In short
Clinical trials are investigating 204-NETU in children and teenagers with cancer who receive highly emetogenic chemotherapy, which means chemotherapy that often causes nausea and vomiting. The studies look at safety, drug exposure, and how well it prevents chemotherapy-induced nausea and vomiting in different treatment schedules.
Key points
- Clinical trials of 204-NETU are studying prevention of chemotherapy-induced nausea and vomiting in pediatric cancer patients. The trial is in Phase 4 and includes children and teenagers receiving highly emetogenic chemotherapy. One part looks at drug exposure and safety after repeated dosing, while another part compares how well 204-NETU works against an active comparator. The main outcome is complete response, meaning no vomiting and no rescue medicine during the delayed phase after chemotherapy. Safety checks include physical exams, blood tests, urine tests, vital signs, and ECGs.
Clinical trial overview
The available trial on 204-NETU is an interventional study in pediatric cancer patients who receive highly emetogenic chemotherapy, or chemotherapy with a high chance of causing vomiting.
The study is authorised and in Phase 4, which means it is being studied later in development and in a more practical treatment setting.
The trial includes 120 participants.
Study design and trial parts
The study has two main parts.
Part I is open-label, which means the treatment is known during the study, and it focuses on pharmacokinetics, safety, and efficacy after a single cycle and after repeated cycles in children receiving single-day or multi-day highly emetogenic chemotherapy.
In Part I, Cohort 1 measures pharmacokinetics, meaning how the body handles the drug, by looking at maximum concentration (Cmax) and area under the plasma concentration-time curve (AUC), which shows total drug exposure over time.
In Part I, Cohort 2 focuses on safety and tolerability after repeated IV NEPA administration on Days 1 and 3, or on Days 1, 3, and 5, in pediatric cancer patients receiving multi-day highly emetogenic chemotherapy.
Part II is double-blind, which means the treatment assignment is hidden, and it tests efficacy, non-inferiority, and safety in repeated cycles.
Who can participate
The study is designed for pediatric cancer patients, meaning children and teenagers with cancer.
For the pharmacokinetic part, the age range is from 3 months up to under 18 years in one cohort, and under 3 months in another cohort as described in the study summary.
For the efficacy comparison in Part II, the age range is from 6 months up to under 18 years.
All participants are receiving highly emetogenic chemotherapy, which is the type of chemotherapy the study is trying to support with nausea and vomiting prevention.
What is being measured
The main outcome in Part II is the proportion of patients with complete response (CR) during the delayed phase of emesis, which means no vomiting episodes and no rescue medicine between more than 24 hours and 120 hours after chemotherapy starts.
In Part I, the main measurements are netupitant exposure values such as Cmax, AUClast, and AUCinf, which help researchers understand how much drug is in the body and for how long.
Safety is checked through reports of adverse events, physical examinations, vital signs, clinical laboratory tests, and 12-lead ECGs, which are heart rhythm recordings.
Comparators and study treatments
Part II compares IV NEPA, which includes fosnetupitant chloride and palonosetron, with an active comparator made of IV fosaprepitant plus IV ondansetron.
The study also includes sodium chloride injection as a control infusion in the listed interventions.
The trial aims to show non-inferiority, meaning the new approach should not be meaningfully worse than the comparator for preventing delayed nausea and vomiting.
What the trial results may mean
This trial is focused on whether 204-NETU can help prevent nausea and vomiting during and after highly emetogenic chemotherapy in children and teenagers.
It also helps researchers learn whether repeated use is safe and how the drug behaves in younger patients, including very young children.
Because the study includes both safety and efficacy outcomes, it is designed to answer two important questions: can the treatment be used safely, and does it work well enough to control delayed vomiting after chemotherapy?