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Personalized medicine for kids with tough cancers: genetic profiling may point to better treatments

NCT ID NCT02613962

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

791 people

The number who actually took part.

Started

Jan 2016

Finished

Nov 2022

Lead sponsor

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

6 months to 18 years

Sex

Anyone

Healthy volunteers

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

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

  • Fondazione IRCCS Istituto

    Milan, 20133, Italy

  • Gustave Roussy

    Villejuif, Val de Marne, 94805, France

  • Hospital Universitari Vall D'Hebron

    Barcelona, Catalonia, 08035, Spain

  • Our Lady's Children's Hospital

    Dublin, Crumlin, 12, Ireland

  • Schneider Children's Medical Center

    Petah Tikva, 49202, Israel