Active not recruitingPHASE2INTERVENTIONAL

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

The GENERATION HD2 study is investigating a potential new treatment, tominersen, for Huntington's disease. It focuses on individuals in the very early stages of the condition. The main goals are to understand if tominersen is safe, how it changes certain body markers linked to Huntington's, and if it can help improve or slow down the disease's progression. Participants will receive either tominersen or a placebo (a dummy drug) to allow for a fair comparison. This is a vital step in finding effective treatments and aims to gather crucial information about this new approach to managing Huntington's disease.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Hoffmann-La Roche

Enrolment target

301

Start

03 Feb 2023

Estimated completion

01 Apr 2027

Huntington Disease

What is this study about?

Huntington's disease is a condition that affects the brain and can lead to problems with movement, thinking, and mental health. This study, called GENERATION HD2, is exploring a new treatment option called tominersen. Think of tominersen as a specially designed medicine that aims to target the underlying cause of Huntington's in the body.

The main purpose of this research is to see how safe tominersen is for people with Huntington's disease and to understand its effects. Researchers will be looking at 'biomarkers' – these are like clues in your body (e.g., in blood or spinal fluid) that can tell them if the drug is working and how it's affecting the disease process. They'll also be checking if tominersen can help with the symptoms or slow down the progression of the condition in its early stages.

To get clear results, some participants will receive tominersen, while others will receive a placebo. A placebo is a dummy treatment that looks exactly like the real drug but contains no active medicine. This allows researchers to compare the effects of tominersen against what happens naturally or with no active treatment. This type of study is crucial for determining if a new medicine is effective and safe before it can potentially be made available more widely.

Key takeaways

This study is for people in the very early stages of Huntington's disease.
It's testing a new drug called tominersen against a dummy drug (placebo).
Researchers will check safety, body markers (biomarkers), and potential benefits.
Participation involves regular clinic visits and monitoring.
You'll need a study companion to support you.
You can withdraw from the study at any time.

Who may be eligible?

This study is looking for people aged between 25 and 50 years who have been diagnosed with Huntington's disease or are showing very early signs of the condition, even before a full diagnosis. You would need to have specific genetic test results that suggest you're in the early stages of the condition.

There are also some general health requirements, such as weighing more than 40 kilograms and having a body mass index (BMI) within a certain range. You would also need someone to join you as a 'study companion' to support you throughout the research.

You wouldn't be able to join if you've previously used similar types of experimental treatments for Huntington's, if you're taking certain blood-thinning medications, or if you've had certain surgeries or health issues like hydrocephalus (fluid on the brain). Pregnant or breastfeeding individuals, or those planning to become pregnant, are also unable to take part.

Quick self-check

Are you between 25 and 50 years old?
Have you been diagnosed with early Huntington's or are in the very early stages?
Do you have a specific genetic test result called a 'CAP score' within the 400-500 range?
Are you generally in good health with a body weight over 40kg and a BMI between 18-32?
Do you have someone who can act as your 'study companion'?
Are you not currently pregnant, breastfeeding, or planning to be during the study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

Taking part in this study involves receiving regular doses of the study drug, which will either be tominersen or a placebo. You won't know which one you are receiving. It will also involve regular visits to the study clinic for various assessments. These assessments will help doctors monitor your health, check for any side effects, and see how the drug might be affecting your condition. This could include physical examinations, blood tests, neurological assessments, and possibly other tests to look at those 'biomarkers'.

There will be an initial period where you'll receive either the study drug or placebo. If you complete this part of the study, you might then be invited to continue into an 'open-label extension' period where everyone receives tominersen. Throughout the study, there will be follow-up appointments to ensure your safety and monitor your progress. The total duration of your involvement, including all visits and follow-up, will be explained in detail by the study team.

Potential risks and benefits

Participating in a clinical trial offers the potential benefit of accessing an investigational treatment before it's widely available, which in some cases could help manage your condition. It also contributes vital information that can help countless others in the future. However, there are potential risks, including side effects from the study drug, which may be mild or more serious, and the possibility that the treatment may not be effective for you. You also might receive a placebo and not the active drug during the initial phase. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (70)

Uab Medicine
Birmingham, United States
Barrow Neurological Institute
Phoenix, United States
University of California Davis Medical System
Sacramento, United States
CenExel Rocky Mountain Clinical Research, LLC
Englewood, United States
Georgetown University
Washington D.C., United States
University of Florida
Gainesville, United States
University of South Florida
Tampa, United States
Northwestern University
Chicago, United States
University of Iowa Hospitals and Clinics
Iowa City, United States
John Hopkins University School of Medicine
Baltimore, United States
Beth Israel Deaconess Medical Center
Boston, United States
Henry Ford Hospital
Detroit, United States

+58 more sites — see the official record for the full list.

Common questions

What is Tominersen?

Tominersen is an experimental drug designed to target the underlying cause of Huntington's disease. This study is testing its safety and effectiveness.

What is a placebo?

A placebo is a 'dummy' treatment that looks like the study drug but contains no active medicine. It helps researchers fairly compare the real drug's effects.

What are 'biomarkers'?

Biomarkers are measurable clues in your body, like those found in blood or spinal fluid, that can tell doctors if a disease is present or how a treatment is working.

Who is a 'study companion'?

A study companion is someone who supports you throughout your participation in the trial. They are usually a close friend or family member.

Can I stop participating if I want to?

Yes, you have the right to stop participating in the study at any time, for any reason, without it affecting your future medical care.