Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults

Universitaire Ziekenhuizen KU Leuven1,600 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

1,600

Conditions

Pediatric Cancer

Interventions

data collectionOTHER

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.

Eligibility

Sex: ALLMin age: 0 YearsMax age: 25 Years

Medical Language ↔ Plain English

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.

Locations (6)

University Hospital Antwerp

Antwerp, Belgium

NOT_YET_RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)

Brussels, Belgium

NOT_YET_RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, Belgium

NOT_YET_RECRUITING

University Hospital Gent

Ghent, Belgium

NOT_YET_RECRUITING

University Hospital Leuven

Leuven, Belgium

RECRUITING

CHC Montlégia

Liège, Belgium

NOT_YET_RECRUITING

Outcomes

Primary Outcomes

Collect safety data

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.

Time frame: through study completion, an average of 1 year

Collect activity data

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.

Time frame: through study completion, an average of 1 year