Pediatric Solid tumors and primary central nervous system (CNS) tumors
Conditions
Brief summary
1. Dose limiting toxicity DLT during the first cycle (28 days) in pediatric patients with relapsed or refractory solid tumors using the F1 formulation (and subsequently using F06, minitablets). The RP2D will be determined from DLT derived from clinical and laboratory observations in the first treatment cycle according to the NCI CTCAE v4.03 that is related to entrectinib), 2. Objective response rate as assessed by the BICR will be evaluated in the efficacy evaluable population from the Phase II dose expansion cohorts (Cohort B and D)
Detailed description
1. Incidence and severity of adverse events, laboratory and electrocardiogram (ECG) abnormalities, 2. Maximal plasma concentration (Cmax) of entrectinib (F1, F06 given intact, F06 administered via feeding tube, and minitablets), 3. Time of maximal plasma concentration (Tmax) of entrectinib (F1, F06 given intact, F06 administered via feeding tube, and minitablets), 4. Area under the plasma concentration vs. time curve (AUC) of entrectinib (F1, F06 given intact, F06 administered via feeding tube, and minitablets), 5. ORR in the efficacy evaluable and safety evaluable populations, 6. DOR and TTR as assessed by BICR and the investigator in the efficacy evaluable and safety evaluable populations:, 7. ORR, TTR, DOR in the efficacy evaluable and safety evaluable populations as assessed by BICR and the investigator with use of RECIST v1.1, RANO, and the Curie scale, as applicable, 8. CBR and PFS in the efficacy evaluable and safety evaluable populations as assessed by BICR and investigator with use of RECIST v1.1, RANO, and the Curie scale, as applicable, 9. OS in the efficacy evaluable and safety evaluable populations, 10. Assessment of growth (weight, height), puberty (Tanner), neurological function and neurocognitive function of patients on treatment, 11. Acceptability and palatability assessment for F06 capsules and minitablets formulation
Interventions
DRUGRozlytrek
Sponsors
F. Hoffmann-La Roche AG
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Dose limiting toxicity DLT during the first cycle (28 days) in pediatric patients with relapsed or refractory solid tumors using the F1 formulation (and subsequently using F06, minitablets). The RP2D will be determined from DLT derived from clinical and laboratory observations in the first treatment cycle according to the NCI CTCAE v4.03 that is related to entrectinib), 2. Objective response rate as assessed by the BICR will be evaluated in the efficacy evaluable population from the Phase II dose expansion cohorts (Cohort B and D) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Incidence and severity of adverse events, laboratory and electrocardiogram (ECG) abnormalities, 2. Maximal plasma concentration (Cmax) of entrectinib (F1, F06 given intact, F06 administered via feeding tube, and minitablets), 3. Time of maximal plasma concentration (Tmax) of entrectinib (F1, F06 given intact, F06 administered via feeding tube, and minitablets), 4. Area under the plasma concentration vs. time curve (AUC) of entrectinib (F1, F06 given intact, F06 administered via feeding tube, and minitablets), 5. ORR in the efficacy evaluable and safety evaluable populations, 6. DOR and TTR as assessed by BICR and the investigator in the efficacy evaluable and safety evaluable populations:, 7. ORR, TTR, DOR in the efficacy evaluable and safety evaluable populations as assessed by BICR and the investigator with use of RECIST v1.1, RANO, and the Curie scale, as applicable, 8. CBR and PFS in the efficacy evaluable and safety evaluable populations as assessed by BICR and investigator wit | — |
Countries
France, Germany, Italy, Spain
Outcome results
None listed