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APAZUNERSEN

Clinical trials for APAZUNERSEN are not listed in the source data provided here. The trials shown instead study GTX-102 in people with Angelman syndrome, with goals such as safety and efficacy. This article summarizes those trial designs, target groups, phases, and main outcomes.

Table of Contents

Trial overview

The source data provided for this article does not list APAZUNERSEN trials. It lists two authorised clinical trials of GTX-102 in people with Angelman syndrome.[1][2]

Both studies are interventional trials, which means the researchers are giving a study treatment and measuring what happens over time.[1][2]

Patient groups and study setting

One trial is a long-term extension study for patients with Angelman syndrome.[1]

The other trial is for subjects with deletion-type or nondeletion-type Angelman syndrome, which are two forms of the same condition.[2]

In simple terms, a long-term extension study follows people for a longer time so researchers can learn more about the treatment after the earlier study phase.[1]

Study phases and design

The first trial is a Phase 3 study with an enrollment of 98 participants.[1]

The second trial is a Phase 2 study with an enrollment of 63 participants.[2]

Phase 2 trials usually look at early signs of benefit and safety, while Phase 3 trials are later studies that often include more people and help confirm results over a longer period.[1][2]

Main outcomes being measured

Both studies measure treatment-emergent adverse events and serious adverse events, which are unwanted medical events that happen after treatment begins.[1][2]

The Phase 3 extension study measures the frequency, severity, and relationship of these events to the investigational product throughout the study.[1]

The Phase 2 study measures safety across all subprotocols and also measures efficacy, which means whether the treatment may help with symptoms or function.[2]

The Phase 2 study uses several outcome tools to look at cognition, communication, behavior, sleep, and motor function.[2]

Examples of these tools include the Bayley-4 Cognitive raw score, Vineland-3 Communication scores, ABC-C behavior scores, and ASA sleep and motor ratings.[2]

Trial-by-trial summary

Trial 2024-510917-14-00 is a Phase 3 long-term extension study in 98 people with Angelman syndrome.[1] Its main goal is to evaluate the long-term safety profile, with special attention to treatment-emergent adverse events and serious adverse events.[1]

Trial 2024-519393-39-00 is a Phase 2 safety and efficacy study in 63 people with deletion-type or nondeletion-type Angelman syndrome.[2] It studies safety in all subprotocols and measures efficacy through several developmental and functional outcomes at Day 338 or TxD 338, depending on the subprotocol.[2]

In the source data, both studies are marked Authorised, which means they have approval to proceed in the listed setting.[1][2]

Important terms explained

Primary outcome means the main result the researchers want to measure first.[1][2]

Baseline means the starting point before treatment effects are measured.[2]

Change from Baseline means the study compares later test results with the starting test result.[2]

Net response means the overall response measured across several areas at the study time point named in the protocol.[2]

Subprotocol means a smaller part of one larger study, where different groups may have different endpoints or assessments.[2]

Trial ID Phase Condition studied Status Enrollment
2024-510917-14-00 Phase 3 Angelman syndrome Authorised 98
2024-519393-39-00 Phase 2 Angelman syndrome Authorised 63

FAQ

  • What clinical trials are described in the source data? The source data describes two interventional trials in Angelman syndrome. One is a Phase 3 long-term extension study, and the other is a Phase 2 safety and efficacy study.
  • Who can take part in these trials? The trials are for people with Angelman syndrome. One study includes patients with deletion-type or nondeletion-type Angelman syndrome.
  • What are the trials trying to learn? They are mainly studying safety and efficacy. Safety means how often side effects or other unwanted events happen, and efficacy means whether the treatment helps.
  • What phases are the trials in? One trial is Phase 3 and one trial is Phase 2. Phase 2 studies often look at both safety and early signs of benefit, while Phase 3 studies usually look at larger-scale safety and effectiveness.
  • What outcomes are being measured? The studies measure treatment-emergent adverse events and serious adverse events. They also measure changes in areas such as cognition, communication, behavior, sleep, and motor function.

Ongoing Clinical Trials on APAZUNERSEN

  • A Study Testing the Safety and How Well GTX-102 Works in Adults and Children with Angelman Syndrome

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France Italy Portugal

  • 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 genetic condition studied in both trials. The source data says the studies include people with this condition, including deletion-type and nondeletion-type forms in one trial.
  • Interventional study: A clinical trial where researchers give a treatment or procedure to see what happens. These trials test GTX-102 rather than just observing patients.
  • Phase 2: An early-to-mid stage trial. It often looks at both safety and whether the treatment may help.
  • Phase 3: A later-stage trial. It usually includes more participants and looks closely at safety and benefit over a longer period.
  • Long-term extension trial: A study that follows patients for a longer time after an earlier trial. It helps researchers learn about longer-term safety and effect.
  • Safety endpoint: A main result that focuses on harm or unwanted events. In these trials, this includes treatment-emergent adverse events and serious adverse events.
  • Efficacy endpoint: A main result that shows whether the treatment works. The trials measure this using tests of cognition, communication, behavior, sleep, and motor function.
  • Treatment-emergent adverse events (TEAEs): Unwanted medical events that start or get worse after treatment begins.
  • Serious adverse events (SAEs): A type of unwanted medical event that is more severe or more serious than usual side effects.
  • Cognition: Thinking skills such as learning, understanding, and memory.
  • Communication: How a person understands and uses language or other ways to express needs and ideas.
  • Motor function: Body movement skills such as sitting, standing, walking, or other physical actions.

References

  1. https://clinicaltrials.gov/study/2024-510917-14-00
  2. https://clinicaltrials.gov/study/2024-519393-39-00