In short
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.
Key points
- Clinical trial data provided for this article does not list APAZUNERSEN. Instead, it describes two authorised trials of GTX-102 in Angelman syndrome. One is a Phase 3 long-term extension study with 98 participants, and the other is a Phase 2 study with 63 participants. The studies focus on safety and efficacy. Main outcomes include treatment-emergent adverse events, serious adverse events, and changes in cognition, communication, behavior, sleep, and motor function. One trial includes patients with deletion-type or nondeletion-type Angelman syndrome.
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.
Both studies are interventional trials, which means the researchers are giving a study treatment and measuring what happens over time.
Patient groups and study setting
One trial is a long-term extension study for patients with Angelman syndrome.
The other trial is for subjects with deletion-type or nondeletion-type Angelman syndrome, which are two forms of the same condition.
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.
Study phases and design
The first trial is a Phase 3 study with an enrollment of 98 participants.
The second trial is a Phase 2 study with an enrollment of 63 participants.
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.
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.
The Phase 3 extension study measures the frequency, severity, and relationship of these events to the investigational product throughout the study.
The Phase 2 study measures safety across all subprotocols and also measures efficacy, which means whether the treatment may help with symptoms or function.
The Phase 2 study uses several outcome tools to look at cognition, communication, behavior, sleep, and motor function.
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.
Trial-by-trial summary
Trial 2024-510917-14-00 is a Phase 3 long-term extension study in 98 people with Angelman syndrome. Its main goal is to evaluate the long-term safety profile, with special attention to treatment-emergent adverse events and serious adverse events.
Trial 2024-519393-39-00 is a Phase 2 safety and efficacy study in 63 people with deletion-type or nondeletion-type Angelman syndrome. 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.
In the source data, both studies are marked Authorised, which means they have approval to proceed in the listed setting.
Important terms explained
Primary outcome means the main result the researchers want to measure first.
Baseline means the starting point before treatment effects are measured.
Change from Baseline means the study compares later test results with the starting test result.
Net response means the overall response measured across several areas at the study time point named in the protocol.
Subprotocol means a smaller part of one larger study, where different groups may have different endpoints or assessments.