Phase I/II 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 - Phase I/II trial of afatinib in pediatric tumours

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-002123-10-DE

Enrollment

Registered

2015-02-10

Start date

2015-12-17

Completion date

Unknown

Last updated

2021-01-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paediatric patients with recurrent/refractory high grade glioma (HGG), diffuse intrinsic pontine glioma (DIPG), low grade astrocytoma, neuroblastoma, ependymoma, medulloblastoma/primitive neuroectodermal tumours (PNET), rhabdomyosarcoma (RMS) and/or other solid tumours with known ErbB pathway deregulation regardless of tumour histology MedDRA version: 20.0 Level: LLT Classification code 10029091 Term: Neoplasm of unspecified nature of endocrine glands and other parts of nervous system System Or

Interventions

Trade Name: GIOTRIF Product Name: Afatinib Product Code: BIBW 2992 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: afatinib CAS Number: 850140-72-6 Other descriptive name: AFATINIB Concen

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Paediatric patients - aged =1 year - 150 (membrane staining) and/or - HER2 protein expression: H-score > 0 (membrane staining) Inclusion will be based on biomarker assessment prior to inclusion, as detected by central laboratory analysis of tumour biopsy materia - Patients with proven genomic, transcriptomic or proteomic ErbB alterations which are not defined in the above regardless of tumour histology - Performance status >= 50% (Lansky for =12ys) Are the trial subjects under 18? yes Number of subjects for this age range: 55 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: -relevant toxicity from previous treatment -known pre-existing relevant cardiac , hepatic, renal, bone marrow dysfunction, ILD, keratitis

Design outcomes

Primary

Measure	Time frame
Main Objective: -Establish MTD of afatinib in pediatric patients -describe pharmacokinetics of afatinib -investigate objective response (OR) to treatment ;Secondary Objective: -Safety -pharmacokinetics -efficacy by objective reponse, duration of response and Progression free survival ;Primary end point(s): In dose finding part 1) DLT measured during the first course of treatment 2) Pharmacokinetics (AUCt,ss, Cmax,ss) In MTD expansion cohort 3)Objective Response by investigator assessment according to the institutional response evaluation criteria for the given tumour type, assessed every 8 weeks until progression of disease. ;Timepoint(s) of evaluation of this end point: 1: up to 1 year 2: up to 1 year 3: up to 2 years	—

Measure

Time frame

Main Objective: -Establish MTD of afatinib in pediatric patients -describe pharmacokinetics of afatinib -investigate objective response (OR) to treatment ;Secondary Objective: -Safety -pharmacokinetics -efficacy by objective reponse, duration of response and Progression free survival ;Primary end point(s): In dose finding part 1) DLT measured during the first course of treatment 2) Pharmacokinetics (AUCt,ss, Cmax,ss) In MTD expansion cohort 3)Objective Response by investigator assessment according to the institutional response evaluation criteria for the given tumour type, assessed every 8 weeks until progression of disease. ;Timepoint(s) of evaluation of this end point: 1: up to 1 year 2: up to 1 year 3: up to 2 years

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Secondary

Measure	Time frame
Secondary end point(s): In dose finding part 1) Objective Response by investigator assessment according to the institutional response evaluation criteria for the given tumour type, assessed every 8 weeks until progression of disease. 2) Pharmacokinetics (AUC0-24, Cmax, tmax(,ss) and accumulation (or effective) half-life) In MTD expansion cohorts/Phase II part: 3) Progression free survival (PFS) 4) Duration of response 5) Pharmacokinetics (AUC,AUCt(,ss), Cmax,(,ss), tmax(,ss) and accumulation (or effective) half-life);Timepoint(s) of evaluation of this end point: 1: up to 2 years 2: up to 1 year 3: up to 2 years 4: up to 2 years 5: up to 1 year	—

Measure

Time frame

Secondary end point(s): In dose finding part 1) Objective Response by investigator assessment according to the institutional response evaluation criteria for the given tumour type, assessed every 8 weeks until progression of disease. 2) Pharmacokinetics (AUC0-24, Cmax, tmax(,ss) and accumulation (or effective) half-life) In MTD expansion cohorts/Phase II part: 3) Progression free survival (PFS) 4) Duration of response 5) Pharmacokinetics (AUC,AUCt(,ss), Cmax,(,ss), tmax(,ss) and accumulation (or effective) half-life);Timepoint(s) of evaluation of this end point: 1: up to 2 years 2: up to 1 year 3: up to 2 years 4: up to 2 years 5: up to 1 year

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Countries

Australia, Austria, Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

Public ContactQRPE PSC CT Information Disclosure

Boehringer Ingelheim Pharma GmbH & Co. KG

clintriage.rdg@boehringer-ingelheim.com+498002430127

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026