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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 59

Inclusion Criteria

Patients eligible for inclusion in this study have to meet all of the following criteria:
1. Male or female patients from 1 year of age to less than 18 years of age at study entry
2. Newly diagnosed Ph+ CML-CP or Ph+ CML-CP or AP resistant or intolerant to either imatinib or dasatinib (as defined in the protocol)
3. Performance status: Karnofsky =50% for patients >10 years of age and Lansky =50 for patients =10 years of age.
4. Adequate renal, hepatic and pancreatic function (as defined in the protocol)
5. Patients must have potassium, magnesium, phosphorus and total calcium values =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 prior to any screening procedures. However, if requested tests were performed as part of clinical practice within the window required per protocol, this data can be used provided all parameters required per protocol were verified.
Are the trial subjects under 18? yes
Number of subjects for this age range: 59
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

Patients eligible for this study must not meet any of the following criteria:
1. Female patients who are : (a) pregnant, (b) of childbearing potential without a negative hCG pregnancy test prior to baseline and (c) female of childbearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of highly effective contraception
2. 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
3. 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.
4. Acute or chronic liver, pancreatic or severe renal disease considered unrelated to CML
5. History of pancreatitis within 12 months of prior to starting study drug or past medical history of chronic pancreatitis
6. Active or systemic bacterial, fungal, or viral infection as documented by positive cultures, radiological imaging techniques, or septic shock syndrome. Such infections should have been resolved for =2 weeks before including the patient in the study
7. Impaired cardiac function as defined in the protocol
8. Patients with documented T315I mutation in BCR-ABL
9. Previous treatment with more than one TKI for imatinib or dasatinib resistant/intolerant Ph+ CML patients. Previous treatment with any TKI for newly diagnosed Ph+ CML patients is not permitted unless the patient has received imatinib for less than 2 weeks prior to the first dose of study drug and discontinued at least 5 days prior to the first dose of nilotinib.
10. Patients who have received myelosuppressive chemotherapy less than within 3 weeks, imatinib within 5 days, or dasatinib within 3 days prior to the first dose of study drug
11. Patients who have not recovered from all acute toxicities from all prior myelosuppressive chemotherapy prior to starting study drug
12. Patients receiving hydroxyurea greater than 21 days for the treatment of Ph+ CML either prior to initiation of nilotinib or with maximum duration planned to exceed one week post initiation of nilotinib
13. Patients who have received hematopoietic growth factors within 7 days prior to starting study drug
14. Patients who have received Pegfilgrastim (Neulasta®) within 14 days prior to starting study drug
15. 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.
16. In case of radiation therapy: less than 2 weeks if local palliative, less than 3 months after total body irradiation (TBI), or craniospinal radiation therapy or if at least 50% radiation of pelvis; less than 6 weeks after other substantial BM radiation
17. Patients with known Hepatitis B, Hepatitis C, or HIV infection
18. Patients who, in the opinion of the investigator, are unlikely to comply with the protocol or safety monitoring requirements
19. Patients who are breast feeding
20. Patients who have a known hypersensitivity to the active ingredient or any of the excipients including lactose
21. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, mal-absorption,

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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