map to provide access to nilotinib, for patients with relapsed or refractory philadelphia chromosome positive acute lymphoblastic leukemia

Conditions: Acute Lymphoblastic Leukemia (ALL)

Summary: the purpose of this cohort treatment plan is to allow access to nilotinib for eligible patients diagnosed with relapsed or refractory philadelphia chromosome positive (ph+) acute lymphoblastic leukemia (all).

currently, preliminary responses of nilotinib monotherapy in adults with refractory or relapsed ph+ all show limited clinical benefit in this population of patients with a high unmet medical need. remissions induced by imatinib are of short duration and resistance to imatinib represents a major clinical challenge. the exact benefit and role of nilotinib in this leukemia remains to be determined and requires further analysis. until further data is available patients with ph+ all should be treated with nilotinib through the individual patient program.. studies to date indicate that nilotinib may provide clinical benefit to ph+ all patients and may represent a novel treatment option for these patients.

Eligibility criteria:
inclusion criteria: the following criteria must be fulfilled for the provision of managed access and will vary depending on the stage of the product lifecycle: * an independent request should be received from the treating physician (in some instances from health authorities, institutions or governments); * the patient to be treated has a serious or life threatening disease or condition, and no comparable or satisfactory alternative therapy is available to monitor or treat the disease or condition; * the patient is not eligible or able to enroll in a clinical trial; * there is a potential patient benefit to justify the potential risk of the treatment use, and the potential risk is not unreasonable in the context of the disease or condition to be treated; * provision of the investigational product will not interfere with the initiation, conduct or completion of a novartis clinical trial or overall development program and * such access provision as described above is allowed as per local laws and regulations. exclusion criteria: patients eligible for inclusion in this treatment plan have to meet all of the following criteria: 1. male or female patients age ≥ 18 years 2. who performance status of 0, 1 or 2 3. relapsed or refractory ph+ all 4. imatinib (or dasatinib) must be discontinued at least 5 days prior to beginning therapy 5. normal organ, electrolyte and marrow functions as described below: * absolute neutrophil count (anc) ≥ 1.0 x 1000000000/l * platelets 50 x 1000000000/l * potassium ≥ lln (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of nilotinib * total calcium (corrected for serum albumin) ≥ lln * magnesium ≥ lln or corrected to within normal limits with supplements prior to the first dose of nilotinib medication * ast and alt ≤ 2.5 x uln or ≤ 5.0 x ul:n if considered due to tumor * alkaline phosphatase ≤ 2.5 x uln * serum bilirubin ≤ 1.5 x uln * serum amylase and lipase ≤ 1.5 x uln

Keywords: nilotinib acute lymphoblastic leukemia all philadelphia chromosome positive ph+