In breve
Sodium Phosphate is the substance named in this clinical-trial article, which focuses on research studies rather than a drug guide. The trials are evaluating safety and efficacy in people with Angelman syndrome, with attention to long-term follow-up and treatment-related outcomes.
Punti chiave
- Clinical trial data for Sodium Phosphate are linked here to a Phase 3 long-term extension study in people with Angelman syndrome. The study is interventional and includes 98 participants. Its main focus is long-term safety and efficacy, with special attention to treatment-emergent adverse events and serious adverse events. The trial also looks at how often these events happen, how severe they are, and whether they are related to the investigational product. This is a late-stage study designed to gather more information over time in the same patient group.
Overview of the trial
This clinical study is a long-term extension trial investigating the safety and efficacy of GTX-102 in patients with Angelman syndrome. The trial is Authorised and is listed as a Phase 3 interventional study.
The trial brief says the main goal is to evaluate the long-term safety profile of GTX-102 in subjects with AS. In this article, Sodium Phosphate is the substance named in the source data, and the focus is on the clinical research information provided in the trial record.
Who the trial is for
The target population is people with Angelman syndrome. The source data do not give more detailed entry rules, so the key point is that the study is aimed at patients with this condition.
Because this is a long-term extension study, it is meant for participants already connected to the research program and followed over time.
Study design and phase
The trial is an interventional study, which means participants receive a study treatment as part of the research plan. It is in Phase 3, a late stage of clinical testing that usually looks at effects and safety in a larger group of participants.
The study is described as a long-term extension, so it is designed to continue collecting information after earlier study work and to follow participants for a longer period.
What the study measures
The main outcome is treatment-emergent adverse events and serious adverse events throughout the study. These outcomes are measured by their frequency, severity, and relationship to the investigational product.
In simple terms, the researchers want to know how often medical problems happen during the study, how serious they are, and whether they may be linked to the treatment being tested. The brief summary also states that the study aims to evaluate the long-term safety profile of GTX-102 in subjects with AS.
Treatments used in the trial
The intervention list includes GTX-102, a GTX/UX Diluent and Flush Solution, and ELLIOTTS B SOLUTION. The source describes these as drugs used for intrathecal use, which means they are given into the fluid around the spinal cord.
The source data do not provide a broader treatment explanation beyond the trial setup, so the focus remains on how these products are used within the study.
Trial status and enrollment
The trial status is Authorised. The planned enrollment is 98 participants.
This size suggests a focused late-stage study in a specific patient group rather than a very large population trial.