Research and Clinical Trials on Pediatric CML

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

Asciminib

Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP

This Phase II, open-label, single-arm study evaluates the safety and efficacy of asciminib in children aged 1 to under 18 years with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), including both newly diagnosed and previously treated patients, with or without the T315I mutation. It assesses weight-based pediatric or adult formulations over long-term follow-up to better understand treatment outcomes, safety, and effects on growth and development. More Information

ClinicalTrials.gov Identifier: NCT07354074

Sponsor: Novartis Pharmaceuticals, novartis.email@novartis.com

Status: Recruiting (estimated enrollment: 50 participants)

Location (Centres): USA (4), Australia (2), Brazil (3), Canada (2), China (2), France (4), Germany (2), Italy (1), Japan (3), Netherlands (1), Poland (2), South Korea (2), Spain (2)

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

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. More Information

ClinicalTrials.gov Identifier: NCT04925479

Sponsor: Novartis Pharmaceuticals, novartis.email@novartis.com

Status: Recruiting (estimated enrollment: 34 participants)

Location (Centres): United States (8), China (4), France (4), Germany (3), Greece (1), Hungary (1), Italy (4), Japan (3), Netherlands (1), Poland (1), Russian Federation (2), South Korea (2), Thailand (3), Turkey (2)

Bosutinib

A phase I/II study of bosutinib in pediatric patients with newly diagnosed chronic phase or resistant/intolerant Ph+ chronic myeloid leukemia

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. More Information

ClinicalTrials.gov Identifier: NCT04258943

Sponsor: Children's Oncology Group

Status: Active, not recruiting (estimated enrollment: 60 participants)

Location (Centres): United States (45), France (3), Germany (1), Italy (3), Netherlands (2), Spain (2), Switzerland (1), United Kingdom (2)

Ponatinib

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.

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. More information

ClinicalTrials.gov Identifier: NCT03934372

Sponsor: Incyte Biosciences International, globalmedinfo@incyte.com

Status: Recruiting (estimated enrollment: 70 participants)

Location (Centres): Belgium (1), France (4), Italy (9), Netherlands (1), Spain (4), Sweden (1), United Kingdom (3)

Treatment-Free Remission

Stopping tyrosine kinase inhibitors (TKI) to assess treatment-free remission (TFR) in pediatric chronic myeloid leukemia chronic-phase (CML-CP)

The purpose of this study is 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), and 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. More information

ClinicalTrials.gov Identifier: NCT03817398

Sponsor: Children's Oncology Group

Status: Active, not recruiting (estimated enrollment: 110 participants)

Location (Centres): United States (157), Australia (5), Canada (10), Puerto Rico (1)

ICMLPed

International Chronic Myeloid Leukemia Pediatric Study (ICMLPed)

The purpose of the study is to describe and characterize CML in a large pediatric cohort of patients. More Information

ClinicalTrials.gov Identifier: NCT01281735

Sponsor: Poitiers University Hospital, f.millot@chu-poitiers.fr

Status: Recruiting (estimated enrollment: 750 participants)

Location (Centres): Australia (1), Austria (1), Belarus (1), Belgium (1), Brazil (1), Chile (1), China (1), Croatia (1), Czechia (1), Denmark (1), France (1), Germany (1), Greece (1), Japan (1), Lebanon (1), Netherlands (1), Poland (1), Slovakia (1), Spain (1), Türkiye (1)

Mutation status by Next Generation Sequencing

CML Pediatric ITK Response According 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. More information

ClinicalTrials.gov Identifier: NCT05605379

Sponsor: University Hospital Bordeaux, stephanie.dulucq@chu-bordeaux.fr

Status: Unknown status (estimated enrollment: 88 participants)

Location (Centres): France (1)