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Sodium Phosphate

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.

Table of contents

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.[1] The trial is Authorised and is listed as a Phase 3 interventional study.[1]

The trial brief says the main goal is to evaluate the long-term safety profile of GTX-102 in subjects with AS.[1] 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.[1] 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.[1]

Study design and phase

The trial is an interventional study, which means participants receive a study treatment as part of the research plan.[1] It is in Phase 3, a late stage of clinical testing that usually looks at effects and safety in a larger group of participants.[1]

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.[1]

What the study measures

The main outcome is treatment-emergent adverse events and serious adverse events throughout the study.[1] These outcomes are measured by their frequency, severity, and relationship to the investigational product.[1]

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.[1] The brief summary also states that the study aims to evaluate the long-term safety profile of GTX-102 in subjects with AS.[1]

Treatments used in the trial

The intervention list includes GTX-102, a GTX/UX Diluent and Flush Solution, and ELLIOTTS B SOLUTION.[1] The source describes these as drugs used for intrathecal use, which means they are given into the fluid around the spinal cord.[1]

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.[1]

Trial status and enrollment

The trial status is Authorised.[1] The planned enrollment is 98 participants.[1]

This size suggests a focused late-stage study in a specific patient group rather than a very large population trial.[1]

Trial ID Phase Condition studied Status Enrollment
2024-510917-14-00 Phase 3 Angelman syndrome Authorised 98

FAQ

  • What are the clinical trials about Sodium Phosphate studying? They are studying long-term safety and efficacy in people with Angelman syndrome. The main goal is to see how well the investigational treatment is tolerated over time.
  • Who can take part in these trials? The trial data mention patients with Angelman syndrome. Participation is therefore focused on this patient group.
  • What phase is the trial? The trial is in Phase 3. This means it is a late-stage study that looks closely at safety and treatment effects in a larger group.
  • What outcome is being measured? The main outcome is treatment-emergent adverse events and serious adverse events, including how often they happen, how severe they are, and whether they are related to the investigational product.
  • Is this a short study? No. The trial is a long-term extension study, which means it follows patients for an extended period to collect more safety and efficacy information.

Sperimentazioni cliniche in corso su Sodium Phosphate

  • Studio sull’Efficacia e Sicurezza a Lungo Termine di GTX-102 nei Pazienti con Sindrome di Angelman

    In arruolamento

    1 1 1
    Malattie in studio:
    Farmaci in studio:
    Francia Germania Spagna

Glossario

  • Angelman syndrome: A genetic condition studied in this trial. The trial is focused on people who have this syndrome.
  • Long-term extension trial: A study that continues after an earlier trial so researchers can collect more information over a longer time.
  • Safety: How well a treatment is tolerated and whether unwanted medical problems happen during the study.
  • Efficacy: How well a treatment works for the condition being studied.
  • Phase 3: A late stage of clinical research, usually done in a larger group of participants to better understand safety and effects.
  • Interventional study: A study where participants receive a treatment or procedure assigned by the research team.
  • Intrathecal use: Given into the fluid around the spinal cord. This is a special route used in the study.
  • Adverse event (AE): Any unwanted medical problem that happens during a study, whether or not it is caused by the treatment.
  • Serious adverse event (SAE): A more serious unwanted medical problem, such as one that needs hospital care or causes major harm.
  • Investigational product: The treatment being studied in the trial. It is not yet fully established for routine use in this research setting.

Riferimenti

  1. https://clinicaltrials.gov/study/2024-510917-14-00