Phase 2
N=56
Trial of Afatinib in Pediatric Tumours
Neuroectodermal Tumors · Rhabdomyosarcoma
Bottom Line
View on ClinicalTrials.gov: NCT02372006 ↗Enrolled (actual)
56
Serious AEs
58.9%
Results posted
Mar 2021
Primary outcome: Primary: Number of Participants With Objective Response - Maximum Tolerated Dose Expansion (MTD) Cohort — 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- afatinib (Drug)
- Age
- Pediatric, Adult · 1+ yrs
- Sex
- All
- Sponsor
- Boehringer Ingelheim
- Primary completion
- Aug 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Objective Response - Maximum Tolerated Dose Expansion (MTD) Cohort |
3 | — |
| PRIMARY Area Under the Curve Over Dosing Interval τ at Steady State (AUCτ,ss) - Dose Finding Part |
681; 1380 | — |
| PRIMARY Maximum Measured Concentration of the Analyte in Plasma at Steady State (Cmax,ss) - Dose Finding Part |
53.0; 115 | — |
| PRIMARY Number of Participants With Dose Limiting Toxicity Adverse Events - Dose Finding Part |
1; 2 | — |
| SECONDARY Number of Participants With Objective Response - Dose Finding Part |
0; 0 | — |
| SECONDARY Progression Free Survival - Maximum Tolerated Dose (MTD) Expansion Cohort |
8.0 | — |
| SECONDARY Area Under the Concentration-time Curve of the Analyte in Plasma Over the Time Interval From 0 to 24 Hours (AUC0-24) - Dose Finding Part |
383; 512 | — |
| SECONDARY Maximum Measured Concentration (Cmax) - Dose Finding Part/Maximum Tolerated Dose (MTD) Expansion Cohort |
36.4; 43.8; 30.5 | — |
| SECONDARY Time From (Last) Dosing to the Maximum Measured Concentration (Tmax) - Dose Finding Part/Maximum Tolerated Dose (MTD) Expansion Cohort |
3.02; 3.43; 3.98 | — |
| SECONDARY Time From (Last) Dosing to the Maximum Measured Concentration at Steady State (Tmax,ss) - Dose Finding Part/Maximum Tolerated Dose (MTD) Expansion Cohort |
3.00; 2.75; 4.17 | — |
| SECONDARY Accumulation (or Effective) Half-life - Dose Finding Part/Maximum Tolerated Dose (MTD) Expansion Cohort |
18.7; 31.0; 30.3 | — |
| SECONDARY Duration of Objective Response - Maximum Tolerated Dose (MTD) Expansion Cohort |
62 | — |
| SECONDARY Area Under the Curve Over Dosing Interval τ at Steady State (AUCτ,ss) - Maximum Tolerated Dose (MTD) Expansion Cohort |
780 | — |
| SECONDARY Maximum Measured Concentration of the Analyte in Plasma at Steady State (Cmax,ss) - Maximum Tolerated Dose (MTD) Expansion Cohort |
52.5 | — |
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
Eligibility Criteria
Inclusion criteria
- Paediatric patients aged 1 year to = 50% (Lansky 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
Data sourced from ClinicalTrials.gov (NCT02372006). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.