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Study on the Safety and Effects of LY3884961 for Patients with Parkinson's Disease and a GBA1 Mutation

InvestigationalNo placebo

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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 Parkinson's Disease in individuals who have at least one GBA1 mutation. The treatment being tested is called LY3884961, which is a solution for injection. This treatment involves using a special type of virus, known as an adeno-associated viral vector serotype 9, to deliver a modified version of the human GBA gene into the body. The purpose of the study is to find out how safe and effective this treatment is for people with Parkinson's Disease.

Participants in the study will receive the treatment through an injection into a specific area at the base of the skull, known as the cisterna magna. The study will look at two different dose levels of the treatment to see how the body responds. Researchers will monitor participants for any side effects and changes in their health. The study will also measure levels of certain substances in the blood and spinal fluid to understand how the treatment affects the body.

The trial aims to gather information over several years to ensure a thorough understanding of the treatment's safety and effects. Participants will be closely observed throughout the study to track any changes in their condition and overall health. This research is important for developing new ways to manage Parkinson's Disease, especially for those with specific genetic mutations like the GBA1 mutation.

The research process

The trial runs in 6 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 will be conducted to confirm eligibility. This includes verifying age, weight, and a diagnosis of Parkinson's disease with a specific genetic mutation.
A healthcare professional will review your medical history and current medications to ensure they have been stable for at least eight weeks prior to the study.
Step 2
Screening tests
Screening tests will be performed to check for specific health conditions. This includes a test for Mycobacterium tuberculosis and confirmation of a genetic mutation related to the study.
A blood test will be conducted to measure enzyme activity levels, which must be below a certain threshold for participation.
Step 3
Consent and partner involvement
You will be asked to provide written informed consent after understanding the purpose and risks of the study.
A reliable study partner, such as a family member or friend, will be involved to provide information on your health status and abilities.
Step 4
Treatment administration
The treatment involves a single administration of the investigational product, LY3884961, which is a solution for injection.
The injection is given via a procedure called intracisternal use, where the solution is injected into a specific area at the base of the skull.
Step 5
Monitoring and follow-up
After the injection, you will be monitored for any immediate reactions or side effects. This includes checking for any adverse events or changes in your health.
Regular follow-up visits will be scheduled to assess the safety and effects of the treatment. Blood and cerebrospinal fluid samples may be taken to measure enzyme activity and other markers.
Step 6
Study completion
The study will continue until the estimated end date in 2031, with periodic assessments to evaluate long-term safety and effectiveness.
Upon completion, a final assessment will be conducted to summarize the findings and any changes in your health status.

Who can join the trial?

10 criteria

Men or women aged 35 to 80 years.
Body weight between 40 kg (88 lbs) and 110 kg (242 lbs).
Diagnosis of Parkinson's Disease according to specific clinical criteria.
Parkinson's Disease at Stage III-IV when not taking medication. This refers to a specific level of disease progression.
Stable use of medications for at least 8 weeks before starting the study. This includes medications for Parkinson's Disease.
At least one confirmed genetic change (mutation) in the GBA1 gene, with low enzyme activity in the blood. Patients with two mutations or certain genetic forms of Parkinson's Disease are not eligible.
Negative test for Mycobacterium tuberculosis (a bacteria that causes tuberculosis).
Ability to understand the study's purpose and risks, and provide written consent to participate.
Have a reliable study partner, like a family member or friend, who can provide information about the patient's health and abilities.
Women who can have children must not be pregnant or breastfeeding.

Who cannot join the trial?

4 criteria

Patients who do not have **Parkinson's Disease** with at least one **GBA1 mutation** cannot participate. A **mutation** is a change in a gene, and **GBA1** is a specific gene related to Parkinson's Disease.
Patients who are not within the specified age range cannot participate. The age range includes adults and older adults.
Patients who are not willing or able to receive the treatment via **suboccipital injection** cannot participate. This is an injection given at the base of the skull into a fluid-filled space called the **cisterna magna**.
Patients who are part of a vulnerable population, such as those unable to give informed consent, may not be eligible.

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Investigated drugs

LY3884961 is a medication being studied for its potential to help people with Parkinson's Disease who have a specific genetic mutation known as GBA1. This medication is given through an injection into a part of the brain called the cisterna magna, which is located at the base of the skull. The main goal of this study is to see if LY3884961 is safe and how the body reacts to it. Researchers are also looking at how the immune system responds to the medication.

Adeno-Associated Viral Vector Serotype 9 Expressing Codon-Optimized Human Gba Gene

What is already known about the treatment

LY3884961 – LY3884961 is administered through a suboccipital injection, which means it is injected into the cisterna magna, a space at the base of the skull. This medication is currently being studied in clinical trials to assess its safety and effects, particularly for patients with Parkinson’s Disease who have at least one GBA1 mutation. The main goal of this study is to understand how well the medication is tolerated by patients and to observe any immune responses. LY3884961 is being explored for its potential to help manage symptoms of Parkinson’s Disease by targeting specific genetic mutations.

Investigated diseases

Parkinson’s Disease with GBA1 Mutation – Parkinson’s Disease is a progressive neurological disorder that affects movement. It is characterized by tremors, stiffness, and difficulty with balance and coordination. The disease occurs when nerve cells in the brain do not produce enough dopamine, a chemical that helps control movement. The presence of a GBA1 mutation can influence the progression and symptoms of Parkinson’s Disease. This mutation is associated with changes in the metabolism of certain lipids in the brain. Over time, individuals may experience worsening of motor symptoms and the development of non-motor symptoms such as cognitive changes.