Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease

This research study is investigating a new gene therapy called AB-1001 for adults with early-stage Huntington's disease. Gene therapy aims to address the root cause of the disease. In this study, AB-1001 is given directly into specific areas of the brain. The main goals are to find out if the treatment is safe, how well people tolerate it, and if there are any early signs that it might be effective in improving symptoms. The study involves two main parts: first, a small group of participants will help researchers find the best dose. Then, once a safe dose is identified, more participants will receive this chosen dose. This is an early-stage study, meaning it's one of the first times this treatment is being tested in humans.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

AskBio France, SAS, a subsidiary of AskBio Inc.

Enrolment target

Start

12 Oct 2022

Estimated completion

01 Apr 2028

Huntington Disease

What is this study about?

Huntington's disease is a condition that affects the brain, causing difficulties with movement, thinking, and mood. This study is exploring a new approach to treating early-stage Huntington's disease using something called gene therapy. Gene therapy works by introducing new genetic material into the body to help correct or replace faulty genes that cause diseases. In this particular study, the gene therapy, named AB-1001, is delivered directly into specific parts of the brain.

The main purpose of this study is to carefully check if AB-1001 is safe for people with Huntington's disease and if their bodies can handle the treatment well. Researchers will also be looking for any early signs that the treatment might be making a difference to the disease. This type of study, called a Phase I/II study, is usually one of the first times a new treatment is tested in humans after being studied in laboratories.

Researchers will start by testing two different amounts (doses) of the treatment in a small number of people to figure out which dose is the safest and most suitable. Once a good dose is found, more people will join the study to receive that specific dose. The hope is that this gene therapy could one day offer a new way to help people living with Huntington's disease.

Key takeaways

This study tests a new gene therapy (AB-1001) for early Huntington's disease.
The treatment is given directly into the brain via surgery.
The main goals are to check safety, tolerability, and early signs of effectiveness.
It's a Phase I/II study, focusing on finding the right dose.
The total study commitment could be around five years.
Participation involves a screening period, surgery, and long-term follow-up visits.

Who may be eligible?

To join this study, you need to be an adult between 18 and 65 years old. You must have a confirmed diagnosis of Huntington's disease through a genetic test and be in the early stages of the disease, as determined by specific medical scores. You'll also need to be able to understand and agree to all the study procedures.

There are also certain criteria related to your brain scans. Specifically, areas of your brain called the 'putamen' and 'caudate' need to be of a certain size. If you're taking other medications for Huntington's disease, they need to have been stable for at least 30 days before you start the study.

However, some things would prevent you from taking part. For example, if you have other serious medical conditions like certain cancers, or significant heart, kidney, or liver problems. If you have a severe untreated mood disorder or a history of recent suicide attempts, you wouldn't be able to join. Also, if you've had previous brain surgery for other experimental treatments, or a stroke, or certain brain conditions, you would not be eligible. The medical team will ultimately decide if it's safe for you to participate, especially regarding suitability for the surgical procedure.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 65 years old?
Do you have a genetically confirmed diagnosis of Huntington's disease?
Are you in the early stages of Huntington's disease (as determined by your doctor)?
Are you able to undergo brain surgery?
Have you been on stable medication for Huntington's for at least 30 days?
Do you have any serious underlying health conditions like certain cancers, severe heart, kidney, or liver problems, or serious untreated mood disorders?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the process will involve a few key steps over a long period. First, there's a screening phase that can last between 8 and 12 weeks. During this time, you'll have various tests and evaluations to make sure you meet all the study requirements.

If you're eligible, you'll then enter a treatment phase. This involves a one-time surgical procedure where the AB-1001 gene therapy is carefully injected into specific parts of your brain (the striatum) on both sides. After the surgery, you'll have an initial follow-up period that lasts for 52 weeks (about one year). During this time, you'll have regular hospital visits for assessments to check how you're doing, the safety of the treatment, and if there are any signs it's working.

Following the first year, there will be a much longer follow-up phase, lasting for four years. This means the total commitment to the study, if you participate fully, could be about five years. Throughout this time, you'll continue to have check-ups, though likely less frequently in the later stages, to monitor your health and the long-term effects of the treatment.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks to consider. Potential benefits are not guaranteed, but people taking part may contribute valuable information that could help others with Huntington's disease in the future. In some cases, participants might experience an improvement in their condition, but this is not certain, and the main goal of this early study is to check safety. Potential risks include those associated with brain surgery itself, such as infection, bleeding, or other complications. There may also be side effects from the gene therapy, some of which might not be known yet. You will be closely monitored throughout the study for any issues. Remember, you can withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Institut du Cerveau (ICM), Hôpital La Pitié Salpêtrière APHP
Verified postcode
Paris, France

Common questions

What is gene therapy?

Gene therapy is a new type of treatment that aims to fix faulty genes that cause diseases. In this study, it involves introducing new genetic material into the brain.

What does 'early manifest' Huntington's disease mean?

It means you have a confirmed diagnosis of Huntington's disease and are experiencing initial noticeable symptoms, but the disease has not yet advanced significantly.

Will I know if I'm getting the active treatment?

Yes, this is an 'open-label' study, which means both you and the study team will know you are receiving the active AB-1001 gene therapy.

What does a Phase I/II study mean?

This means it's an early-stage study primarily focused on checking the treatment's safety and finding the right dose in humans, while also looking for early signs it might be effective.

How long will the study last for each participant?

If you join, the study involves a screening period, then treatment, followed by about a year of close follow-up, and then a longer four-year follow-up. The total commitment could be around five years.