Personalized medicine for kids with tough cancers: genetic profiling may point to better treatments
NCT ID NCT02613962
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looked at whether analyzing the DNA and RNA of children's relapsed or treatment-resistant tumors can help doctors pick targeted therapies. Researchers profiled tumors from 791 children and adolescents using advanced genetic sequencing. The goal was to see how many patients could be matched to a specific targeted drug based on their tumor's unique molecular changes.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this could help doctors choose more effective, personalized treatments for children with cancer that has come back or not responded to standard therapy.
- What could go wrong
- This is an early-stage, observational study focused on feasibility, not a treatment trial. Many children may not have a targetable alteration, and even if they do, the matched therapy may not work or may have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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791 people
The number who actually took part.
- Started
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Jan 2016
- Finished
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Nov 2022
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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6 months to 18 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion criteria: * Written informed consent signed by the patient, or parents or legal representative and assent of the minor child to perform biopsy/surgical resection/aspiral/sample and molecular analysis of the tumor and blood sample * Patient with confirmed solid tumor or leukemia which is recurrent or refractory to standard treatment and who is eligible for an early phase clinical trial * In case of solid tumor, lesion must be accessible for biopsy or surgical resection or cytological puncture * Age: Patients aged ≥ 6 months at time of inclusion and aged ≤ 18 years at the time of initial diagnosis * Performance status and life expectancy that allows treatment in an experimental trial: Karnofsky performance status ≥ 70% for patients \> 12 years of age, Lansky play scale ≥ 70% for patients ≤ 12 years of age * Adequate organ function: Adequate hematopoietic function for patients with solid tumor (Leukemia patients are excluded from hematological criteria) : Neutrophils \>1.0 x 109/l, Platelets \>100 x 109/l, Heamoglobin \>80 g/l (transfusion allowed) In case of bone marrow involvment: Neutrophils \>/= 0.75 x 109/l (unsupported) , Platelet count \>/= 0.75 x 109/l (unsupported) Adequate hepatic function: ALAT/ASAT \<2.5 x ULN, Bilirubin ≤1.5 x ULN (in case of tumor involvement of the liver ALAT/ASAT \<5 x ULN) Adequate renal function: Serum creatinemia \<1.5 x ULN for age. In case serum creatinine \>1.5 ULN according to age, creatinine clearance has to be \>70mL/mL/1.73 m2 or glomerular filtration rate measurement \>70% of the expected value \- Patients affiliated to a Social Security Regimen or beneficiary of the same as per local regulatory requirements Exclusion criteria: * Life expectancy ≤ 3 months * Symptomatic CNS metastases * In case of leukemia, are excluded patients with isolated meningitis relapses. * Coagulation disorder that prevents the accomplishment of a biopsy or surgery * Uncontrolled infections not responsive to antibiotics, antiviral medicines, or antifungal medicines * Presence of ≥ CTCAE grade 2 toxicity (except alopecia, ototoxicity, lymphopenia which are not excluded if grade 3 or less) due to prior cancer therapy * Malignant disease other than that being treated in this study. Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 3 years prior to study entry * Any concurrent illness or laboratory abnormality that in opinion of investigator may interfere with the interpretation of study results, may suppose a risk for the realization of biopsy/surgery, and in the judgment of the investigator would make the patient inappropriate for the study * Evidence of active viral Hepatitis B or C or known diagnosis of human immunodeficiency virus infection
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Fondazione IRCCS Istituto
Milan, 20133, Italy
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Gustave Roussy
Villejuif, Val de Marne, 94805, France
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Hospital Universitari Vall D'Hebron
Barcelona, Catalonia, 08035, Spain
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Our Lady's Children's Hospital
Dublin, Crumlin, 12, Ireland
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Schneider Children's Medical Center
Petah Tikva, 49202, Israel