Table of Contents
- Clinical trial overview
- Who can take part
- What the study is trying to do
- Trial phase and design
- Main endpoint
- Trial summary
Clinical trial overview
The available study is an interventional clinical trial, which means researchers are actively testing a study intervention in participants.[1] The trial is authorised and is focused on Huntington’s disease.[1]
The trial investigates 2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE as part of a biomarker study for disease progression.[1] The brief summary says the goal is to identify biomarkers, used alone or together, that can measure progression from the pre-symptomatic stage onward.[1]
Who can take part
The study includes people with symptomatic and pre-symptomatic Huntington’s disease.[1] Symptomatic means the person already has signs of the disease, while pre-symptomatic means the disease is present but clear symptoms have not started yet.
The source data does not list extra eligibility details such as age limits or test results.[1]
What the study is trying to do
The main aim is to find biomarkers that can measure Huntington’s disease progression in a sensitive way.[1] A biomarker is a measurable sign that can help researchers follow changes in a disease over time.
The study looks at imaging and cognitive biomarkers.[1] Imaging biomarkers come from scans or pictures of the body, and cognitive biomarkers relate to thinking skills such as memory and attention.
Trial phase and design
This is a Phase 3 trial.[1] Phase 3 studies usually involve larger groups and help researchers see how well a study approach works in a broader patient population.
The planned enrollment is 100 participants.[1] The trial is designed as a clinical research study, not a routine care program.
Main endpoint
The primary endpoint is effect size, measured by the standardized mean difference, also called Cohen’s d.[1] This tells researchers how large the change is in each biomarker over time.
The endpoint compares biomarker values measured at the start of the study with the values at the 2-year follow-up.[1] This helps show whether the biomarkers can track disease progression across two years.
Trial summary
The single listed study, NCT05808153, is an authorised Phase 3 interventional trial in 100 people with symptomatic or pre-symptomatic Huntington’s disease.[1] Its purpose is to identify biomarkers that can better measure disease progression from the early stages onward.[1]
