Study to Determine the Dose and Safety of Asciminib in Children with Chronic Myeloid Leukemia

Sponsor: Novartis Pharmaceuticals

Sponsor score: 60
Inactive

ClinicalTrials ID:NCT04925479

Provider View

This clinical trial is testing the safety and appropriate dose of the drug asciminib in children and teenagers with chronic myeloid leukemia (CML) who have been treated with other medications before. The study aims to see if the drug works as well in children as it does in adults.

Patient Parameters

Program Overview

The purpose of this study is to find the right dose and ensure the safety of asciminib for children and teenagers (ages 1 to under 18) with chronic myeloid leukemia (CML) who have already tried other treatments. Researchers believe that CML in children behaves similarly to CML in adults, so they are using information from adult studies to help guide this research.

Description

This study is focused on children and teenagers with a specific type of leukemia called Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). These patients have already been treated with at least one other medication known as a Tyrosine Kinase Inhibitor (TKI).

The main goal is to understand how the drug asciminib behaves in the body of young patients and to find the right dose that matches the exposure seen in adults taking 40 mg twice a day. The study will include at least 30 participants divided into two age groups: 1 to under 12 years and 12 to under 18 years.

The study is divided into three parts:

  • Part 1: A small group of 4-6 participants will help determine the initial dose based on body weight.
  • Part 2: Once the dose is set, more participants will join until there are at least 20 in total, including those from Part 1.
  • Part 3: After safety checks, a once-daily dose will be tested in 10 more participants.

The study will last up to 5 years, and participants who benefit from the treatment can continue until the end. The main analysis will occur after participants have been on the treatment for at least one year.

Eligibility Criteria

Inclusion Criteria

  • Children and teenagers aged 1 to under 18 for the pediatric group, and 14 to under 18 with a weight of at least 40 kg for the adult group.
  • Must have a specific type of leukemia (Ph+ CML-CP) with certain blood test results:
    • Less than 15% blasts in blood and bone marrow
    • Less than 30% combined blasts and promyelocytes
    • Less than 20% basophils in blood
    • Adequate neutrophil and platelet counts
    • No leukemia outside the bone marrow, except for liver or spleen enlargement
  • Previously treated with at least one TKI and either did not respond or could not tolerate it.
  • Good overall health with a performance status score of at least 50%.
  • Normal kidney, liver, pancreas, and heart function.
  • Normal electrolyte levels or corrected before starting the study.
  • Presence of specific genetic markers (BCR-ABL1) that can be measured.

Exclusion Criteria

  • Known presence of a specific mutation (T315I) before joining the study.
  • Second phase of CML after previous progression.
  • Previous stem cell transplant or planning to have one.
  • Heart problems or abnormal heart rhythms.
  • Severe or uncontrolled medical conditions that could pose safety risks.
  • History of pancreatitis or liver disease.
  • Digestive issues that could affect drug absorption.
  • Pregnant or breastfeeding women.

Other criteria may apply as defined by the study protocol.

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