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

Managed Access Program (MAP) Cohort Treatment Plan CABL001A02401M to Provide Access to Asciminib for Patients With CML in Chronic Phase (CP) Without Documented T315I Mutation After Failure to or Intolerance of Two Prior TKI OR Patients in CML-CP With Documented T315I Mutation and Without Comparable or Satisfactory Alternative Therapy to Treat the Disease

This program provides access to asciminib for patients with CML in chronic phase (CP) without documented T315I mutation after failure to or intolerance of two prior TKI OR patients in CML-CP with documented T315I mutation and without comparable or satisfactory alternative therapy to treat the disease

Study Overview

• Status: Available
• Conditions: Chronic Myeloid Leukemia
• Intervention / Treatment: Drug: Asciminib

• Study Type: Expanded Access

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111
• Study Contact Backup: Name: MAP requests are initiated by a licensed physician.https:// www.novart is.com/healthcare-professionals/managed-access-programs; Phone Number: 1-888-669-6682; Email: novartis.email@novartis.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 99 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female patients ≥ 18 years of age

• Patients with CML in chronic phase who additionally meet one of the following conditions:

  1. Patient with absence of T315I mutation, who were previously treated with at least two prior TKIs and are relapsed, refractory to or intolerant of TKIs as determined by the treating physician or
  2. Patient with presence of T315I mutation who are failing or intolerant to all available treatments or for whom available treatments are contraindicated
• 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. Patients with mild to severe renal and hepatic impairment are eligible:
• Total bilirubin ≤ 3.0 x ULN AST/ALT any
• Aspartate transaminase (AST) ≤ 5.0 x ULN
• Alanine transaminase (ALT) ≤ 5.0 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 ≥ 30 mL/min as calculated using Cockcroft-Gault formula

Exclusion Criteria:

1. History of hypersensitivity to any drugs or metabolites of similar chemical classes as the product
2. Platelets ≤ 50 x 10^9/L; transient prior therapy related thrombocytopenia (< 50 x 10^9/L for ≤ 30 days prior to asciminib treatment start) is acceptable
3. Active uncontrolled cardiovascular conditions, including ongoing cardiac arrhythmias, congestive heart failure, angina, myocardial infarction within the past 3-6 months
4. A current 12-lead ECG showing signs of QTcF prolongation
5. 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), any cardiac repolarization abnormality or Co-administration of any QT prolonging agents.
6. A history of uncontrolled acute pancreatitis within 1 year prior to treatment start, or chronic active pancreatitis
7. Acute or chronic active non-infectious liver disease
8. Active uncontrolled infection, including pneumonia, requiring systemic therapy or other severe infections
9. Patients with active HBV and HCV infection, and/or with HCV/HBV viral load above the limit of quantification after completing curative/suppressive antiviral treatment.
10. Patients with HIV infection with CD4+ T-cell count ≤ 350 cells/uL, and/or patients who are using concurrent strong CYP3A4 inhibitors (e.g. ritonavir, cobicistat), and cannot be switched to an alternate effective ART regimen before starting asciminib.
11. Significant other uncontrolled medical conditions, such as (but not limited to) uncontrolled diabetes, pulmonary hypertension
12. Participation in a prior investigational study within 30 days prior to enrollment or within 5 half-lives of the investigational product, whichever is longer
13. 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)

14. 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.

15. For US: Sexually active males must use a condom during intercourse while taking the drug, and for 3 days after stopping treatment and should not father a child in this period. This applies to men who are vasectomized and men with male partners in order to prevent delivery of the drug via semen.
16. Not able to understand and to comply with treatment instructions and requirements.

17. Treatment with medications that belong to the below classes and that cannot be discontinued at least one week prior to the start of treatment

    • Strong inducers of CYP3A
    • Strong inhibitors of CYP3A

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start: December 7, 2022
• Primary Completion: December 7, 2022
• Study Completion: December 7, 2022

Study Registration Dates

• First Submitted: April 21, 2020
• First Submitted That Met QC Criteria: April 21, 2020
• First Posted (Actual): April 24, 2020

Study Record Updates

• Last Update Posted (Actual): February 8, 2024
• Last Update Submitted That Met QC Criteria: February 7, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: CML; Asciminib; ABL001; Chronic myeloid leukemia; T315I mutation; CML-CP
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Disease Attributes; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Chronic Disease; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; Tyrosine Kinase Inhibitors; Asciminib
• Other Study ID Numbers: CABL001A02401M

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No