Investigating molecular signatures in pediatric chronic myeloid leukemia
CML Pediatric ITK Response According to Molecular Identification at Diagnosis (CML Piramid
This study is trying to find out if certain gene changes in children with chronic myeloid leukemia can help predict how well they will respond to a specific type of cancer treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 88 (estimated) |
| Ages | 6 Years to 18 Years |
| Sex | All |
| Sponsor | University Hospital, Bordeaux Academic / other |
| Locations | 1 site (Bordeaux) |
| Trial ID | NCT05605379 on ClinicalTrials.gov |
What this trial studies
This observational study aims to identify molecular signatures that predict the response to tyrosine kinase inhibitors (TKIs) in children diagnosed with chronic myeloid leukemia (CML). By analyzing DNA and RNA samples from pediatric patients at diagnosis, the study will explore the mutation status of 64 genes and their association with treatment outcomes. The research will also include a comparative analysis of gene expression profiles between patients who achieve complete cytogenetic response and those who do not. The goal is to enhance understanding of the molecular landscape of pediatric CML and improve treatment strategies.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 18 years or younger diagnosed with chronic phase chronic myeloid leukemia and who have a Philadelphia chromosome.
Not a fit: Patients diagnosed with chronic myeloid leukemia in accelerated or blastic phases, or those over 18 years of age, may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more personalized treatment approaches for children with chronic myeloid leukemia, improving their response to therapy.
How similar studies have performed: While there have been limited studies on the molecular status of pediatric CML, this approach of using next-generation sequencing to identify predictive signatures is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age at diagnosis less than or equal to 18 years * Presence of a Philadelphia chromosome detected by cytogenetic analysis (conventional karyotype or Fluorescence In Situ Hybridization (FISH)) and a BCR ::ABL1 transcript e13a2 ou e14a2 * Diagnosis in chronic phase according to the European Leukemia Net (ELN) criteria * First-line treatment with TKIs * Possible pre-treatment with hydroxyurea * DNA available at diagnosis * RNA available for a sub-group patients (8 responders vs 8 no responders) Exclusion Criteria: * Age at diagnosis more than 18 years * Diagnosis in accelerated phase or blastic phase * First line treatment other than TKI
Where this trial is running
Bordeaux
- CHU de Bordeaux, Service Hématologie Biologique — Bordeaux, France (Recruiting)
Study contacts
- Study coordinator: Stéphanie DULUCQ
- Email: stephanie.dulucq@chu-bordeaux.fr
- Phone: 05 57 82 14 98
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.