Managed Access Program (MAP)* to Provide Access to Asciminib for Patients With CML in Chronic Phase
Study Details
Study Description
Brief Summary
This program provides access to asciminib for patients with CML in chronic phase, with or without documented T315I mutation, without comparable or satisfactory alternative therapy to treat the disease
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
Study Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Inclusion Criteria:
Patients eligible for inclusion in this treatment plan have to meet all of the following criteria:
Written patient informed consent must be obtained prior to start of treatment, including all necessary consents (or their legal representatives, where applicable).
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Male or female patients ≥ 18 years of age
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Patients with CML in chronic phase with or without T315I mutation, who were previously treated with all commercially available tyrosine kinase inhibitors (TKIs) for the specific market and are relapsed, refractory to or intolerant of TKIs as determined by the treating physician or for whom the treatment with one or more available TKIs is contraindicated based on approved label
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Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
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Patient is deemed by the treating physician to have the initiative and means to be compliant with treatment and follow-up requested
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Adequate end organ function, within 14 days before the first dose of asciminib treatment, as defined by:
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Total bilirubin ≤ 1.5 x ULN except for patients with Gilbert's syndrome who may only be included if total bilirubin ≤ 3.0 x ULN or direct bilirubin ≤ 1.5 x ULN
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Aspartate transaminase (AST) ≤ 3.0 x ULN
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Alanine transaminase (ALT) ≤ 3.0 x ULN
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Serum lipase ≤ 1.5 x ULN. For serum lipase > ULN - ≤ 1.5 x ULN, value should be considered not clinically significant and not associated with risk factors for acute pancreatitis
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Alkaline phosphatase ≤ 2.5 x ULN
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Creatinine clearance ≥ 50 mL/min as calculated using Cockcroft-Gault formula
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Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2
Exclusion Criteria:
Patients eligible for this treatment plan must not meet any of the following criteria:
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Patient must not have comparable or satisfactory alternative therapy available to treat the disease
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History of hypersensitivity to any drugs or metabolites of similar chemical classes as the product
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Platelets ≤ 50 x 109/L; transient prior therapy related thrombocytopenia (< 50 x 109/L for ≤ 30 days prior to asciminib treatment start) is acceptable
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Active uncontrolled cardiovascular conditions, including ongoing cardiac arrhythmias, congestive heart failure, angina, myocardial infarction within the past 3-6 months
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A current 12-lead ECG showing signs of QTcF prolongation
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A history of active long QT syndrome or risk of Torsades de pointes (TdP) and risk factors for TdP, such as uncorrected hypokalemia or hypomagnesemia (patient can be corrected before start of asciminib treatment), or any cardiac repolarization abnormality
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A history of uncontrolled acute pancreatitis within 1 year prior to treatment start, or chronic active pancreatitis
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Acute or chronic active liver disease
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Active uncontrolled infection, including pneumonia, requiring systemic therapy or other severe infections
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Known history of Human Immunodeficiency Virus (HIV), chronic Hepatitis B (HBV), or chronic Hepatitis C (HCV) infection
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Significant other uncontrolled medical conditions, such as (but not limited to) uncontrolled diabetes, pulmonary hypertension
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Participation in a prior investigational study within 30 days prior to enrollment or within 5 half-lives of the investigational product, whichever is longer
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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)
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Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are:
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Women whose sexual orientation precludes intercourse with a male partner
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Women whose partners have been sterilized by vasectomy or other means
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Using a highly effective method of birth control (i.e. one that results in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, and some intrauterine devices (IUDs); periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) is not acceptable Reliable contraception should be maintained throughout the period of treatment and for 14 days after treatment discontinuation.
Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or six months of spontaneous amenorrhea with serum FSH levels > 40 mIU/mL or have had surgical bilateral oophorectomy (with or without hysterectomy) at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
- Not able to understand and to comply with treatment instructions and requirements
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Novartis Pharmaceuticals
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CABL001A02401M