Study on the Effectiveness and Safety of Asciminib in Children with Ph+ Chronic Myeloid Leukemia in Chronic Phase (CML-CP)
Sponsor: Novartis Pharmaceuticals
ClinicalTrials ID:NCT07354074
This clinical trial is testing a medication called asciminib to see if it is safe and effective for children and teenagers with a specific type of leukemia known as Ph+ CML-CP. The study includes those who are newly diagnosed or have not responded well to previous treatments.
Patient Parameters
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Program Overview
The purpose of this study is to evaluate how well the drug asciminib works and how safe it is for children and teenagers (ages 1 to under 18) with Philadelphia chromosome-positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP). The study will include participants who are newly diagnosed or have not responded well to previous treatments, with or without a specific genetic mutation called T315I. Participants will receive either a pediatric or adult formulation of the drug, depending on their age and condition. The study will last until five years after the last participant starts treatment, allowing researchers to monitor safety, growth, development, and effectiveness.
Description
This study involves children and teenagers with Ph+ CML-CP, a type of leukemia. Participants will be divided into three groups: those newly diagnosed without the T315I mutation, those who have not responded well or cannot tolerate previous treatments without the T315I mutation, and those with the T315I mutation regardless of past treatments. There is no set duration for how long participants will receive the study drug, asciminib. The study will continue for five years after the last participant begins treatment to ensure thorough monitoring of safety and effectiveness. Researchers aim to gather enough information on how the drug affects growth, development, and overall health.
Eligibility Criteria
Inclusion Criteria
- Must provide signed consent to participate.
- Boys and girls aged 1 to under 18 years.
- Diagnosed with Ph+ CML-CP, confirmed by specific genetic tests.
- Either newly diagnosed within three months or at high risk of not responding to previous treatments.
- Must show specific genetic markers (BCR::ABL1) that can be measured by standard tests.
- Must be in good general health, with a performance status score of at least 50%.
Exclusion Criteria
- Have had a second phase of CML after progressing to more advanced stages.
- Have previously received a stem cell transplant.
- Plan to undergo a stem cell transplant.
- Have a genetic mutation known to resist the study drug, according to the latest clinical guidelines.
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Rutgers Cancer Institute of New Jersey | 08901 | New Brunswick | New Jersey |
| Columbia University Medical Center New York Presbyterian | 10032 | New York | New York |
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