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We are aware about the following current ongoing studies in pediatric CML:

Asciminib Study:
A multi-centre, 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
( Identifier: NCT04925479)

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.

Sponsor: Novartis Pharmaceuticals
Status: Recruiting (estimated enrollment: 34 participants)
More information:

Bosutinib Phase I/II Study:
A phase I/II study of bosutinib in pediatric patients with newly diagnosed chronic phase or resistant/intolerant Ph+ chronic myeloid leukemia
( Identifier: NCT04258943)
Phase 1-2, multicenter, international, single-arm, open-label study designed to identify a recommended dose of bosutinib administered orally once daily in pediatric patients with newly diagnosed chronic phase Ph+ CML (ND CML) and pediatric patients with Ph+CML who have received at least one prior TKI therapy (R/I CML), to preliminary estimate the safety and tolerability and efficacy, and to evaluate the PK of bosutinib in this patient population.

Sponsor: Children's Oncology Group
Status: Recruiting (estimated enrollment: 60 participants)
More information:

Ponatinib Phase I/II Study:
An open-label, single-arm, phase 1/2 study evaluating the safety and efficacy of ponatinib for the treatment of recurrent or refractory leukemias or solid tumors in pediatric patients
( Identifier: NCT03934372)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to < 18 years with advanced leukemias, lymphomas, and solid tumors.

Sponsor: Incyte Biosciences International
Status: Recruiting (estimated enrollment: 60 participants)
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TFR Study
Stopping tyrosine kinase inhibitors (TKI) to assess treatment-free remission (TFR) in pediatric chronic myeloid leukemia chronic-phase (CML-CP)
( Identifier: NCT03817398)

  • To determine the 2-year treatment free remission (TFR) rate of children, adolescents, and young adults with chronic myeloid leukemia - chronic phase (CML-CP) following discontinuation tyrosine kinase inhibitor (TKI).
  • To estimate the re-induction rate and maintenance of molecular remission (BCR-ABL1 =< 0.1%) at 1 year after restarting TKI for children, adolescents, and young adults.

Sponsor: Children's Oncology Group
Status: Recruiting (estimated enrollment: 110 participants)
More information:

International Chronic Myeloid Leukemia Pediatric Study (ICMLPed)
Treatments and outcomes in children and adolescents

Prospective, observational study to describe and characterize CML in a large pediatric cohort of patients
( Identifier: NCT01281735)

Sponsor: Poitiers University Hospital
Status: Recruiting (estimated enrollment: 750 participants)
More information:

Mutation status by Next Generation Sequencing
CML pediatric ITK response acording to molecular identification at diagnosis (CML Piramid)
Observational study to investigate retrospectively on DNA at diagnosis of 88 CP-CML children the mutation status of 64 genes by next generation sequencing and to see if there is an association with the response to TKI treatment.
( Identifier: NCT05605379)

Sponsor: University Hospital Bordeaux
Status: Not yet recruiting (estimated enrollment: 88 participants)
More information:

Please also see the list of published scientific articles about pediatric CML.