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Managed Access Program (MAP)* to Provide Access to Asciminibfor Patients With CML in Chronic Phase

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ClinicalTrials.gov Identifier: NCT04360005
Expanded Access Status : Available
First Posted : April 24, 2020
Last Update Posted : April 24, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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
Chronic Myeloid Leukemia Drug: Asciminib

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Study Type : Expanded Access
Official Title: Managed Access Program (MAP) to Provide 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



Intervention Details:
  • Drug: Asciminib
    Asciminib 20 or 40 mg strength tablets will be administered orally twice-daily, without food

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).

  • Male or female patients ≥ 18 years of age
  • 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
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
  • Patient is deemed by the treating physician to have the initiative and means to be compliant with treatment and follow-up requested
  • Adequate end organ function, within 14 days before the first dose of asciminib treatment, as defined by:
  • 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
  • Aspartate transaminase (AST) ≤ 3.0 x ULN
  • Alanine transaminase (ALT) ≤ 3.0 x ULN
  • 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
  • Alkaline phosphatase ≤ 2.5 x ULN
  • Creatinine clearance ≥ 50 mL/min as calculated using Cockcroft-Gault formula
  • 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:

  • Patient must not have comparable or satisfactory alternative therapy available to treat the disease
  • History of hypersensitivity to any drugs or metabolites of similar chemical classes as the product
  • Platelets ≤ 50 x 109/L; transient prior therapy related thrombocytopenia (< 50 x 109/L for ≤ 30 days prior to asciminib treatment start) is acceptable
  • Active uncontrolled cardiovascular conditions, including ongoing cardiac arrhythmias, congestive heart failure, angina, myocardial infarction within the past 3-6 months
  • A current 12-lead ECG showing signs of QTcF prolongation
  • 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
  • A history of uncontrolled acute pancreatitis within 1 year prior to treatment start, or chronic active pancreatitis
  • Acute or chronic active liver disease
  • Active uncontrolled infection, including pneumonia, requiring systemic therapy or other severe infections
  • Known history of Human Immunodeficiency Virus (HIV), chronic Hepatitis B (HBV), or chronic Hepatitis C (HCV) infection
  • Significant other uncontrolled medical conditions, such as (but not limited to) uncontrolled diabetes, pulmonary hypertension
  • Participation in a prior investigational study within 30 days prior to enrollment or within 5 half-lives of the investigational product, whichever is longer
  • 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)
  • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are:
  • Women whose sexual orientation precludes intercourse with a male partner
  • Women whose partners have been sterilized by vasectomy or other means
  • 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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04360005


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04360005    
Other Study ID Numbers: CABL001A02401M
First Posted: April 24, 2020    Key Record Dates
Last Update Posted: April 24, 2020
Last Verified: April 2020
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Chronic myeloid leukemia
CML
T315I mutation
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia
Neoplasms by Histologic Type
Neoplasms
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases