Study to evaluate the safety and efficacy of asciminib in pediatric participants with chronic myeloid leukemia.
Key facts
- Where
- Debrecen, MagyarországSee the site
- Phase
- 2. fázis
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- What type of cancer has the patient been diagnosed with?
- Has the patient had a stem cell transplant (a transplant of blood-forming cells from a donor or from their own body)?
Where in Magyarország
- University of DebrecenDebrecen, MagyarországToborzás még nem indult
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharma AG
- Trial code
- EU 2025-522138-29-00
- Record updated
- 2026. augusztus 31.
- Original description
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 treatmentRead full original descriptionShow less
A phase II, multicenter, open-label, single arm study to evaluate the safety and efficacy of asciminib in pediatric participants newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation
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
Inclusion: 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.
Exclusion: 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.