 24 months or date of last contact in follow-up 5. up to 24 months 6. up to 24 months 7. up to 24 months	—

Measure

Time frame

Secondary end point(s): 1. Time to response of primary endpoints 2. Duration of response 3. Time to disease progression 4. Overall Survival (OS) 5. Rate of major cytogenetic response (MCyR) and confirmed cytogenetic response (CCR) in all patients for timepoints not already analyzed in primary endpoints 6. BCR-ABL transcript levels determined with standard protocols in peripheral blood and bone marrow for all time points with available data 7. Rate of MMR and CHR in all patients for timepoints not already analyzed in primary endpoints;Timepoint(s) of evaluation of this end point: 1. 1, 3, 6, 9, 12 months 2. up to 24 months 3. up to 24 months 4. up to 24 months or date of last contact in follow-up 5. up to 24 months 6. up to 24 months 7. up to 24 months

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Countries

Australia, Austria, Belgium, Canada, France, Hungary, Italy, Japan, Korea, Republic of, Netherlands, New Zealand, Russian Federation, Spain, Thailand, Turkey, United Kingdom, United States

Contacts

Public ContactDrug Regulatory Affairs

NOVARTIS FARMA S.p.A.

info.studiclinici@novartis.com+390296541

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026