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Study to evaluate the safety and efficacy of asciminib in pediatric participants with chronic myeloid leukemia.
EU CTIS ID: 2025-522138-29-00
What this study is testing
To estimate the efficacy of asciminib in pediatric participants with: Newly diagnosed Ph+ CML-CP without known T315I mutation Ph+ CML-CP without known T315I mutation resistant or intolerant to previous TKI Ph+ CML-CP with known T315I mutation irrespective of prior TKI treatment
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Signed informed consent must be obtained prior to participation in the study
- Male or female participants 1and <18 years of age at study enrollment
- Diagnosis of CML-CP (Apperley et al 2025) with cytogenic confirmation of Philadelphia positive (Ph+) chromosome
- For participants with CML-CP newly diagnosed within 3 months of screening
- For participants with CML – CP with high risk of developing resistance or intolerance to previous TKI a. Unfavourable response to TKI is defined following the Apperley et al 2025 Guidelines as: · 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 b. Intolerance to TKI is defined as: · 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); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal) · 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
- Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.
You likely can't join if
- Known second chronic phase 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::ABL mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) at any time prior to study entry.
See the full eligibility criteria
- Signed informed consent must be obtained prior to participation in the study
- Male or female participants 1and <18 years of age at study enrollment
- Diagnosis of CML-CP (Apperley et al 2025) with cytogenic confirmation of Philadelphia positive (Ph+) chromosome
- For participants with CML-CP newly diagnosed within 3 months of screening
- For participants with CML – CP with high risk of developing resistance or intolerance to previous TKI a. Unfavourable response to TKI is defined following the Apperley et al 2025 Guidelines as: · 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 b. Intolerance to TKI is defined as: · 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); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal) · 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
- Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.
- Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants < 16 years of age at the time of screening.
- Known second chronic phase 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::ABL mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) at any time prior to study entry.
The study team makes the final eligibility decision.
Where it's taking place
- Korea, Republic of
- China
- Singapore
- Australia
- Brazil
- United States
- Japan
- Vietnam
- Mexico
- Turkey
- Thailand
- Canada
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 - 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, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Korea, Republic of; China; Singapore; Australia; Brazil; United States and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.