Official Title
Managed Access Program to Provide Access to Nilotinib, for Patients With Imatinib-intolerant and/or Resistant Ph+ Chronic Myelogenous Leukemia (CML) in Chronic Phase or CML in Accelerated Phase
Brief Summary

The purpose of this Cohort Treatment Plan is to allow access to Nilotinib for eligible patients diagnosed with imatinib-intolerant and/or resistant Philadelphia Chromosome positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) or Chronic Myelogenous Leukemia in Accelerated Phase (CML-AP). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Chronic Myelogenous Leukemia (CML)

Drug: Nilotinib
The recommended dosing of nilotinib is 400 mg orally twice daily

Eligibility Criteria

Inclusion Criteria: 1. Age ≥ 18 years 2. One of the diagnoses listed below - Imatinib resistant Philadelphia chromosome positive CML in chronic phase and the presence of the following criteria: - < 15% blasts in peripheral blood or bone marrow - < 30% blasts plus promyelocytes in peripheral blood and bone marrow - < 20 % basophils in the peripheral blood - ≥ 100 x 10^9 / L (≥ 100,000/mm3) platelets - No evidence of extramedullary leukemic involvement, with the exception of liver or spleen - Imatinib resistant Philadelphia chromosome positive CML in accelerated phase defined as never in blast crisis before starting treatment with one or more of the following criteria present within 4 weeks prior to beginning treatment: - ≥ 15% but < 30% blasts in blood or bone marrow - ≥ 30% blasts plus promyelocytes in peripheral blood or bone marrow (providing that 450 and electrolytes are not within normal ranges, electrolytes should be corrected and then the patient rescreened for QTc Myocardial infarction within 12 months prior to starting nilotinib Other clinically significant heart disease (e.g. unstable angina, congestive heart failure or uncontrolled hypertension). 4. any medications that prolong the QT interval and CYP3A4 inhibitors if the treatment cannot Use of be either safely discontinued or switched to a different medication prior to starting treatment. Please see for a comprehensive list of agents that prolong the QT interval 5. Severe and/ or uncontrolled concurrent medical disease that in the opinion of the treating physicians could cause unacceptable safety risks or compromise compliance with the compassionate use treatment (e.g. impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of nilotinib, ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection, uncontrolled diabetes, ) 6. Patients who have undergone major surgery ≤ 2 weeks prior to Visit 1 or who have not recovered from side effects of such surgery 7. Known Cytopathologically confirmed Central Nervous System infiltration. 8. Use of therapeutic warfarin. 9. Acute or chronic liver or renal disease considered unrelated to tumor.

10. Treatment with any hematopoietic colony-stimulating growth factors ≤ 1 week prior to starting Nilotinib. Erythropoietin is allowed.

11. Patient who has not recovered from side effects of prior chemotherapy, immunotherapy, other investigational drugs, wide field radiotherapy, or major surgery. Patient who has received imatinib < 5 days prior to starting nilotinib or has not recovered from side effects of therapy. Hydroxyurea is permitted during the first 28 days of treatment (up to 5 g/day) for a maximum of 7 days.

12. Patient with a history of another primary malignancy that is currently clinically significant or requires active intervention.

13. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL).

14. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods. The two methods can be a double barrier method or a barrier method plus a hormonal method.

Adequate barrier methods of contraception include: diaphragm, condom (by the partner),
intrauterine device (copper or hormonal), sponge or spermicide. Hormonal contraceptives
include any marketed contraceptive agent that includes an estrogen and/or a progestational

Eligibility Gender
Eligibility Age
Minimum: 18 Years~Maximum: 99 Years

Novartis Pharmaceuticals

Novartis Pharmaceuticals

Novartis Pharmaceuticals
NCT Number
Imatinib resistant
Philadelphia Chromosome positive
Chronic Myelogenous Leukemia in Chronic Phase
Chronic Myelogenous Leukemia in Accelerated Phase
MeSH Terms
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive