Actively Recruiting
CML Pediatric ITK Response According to Molecular Identification at Diagnosis (CML Piramid)
Led by University Hospital, Bordeaux · Updated on 2024-08-14
88
Participants Needed
1
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating chronic myeloid leukemia (CML) in children, a rare condition that differs in disease features and treatment response compared to adults. The study focuses on understanding molecular factors that might predict how children respond to tyrosine kinase inhibitors (TKIs), a revolutionary treatment for CML. Since resistance to TKIs can occur and is not well understood in children, this research aims to identify genetic signatures linked to treatment outcomes. The study uses targeted next generation sequencing of DNA and RNA from blood samples taken at diagnosis. It retrospectively analyzes mutations in 64 genes and genetic expression profiles to find associations with response to TKI treatment. Children diagnosed with chronic phase CML and treated with TKIs are studied, including a subgroup with detailed RNA analysis. The goal is to compare molecular profiles of those who do and do not achieve complete cytogenetic response (CCR) at 12 months. Participants provide blood samples at diagnosis for genetic testing. Researchers track treatment response at 3, 12, 18, and 24 months using molecular and cytogenetic measures, as well as monitoring disease progression and survival. The main outcome is CCR at 12 months after starting TKI treatment. This observational study provides insights into genetic markers that might predict TKI response in pediatric CML, with total participation spanning at least two years of follow-up.
CONDITIONS
Brief Title
CML Pediatric ITK Response According to Molecular Identification at Diagnosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age at diagnosis less than or equal to 18 years
- Presence of Philadelphia chromosome detected by cytogenetic analysis (karyotype or FISH) and BCR::ABL1 transcript e13a2 or e14a2
- Diagnosis in chronic phase according to European Leukemia Net criteria
- First-line treatment with tyrosine kinase inhibitors (TKIs)
- Possible pre-treatment with hydroxyurea
- DNA sample available at diagnosis
- RNA sample available for a subgroup of patients (8 responders vs 8 non-responders)
You will not qualify if you...
- Age at diagnosis more than 18 years
- Diagnosis in accelerated or blastic phase
- First-line treatment other than tyrosine kinase inhibitors (TKIs)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At diagnosis
Participants provide DNA samples at diagnosis for targeted next generation sequencing. A subgroup also provides RNA samples for further analysis.
1 visit (in-person)
Duration - Up to 24 months
Participants are monitored for molecular and cytogenetic responses to tyrosine kinase inhibitor treatment, including assessment of resistance and survival outcomes.
Visits at 3, 12, 18, and 24 months
Trial Site Locations
Total: 1 location
1
CHU de Bordeaux, Service Hématologie Biologique
Bordeaux, France
Actively Recruiting
Research Team
S
Stéphanie DULUCQ
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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