CompletedPhase 1/2

Trial of Afatinib in Pediatric Tumours

United States, Australia, Austria56 participantsStarted 2015-04-29

Plain-language summary

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

Who can participate

Age range

1 Year – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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.

Area Under the Curve Over Dosing Interval τ at Steady State (AUCτ,ss) - Dose Finding Part

Timeframe: Pre-dose before afatinib administration then 1 hour (h), 2 h, 3 h, 4 h, 5 h, 6 h, 8 h and 24 h after administration at steady state on Day 8.

Maximum Measured Concentration of the Analyte in Plasma at Steady State (Cmax,ss) - Dose Finding Part

Timeframe: Pre-dose before afatinib administration then 1 hour (h), 2 h, 3 h, 4 h, 5 h, 6 h, 8 h and 24 h after administration at steady state on Day 8.

Number of Participants With Dose Limiting Toxicity Adverse Events - Dose Finding Part

Timeframe: During the first course (28 days) of treatment.

Trial details

NCT IDNCT02372006

SponsorBoehringer Ingelheim

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-08-05

Results submitted2021-02-04

View on ClinicalTrials.gov More Neuroectodermal Tumors trials

Plain-language summary

Who can participate

Age range

1 Year – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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.

Area Under the Curve Over Dosing Interval τ at Steady State (AUCτ,ss) - Dose Finding Part

Timeframe: Pre-dose before afatinib administration then 1 hour (h), 2 h, 3 h, 4 h, 5 h, 6 h, 8 h and 24 h after administration at steady state on Day 8.

Maximum Measured Concentration of the Analyte in Plasma at Steady State (Cmax,ss) - Dose Finding Part

Timeframe: Pre-dose before afatinib administration then 1 hour (h), 2 h, 3 h, 4 h, 5 h, 6 h, 8 h and 24 h after administration at steady state on Day 8.

Number of Participants With Dose Limiting Toxicity Adverse Events - Dose Finding Part

Timeframe: During the first course (28 days) of treatment.