In short
Clinical trials are investigating Treprostinil Sodium in children and other patients with pulmonary arterial hypertension. These studies mainly look at safety, tolerability, and treatment effects in specific patient groups. The trial data below focuses on who is being studied, what is measured, and the trial phase.
Key points
- Clinical trials of Treprostinil Sodium are studying children below 18 years of age with pulmonary arterial hypertension. The listed study is a Phase 3, open-label, single-arm, non-controlled trial. It is authorised and includes 37 participants. Researchers are mainly looking at safety and tolerability, especially the frequency and seriousness of adverse events and adverse drug reactions during the first 5 months of treatment. The trial includes patients who are treatment naive or who have already received commercially available parenteral treprostinil.
Trial overview
The available trial is an open-label, single-arm, non-controlled study of Treprostinil Sodium in children with pulmonary arterial hypertension (PAH). It is authorised and includes 37 participants.
The study title says it is designed to evaluate the safety and tolerability of treprostinil sodium in children below 18 years of age with PAH. The brief summary also says the study is for paediatric patients with PAH who are either treatment naive or have previously been treated with commercially available parenteral treprostinil formulation.
Who can participate
The trial is for paediatric patients, meaning children and teenagers below 18 years of age. They must have pulmonary arterial hypertension, also called PAH.
The study includes two main patient groups: people who are treatment naive, which means they have not been treated before, and people who have already received commercially available parenteral treprostinil.
What the trial measures
The main outcome is the frequency and seriousness of adverse events and adverse drug reactions during the first 5 months of treatment, or 20 weeks plus or minus 1 week. An adverse event is any health problem that happens during a study, and an adverse drug reaction is a harmful reaction thought to be related to the study drug.
The study uses CTCAE version 5.0, which is a standard system for grading how serious these problems are. This means the trial is mainly focused on safety and tolerability rather than direct comparison with another treatment.
Study design and phase
This is a Phase 3 interventional trial, meaning the researchers are testing a treatment in people and studying its effects in a later-stage trial. Because it is single-arm and non-controlled, all enrolled participants receive the study treatment and there is no separate comparison group.
The interventions listed in the trial include TREPaed_10, TREPaed_5, TREPaed_0.5, TREPaed_2.5, and TREPaed_1, each described as 155 ng and for subcutaneous and intravenous use.
Key patient terms
Subcutaneous means under the skin, and intravenous means into a vein. Parenteral means given by a route other than the digestive system, such as injection or infusion.
Safety in a trial means checking whether the treatment causes medical problems, while tolerability means how well patients can take the treatment without major difficulty. The trial data show that these are the main questions being studied for Treprostinil Sodium in children with PAH.