Chronic Myelogenous Leukemia study: Asciminib single agent
Official title Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP
ClinicalTrials.gov ID: NCT07354074
What this study is testing
What is Asciminib?
an investigational oral tablet being studied as a potential treatment for chronic myelogenous leukemia (CML) in children and adults. is designed to selectively inhibit the BCR‑ABL1 protein by binding to its myristoyl pocket, blocking the signal that drives leukemia cell growth.
Also referred to as ABL001.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study will find out how well asciminib works and how safe it is for kids with this type of leukemia.
- Who it's for
- Children ages 1 to under 18 who have newly diagnosed or resistant/intolerant Philadelphia‑positive chronic myeloid leukemia in chronic phase, with or without the T315I mutation.
- What's involved
- Participants will take asciminib either as a weight‑based pediatric dose taken with food or as an adult dose taken on an empty stomach, and will have regular clinic visits and tests to monitor safety and response.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 18
You may be able to join if
- Signed informed consent must be obtained prior to participation in the study.
- Male or female participants 1 and \< 18 years of age at study enrollment
- Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
- At three months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS (if confirmed within 1-3 months)
- At six months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS
You likely can't join if
- Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planned to undergo allogeneic hematopoietic stem cell transplantation
- Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international...
See the full eligibility criteria
- Signed informed consent must be obtained prior to participation in the study.
- Male or female participants 1 and \< 18 years of age at study enrollment
- Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
- At three months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS (if confirmed within 1-3 months)
- At six months after the initiation of therapy: BCR::ABL1 ratio \> 10% IS
- At twelve months after initiation of therapy: BCR::ABL1 ratio \> 1% IS
- At any time loss of previous response
- At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results
- Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction)
- Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI 6\.
- Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planned to undergo allogeneic hematopoietic stem cell transplantation
- Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g.
The study team makes the final eligibility decision.
Where it is enrolling
- Chicago, Illinois, United States
- New Brunswick, New Jersey, United States
- New York, New York, United States
- Cincinnati, Ohio, United States
- Seattle, Washington, United States
- Brisbane, Queensland, Australia
- North Adelaide, South Australia, Australia
- Rio de Janeiro, Rio de Janeiro, Brazil
- Barretos, São Paulo, Brazil
- São Paulo, São Paulo, Brazil
- Edmonton, Alberta, Canada
- Montreal, Quebec, Canada
- Zhengzhou, Henan, China
- Tianjin, China
- Marseille, France
- Paris, France
- Poitiers, France
- Strasbourg, France
- Frankfurt am Main, Hesse, Germany
- Erlangen, Germany
+ 16 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Chicago, Illinois, United States; New Brunswick, New Jersey, United States; New York, New York, United States; Cincinnati, Ohio, United States; Seattle, Washington, United States; Brisbane, Queensland, Australia and 30 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.