Pediatric Cancer
Conditions
Keywords
observational, data collection, childhood cancer, pediatric cancer, off label medication, CU medication
Brief summary
It involves collecting safety and efficacy data, under the actual conditions of use of off label and compassionate use medicines in children and adolescents approved in humans before 2007, using a validated tool (Ennov EDC) and relying on the network recognised pediatric hemato-oncology centers in Belgium and responsible for the organization of Pediatric National tumor boards which discuss each case of relapse in order to define the best therapeutic options.
Detailed description
The objective is to aim for completeness throughout the national territory. This is why this project will be carried out in close collaboration with all recognised pediatric hemato-oncology centers in Belgium.
Interventions
OTHERdata collection
In particular, the patient's demographic data, medical history, previous and concomitant treatments, data on the treatment of interest (legal framework, compassionate use/ off-label), dosage, start date, etc.), clinical data will be collected. biological (including molecular profiling of the tumor if available) and radiological follow-up, information on adverse effects.
Sponsors
Universitaire Ziekenhuizen KU Leuven
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to 25 Years
Healthy volunteers
No
Inclusion criteria
* Age ≤ 25 at the time of inclusion in the study * Patient with a pediatric malignancy (solid tumor or leukemia), or other related condition * Patient not currently enrolled on any other trial for ongoing therapeutic purposes * Patient treated with compassionate use of experimental drugs or off-label administration use of anti-cancer medicines first approved in adults after 2007 in Europe * Patient discussed in a Pediatric multidisciplinary tumor boards (MDTB) to validate the inclusion in the SACHA study * Consent to participate by the patient or his/her legal representatives in the study if required by ta particular national jurisdiction
Exclusion criteria
* Patient receiving the drug within a clinical trial. * Oral refusal to participate to the study, by the patient or his/her parents/legal representatives.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Collect safety data | through study completion, an average of 1 year | From patients receiving innovative molecules outside a clinical trial (targeted therapies, immunotherapies or chemotherapies off label or compasionate use approved in adults as of 2007), safety data will be collected. Adverse events will be collected through the CTCAE version N°5. |
| Collect activity data | through study completion, an average of 1 year | From patients receiving innovative molecules outside a clinical trial (targeted therapies, immunotherapies or chemotherapies off label or compasionate use approved in adults as of 2007), the response to the treatment (complete response, partial response, stable disease, progression) will be collected. |
Countries
Belgium
Outcome results
None listed