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

Sponsor
Universitaire Ziekenhuizen KU Leuven
Study ID
NCT07727694
Status
Recruiting
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Conditions

  • Pediatric Cancer

Eligibility Criteria

Sex
ALL
Age
0 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • data collection — OTHER
    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.

Study Details

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.

Key Dates

First listed
Jul 27, 2026
Start date
Jul 31, 2026
Status verified
Jul 2026
Primary completion
Dec 31, 2030
Completion
Dec 31, 2030

Study Design

Enrollment
1,600 participants (estimated)

Arms

  • Arm: Patients in therapeutic failure or relapse
    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 a compassionate use or outside marketing authorization, in one of the centers of the Belgian pediatric hemato-oncology centers

Primary Outcome Measure

Collect safety data [ Time Frame: through study completion, an average of 1 year ]

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