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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-25
SponsorUniversitaire Ziekenhuizen KU Leuven

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

Patient receiving the drug within a clinical trial.

Oral refusal to participate to the study, by the patient or his/her parents/legal representatives.

Trial population

Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of compassionate use or outside marketing authorization, in one of the centers of the pediatric hemato-oncology centers in Belgium

Trial design

Design model

Other

Time perspective

Prospective

Treatments tested in this trial

  • data collection

    Other intervention

    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.

Treatment groups

1,600 Participants
are divided into 1 treatment group
Group A: Patients in therapeutic failure or relapse1 intervention

Trial outcomes

Primary outcomes

1

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
2

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

Secondary outcomes

Other outcomes

Sponsors and contacts

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