RecruitingPhase 1 / Phase 2

Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
1 Year to 17 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the dose and safety of asciminib in children ages 1 to 17 with chronic myeloid leukemia (CML), a type of blood cancer. It is for children who have already taken at least one targeted cancer drug called a TKI.

The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML in the pediatric population has the same pathogenesis, similar clinical characteristics and progression pattern as in adults.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Ages 1 to 17 for the child form of the drug. For the adult form, ages 14 to 17 and weight at least 40 kg (about 88 pounds).
  • Have Philadelphia chromosome-positive (Ph+) CML in its chronic, or slow-growing, phase. Blood and bone marrow tests must meet the study’s limits for certain blood cells.
  • Have no leukemia outside the bone marrow, except for an enlarged liver or spleen.
  • Have taken at least one TKI. The most recent TKI must have failed to work well enough or caused side effects that could not be tolerated.
  • Score at least 50 on the age-based study scale for daily activity and ability to care for yourself.
  • Have kidneys, liver, pancreas, and heart that work well enough for the study.
  • Have normal blood levels of minerals called electrolytes, or have them corrected with supplements before the first dose.
  • Have a specific BCR::ABL1 gene signal (e14a2 or e13a2) that the study’s standard test can measure.

Who can't join

  • Have the T315I gene change or another BCR::ABL gene change known to resist the study drug.
  • Have CML that returned to the chronic phase after progressing to a faster-growing phase.
  • Have had a blood-forming stem cell transplant, or plan to have one using donor cells.
  • Have certain heart or heart electrical activity problems.
  • Have a severe or uncontrolled illness that could make the study unsafe or make it hard to follow study rules.
  • Have had sudden swelling of the pancreas in the past year, long-term swelling of the pancreas, or sudden or long-term liver disease.
  • Have a stomach or bowel problem that could greatly change how the body absorbs the study drug.
  • Are pregnant or breastfeeding.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * Male or female participants: * Pediatric formulation group: ≥ 1 and less than 18 years of age at study entry. * Adult formulation group: ≥ 14 and less than 18 years of age and body weight of ≥ 40 kg at study entry. * Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test. 1. \< 15% blasts in peripheral blood and bone marrow 2. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow 3. \< 20% basophils in the peripheral blood 4. Neutrophils ≥ 1.5 x 10\^9/L (or WBC ≥ 3 x 10\^9/L if neutrophils are not available) and platelet count ≥ 100 x 10\^9/L 5. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly * Prior treatment with a minimum of one TKI * Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening. * Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients \< 16 years of age at the time of screening * Participants must have adequate renal, hepatic, pancreatic and cardiac function * Participants must have electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication: * Evidence of typical BCR::ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification. Exclusion Criteria: * Known presence of the T315I mutation prior to study entry or a BCR::ABL mutation with known resistance to study treatment any time prior to study entry. * Known second chronic phase of CML after previous progression to AP/BC. * Previous treatment with a hematopoietic stem-cell transplantation. * Patient planning to undergo allogeneic hematopoietic stem cell transplantation. * Cardiac or cardiac repolarization abnormality * Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol * History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis. * History of acute or chronic liver disease. * Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug * Pregnant or nursing (lactating) females. Other protocol-defined inclusion/exclusion may apply.

Study sites (8)

IN

MA

MS

NY

OH

PA

TX

UT