Back to resultsMAP to Provide Access to Nilotinib, for Patients With Relapsed or Refractory Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
Primary Purpose
Acute Lymphoblastic Leukemia (ALL)
Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
Nilotinib
Sponsored by
About this trial
This is an expanded access trial for Acute Lymphoblastic Leukemia (ALL) focused on measuring Nilotinib, Acute lymphoblastic leukemia, ALL, Philadelphia chromosome positive, Ph+
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:
- 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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT04559555
First Posted
September 16, 2020
Last Updated
August 5, 2022
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT04559555
Brief Title
MAP to Provide Access to Nilotinib, for Patients With Relapsed or Refractory Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
Official Title
Managed Access Program (MAP) to Provide Access to Nilotinib, for Patients With Relapsed or Refractory Philadelphia Chromosome Positive (Ph+) Acute Lymphoblastic Leukemia (ALL)
Study Type
Expanded Access
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
5. Study Description
Brief 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).
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Lymphoblastic Leukemia (ALL)
Keywords
Nilotinib, Acute lymphoblastic leukemia, ALL, Philadelphia chromosome positive, Ph+
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Nilotinib
Intervention Description
The compassionate use dose of nilotinib is 400 mg orally twice daily and should not be taken with food
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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:
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
12. IPD Sharing Statement
Learn more about this trial
MAP to Provide Access to Nilotinib, for Patients With Relapsed or Refractory Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
We'll reach out to this number within 24 hrs