Clinical trial · Interventional
Trial of Afatinib in Pediatric Tumours
Phase I/II Open Label, Dose Escalation Trial to Determine the MTD, Safety, PK and Efficacy of Afatinib Monotherapy in Children Aged ≥1 Year to <18 Years With Recurrent/Refractory Neuroectodermal Tumours, Rhabdomyosarcoma and/or Other Solid Tumours With Known ErbB Pathway Deregulation Regardless of Tumour Histology
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Open-label, dose escalation, monotherapy, basket trial with biomarker specific MTD expansion cohort/Phase II part. The trial will consist of 2 parts: 1. Dose finding part to determine the MTD 2. Biomarker specific MTD expansion cohort/Phase II part to assess clinical anti-tumour activity in included tumour types
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroectodermal Tumors | Primitive Neuroectodermal Tumor | ALIAS | 0.90 |
| Rhabdomyosarcoma | Rhabdomyosarcoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| afatinib | Drug | Afatinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- afatinib
- description
- dose escalation
- interventionNames
- Drug: afatinib
Primary outcomes (4)
- measure
- Number of Participants With Objective Response - Maximum Tolerated Dose Expansion (MTD) Cohort
- timeFrame
- Assessed every 8 weeks until progression of disease, up to 336 days.
- description
- Number of participants with objective response for maximum tolerated dose expansion (MTD) cohort was reported. The objective response was defined as a best overall response of complete response or partial response based on investigator's assessment according to the institutional response evaluation criteria for the given tumour type, assessed every 8 weeks until progression.
- measure
- Area Under the Curve Over Dosing Interval τ at Steady State (AUCτ,ss) - Dose Finding Part
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion criteria: * Paediatric patients aged 1 year to \<18 years at the time of informed consent * diagnosis of HGG, DIPG, low grade astrocytoma, medulloblastoma/PNET, ependymoma, neuroblastoma, RMS and tumours with ErbB deregulation * recurrent/refractory disease after they received at least one prior standard treatment regimen * no effective conventional therapy exists * Performance status \>= 50% (Lansky for =\<12ys; Karnofsky for \>12ys) * Further inclusion criteria apply Exclusion criteria: * relevant toxicity from previous treatment * known pre-existing relevant cardiac , hepatic, renal, bone marrow dysfunction, ILD, keratitis * Further exclusion criteria apply
References
Publications (2)
- DERIVEDGeoerger B, Marshall LV, Nysom K, Makin G, Bouffet E, Defachelles AS, Amoroso L, Aerts I, Leblond P, Barahona P, Van-Vlerken K, Fu E, Solca F, Lorence RM, Ziegler DS. Afatinib in paediatric patients with recurrent/refractory ErbB-dysregulated tumours: Results of a phase I/expansion trial. Eur J Cancer. 2023 Jul;188:8-19. doi: 10.1016/j.ejca.2023.04.007. Epub 2023 Apr 20. PMID 37178647
- DERIVEDAndrade RC, Boroni M, Amazonas MK, Vargas FR. New drug candidates for osteosarcoma: Drug repurposing based on gene expression signature. Comput Biol Med. 2021 Jul;134:104470. doi: 10.1016/j.compbiomed.2021.104470. Epub 2021 May 7. PMID 34004576