In short
Clinical trials are investigating 2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE in people with symptomatic and pre-symptomatic Huntington's disease. These studies aim to see whether imaging and cognitive biomarkers can measure disease progression well over time. The main focus is on trial methods and endpoints rather than treatment benefit.
Key points
- Clinical trials of 2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE are focused on Huntington's disease. The available study includes people with symptomatic and pre-symptomatic disease. It is a Phase 3 interventional trial with 100 participants. The main goal is to identify biomarkers that can measure disease progression over 2 years. The primary endpoint uses effect size, measured by standardized mean difference, also called Cohen's d.
Clinical trial overview
The available study is an interventional clinical trial, which means researchers are actively testing a study intervention in participants. The trial is authorised and is focused on Huntington's disease.
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. The brief summary says the goal is to identify biomarkers, used alone or together, that can measure progression from the pre-symptomatic stage onward.
Who can take part
The study includes people with symptomatic and pre-symptomatic Huntington's disease. 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.
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. 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. 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. 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. 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. 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. 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. Its purpose is to identify biomarkers that can better measure disease progression from the early stages onward.