Not recruitingRare disease

Study on the Safety and Effects of AMT-130 for Adults with Early Huntington's Disease

Investigational

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Huntington's Disease, a genetic disorder that affects the brain and leads to the progressive breakdown of nerve cells. The treatment being tested is called ifezuntirgene inilparvovec, also known by its code name AMT-130. This treatment is designed to lower the levels of a harmful protein called huntingtin, which is involved in the development of Huntington's Disease. The treatment uses a method called RNA interference to target and reduce the production of this protein in specific areas of the brain.

The purpose of the study is to evaluate the safety and tolerability of this new treatment in adults with early-stage Huntington's Disease. Participants in the study will receive the treatment through a procedure that delivers it directly into the brain. The study will monitor participants over time to observe any changes in their condition and to assess the treatment's effects on the disease. Some participants may receive a placebo, which is a substance with no active treatment, to help compare the results.

Throughout the study, various health checks will be conducted, including monitoring vital signs, performing electrocardiograms (ECG), and conducting physical and neurological examinations. Blood and urine tests will also be done to ensure the safety of the participants. The study will also look at changes in brain structure using magnetic resonance imaging (MRI) and will measure the levels of the treatment in the body. The trial is expected to continue until the end of 2029, with the aim of gathering comprehensive data on the treatment's safety and potential benefits for people with Huntington's Disease.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and current medications.
A series of tests are performed, including a magnetic resonance imaging (MRI) scan to measure specific brain areas.
Step 2
Treatment administration
The treatment involves the administration of ifezuntirgene inilparvovec, a solution delivered directly into the brain. This is done through a procedure known as intracerebral use.
The goal is to lower the levels of the huntingtin gene, which is associated with Huntington's Disease.
Step 3
Monitoring and follow-up
After the treatment, regular follow-up visits are scheduled to monitor health and any changes in symptoms.
These visits include physical and neurological examinations, as well as laboratory tests to check blood and cerebrospinal fluid (CSF).
Step 4
Safety and efficacy evaluation
Throughout the trial, the safety and effectiveness of the treatment are evaluated. This includes monitoring for any side effects and changes in vital signs.
Additional assessments involve imaging studies to observe any changes in brain structure.
Step 5
Completion of trial
The trial is expected to continue until the end of 2029. Participants will be informed of their progress and any findings related to their health.
Final evaluations are conducted to summarize the effects of the treatment and overall health status.

Who can join the trial?

8 criteria

Must be able and willing to provide written consent before starting the study or any related procedures.
Participants should be between 25 and 65 years old.
For Cohorts 1 & 2: Must have early signs of Huntington's Disease, shown by a specific score (UHDRS TFC score of 9 to 13) and either a DCL of 4 or a DCL of 3 with certain conditions. For Cohort 3: Must have early signs of Huntington's Disease, shown by a specific score (UHDRS TFC score of 11 or more) and either a DCL of 4 or a DCL of 3 with certain conditions.
Must have a specific genetic test result showing 40 or more CAG repeats in the HTT gene.
Must meet certain brain scan (MRI) size requirements: Putamen must be 2.5 cm³ or larger on each side, and Caudate must be 2.0 cm³ or larger on each side.
All medications for Huntington's Disease symptoms must be stable for 3 months before the study starts, with no changes needed due to symptoms.
Must be able and willing to follow all study procedures and attend all scheduled visits.
All female participants who can have children must have a negative pregnancy test at the start and not be breastfeeding. Both females and males must use a highly effective birth control method.

Who cannot join the trial?

5 criteria

Individuals who do not have a diagnosis of Huntington's Disease cannot participate. Huntington's Disease is a condition that affects the brain and causes movement, thinking, and emotional problems.
Participants must be adults, meaning they are 18 years or older.
Only those with early signs of Huntington's Disease are eligible. This means the disease has started to show symptoms but is not in the advanced stages.
Both men and women can participate, but they must meet all other criteria.
People who are considered part of a vulnerable population, such as those unable to give consent or those with certain disabilities, are not eligible.

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Investigated drugs

AMT-130 is a gene therapy designed to lower the levels of the huntingtin protein, which is involved in Huntington's Disease. This therapy is administered directly into the brain, specifically targeting the striatum, a region affected by the disease. The goal of AMT-130 is to reduce the production of the harmful protein, potentially slowing the progression of the disease and improving symptoms.

Ifezuntirgene Inilparvovec

What is already known about the treatment

AMT-130 – This medication is administered directly into the brain through a surgical procedure, specifically targeting the striatum, a region affected in Huntington's Disease. Currently, AMT-130 is in the clinical trial phase, being studied for its safety and effectiveness in treating early-stage Huntington's Disease. The main therapeutic indication is to lower the levels of the huntingtin protein, which is believed to cause the symptoms of the disease. At the molecular level, AMT-130 works by using a viral vector to deliver microRNA that reduces the production of the huntingtin protein. It is classified as a gene therapy, aiming to modify the genetic material to treat or prevent disease.

Investigated diseases

Huntington's Disease – Huntington's Disease is a genetic disorder that affects the brain, leading to the progressive breakdown of nerve cells. It typically begins in adulthood and is characterized by movement disorders, cognitive decline, and psychiatric symptoms. As the disease progresses, individuals may experience involuntary movements, difficulty with coordination, and changes in mood and behavior. Cognitive abilities, such as thinking and reasoning, gradually deteriorate, impacting daily functioning. The symptoms usually worsen over time, affecting the individual's ability to perform everyday tasks. The progression of the disease varies among individuals, but it generally leads to significant impairment in motor and cognitive functions.