RECRUITING

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

Conditions

Myeloid Leukemia, Philadelphia Positive Asciminib pediatric patients Philadelphia chromosome positive chronic myeloid leukemia in chronic phase Ph+ CML-CP ABL001

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2021-12-27

Study Completion:2031-11-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04925479

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Year to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* 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.	* 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.

Inclusion Criteria

Exclusion Criteria

* 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.

* 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.

Contacts and Locations

Study Contact

Novartis Pharmaceuticals

CONTACT

1-888-669-6682

novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Principal Investigator

Novartis Pharmaceuticals

STUDY_DIRECTOR

Novartis Pharmaceuticals

Study Locations (Sites)

Dana Farber Cancer Institute

Boston, Massachusetts, 02215

United States

University of Mississippi Medical Center

Jackson, Mississippi, 39216-4505

United States

Columbia University Medical Center New York Presbyterian

New York, New York, 10032

United States

Cinn Children Hosp Medical Center

Cincinnati, Ohio, 45229-3039

United States

Uni Of Texas MD Anderson Cancer Ctr

Houston, Texas, 77024

United States

University Of Utah

Salt Lake City, Utah, 84132

United States

Collaborators and Investigators

Sponsor: Novartis Pharmaceuticals

Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-12-27

Study Completion Date2031-11-01

Study Record Updates

Study Start Date2021-12-27

Study Completion Date2031-11-01

Terms related to this study

Keywords Provided by Researchers

Asciminib
pediatric patients
Philadelphia chromosome positive chronic myeloid leukemia in chronic phase
Ph+ CML-CP
ABL001

Additional Relevant MeSH Terms

Myeloid Leukemia, Philadelphia Positive