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

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 0 to 25 years
• All sexes
• No healthy volunteers
• Child, Adult

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

Trial locations and contacts

6 sites in 1 country. Contact the study team or a site near you to ask about taking part.

About this study

Conditions

Pediatric Cancer

Being tested

data collection

Keywords

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

Medical subject headings

Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• 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.

What is being measured

The primary outcome is the main question the study is designed to answer.

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.

   Measured: 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.

   Measured: through study completion, an average of 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted27 Jul 2026
2. Study startJul 2026 (estimated)
3. Primary completion31 Dec 2030 (estimated)
4. Study completion31 Dec 2030 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Other trials for Pediatric CancerStudies of the same condition, open and under way.
• More trials run by Universitaire Ziekenhuizen KU LeuvenThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.