Research and Clinical Trials on Pediatric CML
We are aware of the following current ongoing studies in pediatric CML:
Molecular signature to predict response to TKI
CML pediatric ITK response according to molecular identification at diagnosis (CML Piramid)
This observational study investigates 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: Recruiting (estimated enrollment: 88 participants)
Location (Centres): France (1)
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: Not yet recruiting (estimated enrollment: 50 participants)
Location (Centres): Location data not yet available
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: 44 participants)
Location (Centres): United States (9), China (4), France (4), Germany (3), Greece (1), Hungary (1), Italy (4), Japan (3), Rep. of Korea (2), Netherlands (1), Poland (1), Russian Federation (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 (2), United Kingdom (4)
Treatment-Free Remission
Treatment-free remission (TFR) in CML Patients (CML-CP) Study
This multicenter, prospective observational registry study investigates real-world treatment strategies to increase deep molecular response (DMR) rates in patients with chronic-phase chronic myeloid leukemia (CML-CP), with the goal of improving the likelihood of achieving treatment-free remission (TFR). It follows patients for at least five years to assess molecular responses during therapy and the 2-year TFR rate after discontinuation of tyrosine kinase inhibitors. More information
ClinicalTrials.gov Identifier: NCT05440747
Sponsor: Guangdong Provincial People's Hospital, gdph_wjy21@gd.gov.cn
Status: Not yet recruiting (estimated enrollment: 203 participants)
Location (Centres): Location data not yet available
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: Recruiting (estimated enrollment: 88 participants)
Location (Centres): France (1)