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Glucose, Anhydrous

This article explores the ongoing clinical trials investigating the long-term safety and effectiveness of GTX-102, a treatment for Angelman Syndrome. The trials involve the use of a solution containing various substances, including glucose, anhydrous, which is administered intrathecally. We’ll discuss the key aspects of these trials, their objectives, and what they mean for patients with Angelman Syndrome.

Table of Contents

What is GLUCOSE, ANHYDROUS?

GLUCOSE, ANHYDROUS is one of the active substances in a medication called ELLIOTTS B SOLUTION. This solution is designed for intrathecal use, which means it is injected directly into the fluid surrounding the brain and spinal cord[1]. Intrathecal administration is a method used to deliver medications directly to the central nervous system.

ELLIOTTS B SOLUTION is described as a sterile, nonpyrogenic (doesn’t cause fever), isotonic solution. It contains several other components besides glucose, including calcium chloride, sodium hydrogen carbonate, potassium chloride, sodium chloride, sodium phosphate, and magnesium sulfate[1].

Angelman Syndrome: An Overview

Angelman Syndrome (AS) is a rare genetic disorder that primarily affects the nervous system. It is characterized by developmental delays, intellectual disability, severe speech impairment, and problems with movement and balance. People with Angelman Syndrome often have a happy, excitable demeanor with frequent smiling and laughter[1].

Clinical Trial: Long-Term Safety and Efficacy Study

A clinical trial is being conducted to investigate a treatment called GTX-102 for patients with Angelman Syndrome. This is a Phase III long-term extension (LTE) study for an unapproved product[1]. The study aims to evaluate the long-term safety and effectiveness of GTX-102 in patients who have previously participated in a GTX-102 clinical trial.

Treatment Details

In this study, patients will receive GTX-102 through an intrathecal lumbar puncture. This means the medication is injected directly into the fluid surrounding the spinal cord in the lower back area. The treatment period may continue until GTX-102 is approved and/or becomes available in the patient’s geographical region[1].

Eligibility Criteria

To participate in this study, patients must meet certain criteria:

  • Have previously participated in a clinical trial with GTX-102
  • Have completed the Loading phase/period of the previous study
  • Have approval from the Ultragenyx Medical Monitor
  • Be screened within 6 months of the last visit in the prior GTX-102 study
  • For females of childbearing potential who are sexually active, use highly effective contraception or abstinence
  • For males, agree to remain abstinent or use acceptable contraceptive methods during the study and for at least 3 months after the final dose[1]

Patients may be excluded from the study if they:

  • Discontinued the prior GTX-102 study due to treatment-related adverse events
  • Have any medical, neurological, or psychological condition that could put them at risk or interfere with study assessments
  • Have known hypersensitivity to GTX-102 or its components
  • Are pregnant, breastfeeding, or planning to become pregnant during the study[1]

Study Objectives and Endpoints

The main objective of this study is to evaluate the long-term safety profile of GTX-102 in subjects with Angelman Syndrome. Secondary objectives include assessing the long-term efficacy of GTX-102 in improving cognitive function, communication, and motor skills[1].

The study will measure:

  • Treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
  • Changes in cognitive abilities, receptive and expressive communication, and gross and fine motor skills using the Bayley-4 assessment tool[1]

Safety Considerations

Patient safety is a primary concern in this study. The following measures are in place:

  • Regular monitoring of adverse events throughout the study
  • A safety follow-up call 30 days after the end of study visit for patients with new or ongoing treatment-emergent adverse events
  • Strict inclusion and exclusion criteria to ensure patient safety
  • Continuous assessment of the risk-benefit ratio for each participant[1]

It’s important to note that while GLUCOSE, ANHYDROUS is a component of ELLIOTTS B SOLUTION, which is used in this study, the primary focus of the trial is on the investigational product GTX-102. The exact role of GLUCOSE, ANHYDROUS in the treatment of Angelman Syndrome is not specified in the provided information.

Aspect Details
Trial Name A Long-Term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome
Main Objective Evaluate the long-term safety profile of GTX-102 in subjects with Angelman Syndrome
Secondary Objective Evaluate long-term efficacy in cognitive function, communication, and motor skills
Treatment GTX-102 administered via intrathecal lumbar puncture
Key Substance Glucose, Anhydrous (part of ELLIOTTS B SOLUTION)
Primary Endpoint Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product
Secondary Endpoints Changes in Bayley-4 scores for cognition, communication, and motor skills
Study Duration Until GTX-102 is approved or available in the patient’s region

FAQ

  • What is the main purpose of this clinical trial? The main purpose of this clinical trial is to evaluate the long-term safety profile of GTX-102 in patients with Angelman Syndrome. It also aims to assess the long-term efficacy of GTX-102 in improving cognitive function, communication, and motor skills.
  • How is GTX-102 administered to patients? GTX-102 is administered via intrathecal (IT) lumbar puncture (LP). This means the medication is injected directly into the fluid surrounding the spinal cord.
  • What is the role of glucose, anhydrous in this trial? Glucose, anhydrous is one of the active substances in the solution used in this trial. It is part of a buffered intrathecal electrolyte/dextrose injection called ELLIOTTS B SOLUTION, which is used alongside GTX-102.
  • How long does the trial last? The trial is a long-term extension study. Patients may remain in the study until GTX-102 is approved and/or becomes available in their geographical region.
  • What are the main outcomes being measured in this trial? The main outcomes being measured are treatment-emergent adverse events (AEs) and serious adverse events (SAEs), as well as changes in cognitive, communication, and motor skills using the Bayley-4 assessment tool.

Ongoing Clinical Trials on Glucose, Anhydrous

  • A study testing ASP3082 in patients with advanced or spreading non-small cell lung cancer and pancreatic ductal adenocarcinoma

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Spain

Glossary

  • Angelman Syndrome: A rare genetic disorder that affects the nervous system and causes developmental delays, intellectual disability, speech problems, and movement issues.
  • Intrathecal: A route of administration where medication is injected directly into the fluid surrounding the spinal cord.
  • Lumbar Puncture (LP): A medical procedure where a needle is inserted between two vertebrae in the lower back to collect cerebrospinal fluid or to inject medication.
  • GTX-102: The investigational drug being studied for the treatment of Angelman Syndrome in this clinical trial.
  • Glucose, Anhydrous: A form of sugar without water molecules, used as one of the active substances in the solution administered during the trial.
  • Bayley-4: An assessment tool used to measure cognitive, communication, and motor skills in young children.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease that occurs during a clinical trial, whether or not it is related to the treatment being studied.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization, causes persistent disability, or leads to birth defects.
  • Long-Term Extension (LTE) Study: A type of clinical trial that continues to monitor and treat patients after the initial study has ended, to gather more data on long-term safety and efficacy.
  • ELLIOTTS B SOLUTION: A buffered intrathecal electrolyte/dextrose injection used in the trial, which contains glucose, anhydrous among other substances.

References

  1. http://clinicaltrials.eu/trial/study-on-the-long-term-safety-of-gtx-102-for-patients-with-angelman-syndrome/