Table of contents
Trial overview
The available trial is an open-label extension study called “Open-Label Extension Study to Provide Access to ATH434 in Patients with Multiple System Atrophy.”[1] It is authorised and includes 7 participants.[1]
The study is looking at 5,7-DICHLORO-2-((ETHYLAMINO)METHYL)-8-HYDROXY-3-METHYLQUINAZOLIN-4(3H)-ONE METHANESULFONATE in people with multiple system atrophy, with the main focus on long-term safety and tolerability.[1]
Who is being studied
The target population is people with multiple system atrophy.[1] The trial data do not provide more detailed inclusion or exclusion rules, so only the condition and the small study size are known from the source.[1]
The intervention listed in the trial is ATH434-DP2, given by oral use, and the study is interventional, meaning participants receive a study treatment as part of the research.[1]
Study design and phase
This is a Phase 2 trial.[1] Phase 2 studies are usually used to learn more about safety and early treatment effects in a smaller group of participants.
The study is also an extension study, which means it follows earlier research and continues treatment access while collecting more information over time.[1] Because it is open-label, participants and researchers know what treatment is being given.[1]
What the study measures
The primary outcome is long-term safety and tolerability.[1] In simple terms, this means researchers want to see how safe the treatment is over time and how well people can continue taking it.
The study measures several safety-related outcomes, including the incidence and severity of adverse events and serious adverse events.[1] Adverse events are unwanted health problems that happen during a study, and serious adverse events are more severe problems that may need urgent care.
Researchers will also track changes in laboratory values and vital signs, including orthostatic measures.[1] Orthostatic measures are checks of body signals when a person changes position, such as standing up, and they can help show whether the body is responding safely.
Other measured outcomes include exposure to ATH434, treatment discontinuations due to adverse events, and deaths.[1] These measures help show how much treatment was taken and whether safety concerns affected continued use.
Important terms
Authorised means the trial has been approved to run.[1] Interventional means the study gives a treatment to participants as part of the research.[1]
Open-label means there is no blinding, so everyone knows which treatment is being used.[1] Extension study means the research continues after an earlier study period so more information can be collected.[1]
