In short
Clinical trials investigating Sodium Phosphate are being used as part of a study in people with Angelman Syndrome. The trial looks at long-term safety and efficacy in a Phase 3 setting and follows patients over time. It mainly includes people with Angelman Syndrome who are receiving intrathecal treatment in the study.
Key points
- Clinical trial data for Sodium Phosphate is linked to one authorised Phase 3 interventional study in people with Angelman Syndrome. The trial is a long-term extension study, which means it follows patients for a longer period. Its main focus is safety, including treatment-emergent adverse events and serious adverse events. The study also looks at efficacy, or how well the treatment works, over time. The trial includes 98 planned participants. The study uses intrathecal treatment, meaning the product is given in the space around the spinal cord.
Trial overview
The clinical trial data shows one authorised study investigating Sodium Phosphate in people with Angelman Syndrome. The study is described as a long-term extension trial, which means it continues research over a longer period after earlier study work.
The brief summary says the trial aims to evaluate the long-term safety profile of GTX-102 in subjects with Angelman Syndrome. The study also includes efficacy, meaning it looks at how well the treatment works over time.
Study design and phase
This is an interventional study, which means researchers give study treatment and then observe the results. The trial is in Phase 3, a later stage of clinical testing that usually includes more patients and focuses on safety and outcomes in a larger group.
The study status is Authorised, and the planned enrollment is 98 participants. This gives a sense of the trial size and that it is open for the research process described in the source data.
Who can participate
The target population is patients with Angelman Syndrome. The trial description does not list broader groups, so the study is focused on this specific condition.
The intervention list includes study products given by intrathecal use, which means they are delivered into the space around the spinal cord. The listed products are GTX-102, GTX/UX Diluent and Flush Solution, and ELLIOTTS B SOLUTION.
What is being measured
The main endpoint is treatment-emergent adverse events and serious adverse events, including their frequency, severity, and relationship to the investigational product throughout the study. In simple terms, the researchers want to know what medical problems happen after treatment starts, how serious they are, and whether they may be linked to the study treatment.
This endpoint is important because it helps show the long-term safety profile of the treatment under study. The source also states that the study is evaluating efficacy, but it does not give a detailed list of secondary outcomes in the trial data provided.
Study treatments
The intervention section lists GTX-102, which is the investigational product named in the trial summary. It also lists GTX/UX Diluent and Flush Solution and ELLIOTTS B SOLUTION as intrathecal study-related products.
The source data does not provide a broader treatment comparison or placebo description, so the key point is that the study is following patients receiving these trial products as part of the research plan.
Patient-friendly terms
Long-term extension trial means the study continues for a longer time so researchers can learn more about safety and results over time. Efficacy means how well the treatment works for the condition being studied.
Safety profile means the overall pattern of side effects or medical problems seen in the study. Frequency means how often an event happens, and severity means how serious it is.
Relationship to the investigational product means researchers judge whether a medical event may be caused by the study treatment. These terms help patients understand what the trial team is watching during the study.