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 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 (8), 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), Türkiye (4)
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: 60 participants)
Location (Centres): Belgium (2), Denmark (2), France (4), Germany (2), Italy (10), Netherlands (1), Spain (5), Sweden (2), United Kingdom (5)
Dasatinib
A phase II Study of dasatinib in children and adolescents with newly diagnosed chronic phase chronic myelogenous leukemia (CML) or with Ph+ leukemias resistant or intolerant to imatinib
The purpose of this study is to determine whether dasatinib is safe and effective in children and adolescents with newly diagnosed chronic myeloid leukemia (CML), or in children with Ph+ acute lymphoblastic leukemia (ALL), accelerated or blast phases CML who relapse after imatinib or who are resistant or intolerant to imatinib. The side effects of this oral investigational drug in children and adolescents will be evaluated. More information
ClinicalTrials.gov Identifier: NCT00777036
Sponsor: Bristol-Myers Squibb
Status: Active, not recruiting (estimated enrollment: 130 participants)
Location (Centres): United States (35), Argentina (6), Australia (10), Brazil (10), Canada (14), France (11), Germany (4), India (16), Italy (10), Rep. of Korea (4), Mexico (13), Netherlands (3), Romania (3), Russian Federation (6), Singapore (2), South Africa (8), Spain (13), United Kingdom (6)
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: Recruiting (estimated enrollment: 88 participants)
Location (Centres): France (1)