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Tests treatment safety and results for Myeloid Leukemia, Philadelphia Positive
Official title Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
ClinicalTrials.gov ID: NCT04925479
What this study is testing
What is Asciminib Pediatric formulation group?
Asciminib Pediatric formulation group is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for myeloid leukemia, philadelphia positive.
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 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.
- 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 17
You may be able to join if
- 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...
- \< 15% blasts in peripheral blood and bone marrow
You likely can't join if
- 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...
- 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...
See the full eligibility 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...
- \< 15% blasts in peripheral blood and bone marrow
- \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
- \< 20% basophils in the peripheral blood
- 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
- 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.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Indianapolis, Indiana, United States
- Boston, Massachusetts, United States
- Jackson, Mississippi, United States
- New York, New York, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- Hangzhou, Zhejiang, China
- Beijing, China
- Shanghai, China
- Tianjin, China
- Bordeaux, France
- Lille, France
- Paris, France
- Poitiers, France
- Erlangen, Germany
- Essen, Germany
- Hamburg, Germany
- Athens, Greece
+ 18 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 - 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 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Indianapolis, Indiana, United States; Boston, Massachusetts, United States; Jackson, Mississippi, United States; New York, New York, United States; Cincinnati, Ohio, United States; Philadelphia, Pennsylvania, United States and 32 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 ClinicalTrials.gov; the study team decides eligibility.