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

NCT07727694 | Recruiting

• 1 other identifier: S67492
• Study Type: observational
• Target: 1,600
• Locations: 1 country
• Sites: 6

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.

Conditions

Pediatric Cancer

Keywords

observational; data collection; childhood cancer; pediatric cancer; off label medication; CU medication

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  through study completion, an average of 1 year

Study Arms (1)

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

Other: data collection

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.

Patients in therapeutic failure or relapse

Eligibility Criteria

• Age: 0 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study 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

You may qualify if:

• 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
• Consent to participate by the patient or his/her legal representatives in the study if required by ta particular national jurisdiction

You may not qualify if:

• Patient receiving the drug within a clinical trial.
• Oral refusal to participate to the study, by the patient or his/her parents/legal representatives.

Sponsors & Collaborators

• Universitaire Ziekenhuizen KU Leuven: lead

Study Sites (6)

University Hospital Antwerp

Antwerp, 2650, Belgium

NOT YET RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)

Brussels, 1020, Belgium

NOT YET RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

NOT YET RECRUITING

University Hospital Gent

Ghent, 9000, Belgium

NOT YET RECRUITING

University Hospital Leuven

Leuven, 3000, Belgium

RECRUITING

CHC Montlégia

Liège, 4000, Belgium

NOT YET RECRUITING

MeSH Terms

Conditions

Neoplasms

Interventions

Data Collection

Intervention Hierarchy (Ancestors)

Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2026
• First Posted: July 27, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): December 31, 2030
• Study Completion (Estimated): December 31, 2030
• Last Updated: July 27, 2026

Record last verified: 2026-07

Locations

BE (6)