New insights and therapeutic approaches

Brain tumors and other tumors of the central nervous system (CNS) are the second most common cancer in childhood, accounting for about 24 percent of all cases, and simultaneously the most frequent group of "solid" (i.e., solid, initially spatially limited) tumors.

With regard to trials, we distinguish between clinical trials, diagnostic platforms and registry studies. Clinical trials mean that a new treatment is being tested for efficacy and tolerability among other things. Diagnostic platforms offer molecular analyses of tumor material, while registry studies are observational studies in which data is collected during course of the disease.

You can find an overview of all our clinical studies, as well as our diagnostic platforms and registry studies on solid and brain tumors here below. Additionally, there is an overview of the diagnostic options to facilitate allocation.
 

 

Clinical Trials

Green indicates trials for which recruitment is still open. Red, on the other hand, are those for which recruitment has been completed. Diagnostic platforms and registry studies follow, as does our overview of diagnostic options.

Alectinib

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors.

 

Avapritinib

Phase I/II single-arm study to assess the safety, pharmacokinetics, and antitumor activity of avapritinib in pdiatric patients with tumors dependent on the KIT or PGFRA pathway (BLU-285-3101).

 

FIREFLY-1

Pan-RAF inhibitor DAY101 in BRAF-modified, relapsed or progressive LGGs.

 

LOGGIC/FIREFLY-2

A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients With Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy.

 

INFORM2-NivEnt

Immunotherapy in children and adolescents with refractory high-risk cancers.

 

Niraparib/ Dostarlimab

Unblinded, multicenter dose-escalation and cohort expansion study of niraparib and dostarlimab in pediatric patients with recurrent or progressive solid tumors (SCOOP).

 

Abemaciclib

Abemaciclib in children with solid tumors (I3Y-MC-JPCS).

 

Entrectinib

An open-label, dose-escalation, expansion phase I/II study of entrectinib in children with advanced or metastatic solid tumors or primary central nervous system tumors with/or without sufficient treatment options.

 

Larotrectinib (LOXO-101)

Larotrectinib as a TRK inhibitor in children with solid tumors.

 

Selpercatinib

A phase I/II study of the oral RET inhibitor LOXO-292 in pediatric patients with advanced solid or primary central nervous system tumors with alterations in the RET gene.
 

 

Diagnostic options

Which program is suitable for which patient? To provide some clarity here, we have created a flowchart. If you have any questions or need additional information, you can find contact information on the respective program pages.

 

Diagnostic platforms and registry studies

Here are our diagnostic platforms and registry studies. Clinical trials have been listed above.

 

INFORM

INdividualized therapy FOr Relapsed Malignancies in Childhood: a unique multi-country genome sequencing program for children with cancer in Europe.

 

MNP Int R

The current MNP Int-R follow-up registry study aims to establish an international registry for the generation and coordination of molecular data in pediatric neuro-oncology.

 

LOGGIC Core

The LOGGIC Core BioClinical database (LOGGIC Core) is a registry of molecular and clinical data for pediatric low-grade gliomas (LGG).