MAP to Provide Access to Nilotinib, for Patients With Relapsed or Refractory Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia


Study First Submitted Date 2020-09-16
Study First Posted Date 2020-09-23
Last Update Posted Date 2022-08-09
Verification Month Year May 2022
Verification Date 2022-05-31
Last Update Posted Date 2022-08-09

Detailed Descriptions

Sequence: 20832024
Description 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.


Sequence: 52454314
Name Acute Lymphoblastic Leukemia (ALL)
Downcase Name acute lymphoblastic leukemia (all)

Id Information

Sequence: 40361231
Id Source org_study_id
Id Value CAMN107A2412


Sequence: 52764407
Intervention Type Drug
Name Nilotinib
Description The compassionate use dose of nilotinib is 400 mg orally twice daily and should not be taken with food


Sequence: 80252941 Sequence: 80252937 Sequence: 80252938 Sequence: 80252939 Sequence: 80252940
Name Ph+ Name Nilotinib Name Acute lymphoblastic leukemia Name ALL Name Philadelphia chromosome positive
Downcase Name ph+ Downcase Name nilotinib Downcase Name acute lymphoblastic leukemia Downcase Name all Downcase Name philadelphia chromosome positive

Browse Conditions

Sequence: 194567971 Sequence: 194567972 Sequence: 194567973 Sequence: 194567974 Sequence: 194567975 Sequence: 194567976 Sequence: 194567977 Sequence: 194567978 Sequence: 194567979 Sequence: 194567980 Sequence: 194567981 Sequence: 194567982 Sequence: 194567983
Mesh Term Leukemia Mesh Term Precursor Cell Lymphoblastic Leukemia-Lymphoma Mesh Term Leukemia, Lymphoid Mesh Term Philadelphia Chromosome Mesh Term Neoplasms by Histologic Type Mesh Term Neoplasms Mesh Term Lymphoproliferative Disorders Mesh Term Lymphatic Diseases Mesh Term Immunoproliferative Disorders Mesh Term Immune System Diseases Mesh Term Translocation, Genetic Mesh Term Chromosome Aberrations Mesh Term Pathologic Processes
Downcase Mesh Term leukemia Downcase Mesh Term precursor cell lymphoblastic leukemia-lymphoma Downcase Mesh Term leukemia, lymphoid Downcase Mesh Term philadelphia chromosome Downcase Mesh Term neoplasms by histologic type Downcase Mesh Term neoplasms Downcase Mesh Term lymphoproliferative disorders Downcase Mesh Term lymphatic diseases Downcase Mesh Term immunoproliferative disorders Downcase Mesh Term immune system diseases Downcase Mesh Term translocation, genetic Downcase Mesh Term chromosome aberrations Downcase Mesh Term pathologic processes
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48582299
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Novartis Pharmaceuticals


Sequence: 30927884
Gender All
Minimum Age 18 Years
Maximum Age N/A
Healthy Volunteers No
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: Male or Female patients age ≥ 18 years WHO Performance Status of 0, 1 or 2 Relapsed or refractory Ph+ ALL Imatinib (or dasatinib) must be discontinued at least 5 days prior to beginning therapy 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
Adult True
Child False
Older Adult True

Calculated Values

Sequence: 254204375
Registered In Calendar Year 2020
Were Results Reported False
Has Single Facility False
Minimum Age Num 18
Minimum Age Unit Years

Responsible Parties

Sequence: 29040262
Responsible Party Type Sponsor