Table of contents
- Trial overview
- Who can participate
- What is being studied
- Study phase and size
- Endpoints and measurements
- Patient glossary
Trial overview
The available study is an interventional clinical trial, which means researchers are actively testing a study intervention and then measuring the results.[1] It is authorised and focuses on people with Huntington’s disease, including both symptomatic and pre-symptomatic patients.[1]
The brief summary says the study aims to identify biomarkers that can measure the progression of Huntington’s disease in a sensitive way, including from the pre-symptomatic stage onward.[1]
Who can participate
The trial includes patients with symptomatic and pre-symptomatic Huntington’s disease.[1] This means the study is not limited to people who already have symptoms; it also includes people who may not yet show clear signs of the disease.[1]
The planned enrollment is 100 participants, so the study is designed for a relatively small group compared with very large trials.[1]
What is being studied
The intervention listed in the trial is 2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE, given as [18F]MNI-659 by intravenous administration at 5 µg.[1] The study data do not present this trial as a treatment trial for symptom relief; instead, the goal is to study biomarkers for tracking disease progression.[1]
In simple terms, a biomarker is a measurable sign that can help show how a disease is changing over time.[1] Here, the researchers want to know whether one biomarker or a combination of biomarkers can detect Huntington’s disease progression well.[1]
Study phase and size
This is a Phase 3 trial.[1] Phase 3 studies are later-stage clinical trials and are often used to test how well a research approach performs in a broader group of participants.[1]
The study plans to include 100 people.[1] The trial type is interventional, which means the study team is not just observing patients but is actively using the study intervention as part of the research plan.[1]
Endpoints and measurements
The primary endpoint is effect size, measured by the standardized mean difference, also called Cohen’s d.[1] This tells the researchers how large the change is in each biomarker over time.[1]
The trial compares biomarker values measured at the start of the study with the values measured at the 2-year follow-up.[1] The main goal is to see which biomarkers, alone or together, are most sensitive for measuring progression of Huntington’s disease in clinical trials.[1]
Patient glossary
Huntington’s disease is the condition being studied in this trial.[1] Symptomatic means the person already has symptoms, while pre-symptomatic means the person does not yet show symptoms.[1]
Standardized mean difference and Cohen’s d are ways to describe how big a change is in a study.[1] A follow-up is a later check to see what has changed after time has passed.[1]
