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Study on the Safety and Effects of LY3884963 for Patients with Fronto-Temporal Dementia with Progranulin Mutations

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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 a condition known as Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN). This is a type of dementia that affects the brain, leading to changes in behavior, language, and movement. The study is testing a new treatment called LY3884963, which is an investigational gene therapy. This therapy uses a special viral vector to deliver a gene into the patient's cells, aiming to increase levels of a protein called progranulin, which is important for brain health.

The purpose of the study is to evaluate the safety and effects of different doses of LY3884963 on progranulin levels in patients with FTD-GRN. Participants will receive the treatment through an injection into a specific area at the base of the skull. The study will monitor the participants over time to see how their progranulin levels change in their blood and cerebrospinal fluid, which is the fluid surrounding the brain and spine. Some participants may also receive other medications like Sirolimus or Rituximab to help manage their condition.

Throughout the study, researchers will keep track of any side effects or changes in the participants' health. They will also use imaging techniques like MRI to check for any changes in the brain and spine. The study aims to provide valuable information on how safe and effective LY3884963 is for treating FTD-GRN, potentially leading to new treatment options for this challenging condition.

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 is conducted to confirm eligibility. This includes reviewing medical history and current health status.
Eligibility criteria include age between 30 and 85 years, a reliable study partner, and a confirmed mutation in the progranulin gene.
Step 2
Baseline measurements
Baseline measurements are taken to establish initial health parameters. This includes blood tests and possibly imaging studies.
These measurements help in monitoring changes throughout the study.
Step 3
Medication administration
The study involves the administration of the investigational product LY3884963 via suboccipital injection into the cisterna magna. This is a space in the lower part of the brain.
The study evaluates three different dose levels to assess safety and effects on progranulin levels.
Step 4
Additional medications
Participants may receive additional medications such as prednisone and sirolimus in tablet form. Prednisone is administered orally, while sirolimus is also taken orally in coated tablet form.
Methylprednisolone may be administered intravenously as a solution for injection.
Step 5
Monitoring and follow-up
Regular monitoring is conducted to assess the safety and effects of the treatment. This includes tracking any adverse events and changes in health status.
Blood and cerebrospinal fluid (CSF) samples are collected periodically to measure progranulin levels and other biomarkers.
Step 6
End of study evaluation
At the end of the study, a comprehensive evaluation is performed to assess the overall impact of the treatment.
This includes a final review of health status, any changes in symptoms, and the results of laboratory tests.

Who can join the trial?

13 criteria

Men or women aged 30 to 85 years can participate.
The patient must have a reliable study partner, like a family member or friend, who can help provide information about the patient's health and abilities.
The patient should be able to walk without needing a walker or wheelchair.
The patient should live in the community, not in a nursing home. Some assisted living situations might be allowed.
The patient needs to have received vaccines for pneumococcal pneumonia and shingles within the last 10 years. These can be given during the screening process but must be done at least 4 weeks before starting certain medications.
The patient should weigh between 88 pounds (40 kg) and 242 pounds (110 kg) and have a body mass index (BMI) between 18 and 34.
The patient must have symptoms of **frontotemporal dementia (FTD)**, which can include mild issues with behavior, thinking, movement, or language.
The patient needs to score between 0.5 and 15 on a specific test that measures dementia symptoms.
The patient should have been using any regular medications consistently for at least 8 weeks before starting the study medication.
The patient must carry a specific gene mutation related to **progranulin**, confirmed by a central laboratory.
The patient must test negative for **Mycobacterium tuberculosis (MTB)**, a bacteria that causes tuberculosis, or have a documented negative test within the past year.
All cancer screenings appropriate for the patient's age and gender should be up to date, as determined by the study doctor and local healthcare standards.
The patient or their legally authorized representative must understand the study's purpose and risks and provide written consent to participate and share health information.

Who cannot join the trial?

4 criteria

Individuals who do not have a diagnosis of Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) cannot participate. This is a specific type of dementia related to genetic changes.
Participants must be within a certain age range, which is not specified here, but typically means they must be adults.
Both males and females are eligible, but certain health conditions or factors might exclude someone.
People who are considered part of a vulnerable population may not be eligible. This term often refers to groups who might need special protection, like children, pregnant women, or those unable to give consent.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
Verified
Centre Hospitalier Universitaire De Lille
Lille, France
Responsive
Katholieke Universiteit te Leuven
Leuven, Belgium

Trial locations

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Investigated drugs

LY3884963 is a medication being studied for its potential effects on patients with Fronto-Temporal Dementia who have mutations in the progranulin gene. The trial aims to assess the safety and tolerability of this medication when administered through an injection into a specific area of the brain. Researchers are also interested in understanding how this medication affects the levels of progranulin, a protein, in the blood and cerebrospinal fluid of the patients.

What is already known about the treatment

LY3884963 – This medication is administered through a suboccipital injection into the cisterna magna, a space in the brain. It is currently being studied in clinical trials for its safety and effects on progranulin levels in patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN). The main therapeutic indication is to address this specific type of dementia. LY3884963 works by potentially modifying progranulin levels, which are important for brain health. It is classified under investigational drugs, as it is still in the research phase and not yet approved for general medical use.

Investigated diseases

Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) – This is a type of dementia that primarily affects the frontal and temporal lobes of the brain, leading to changes in personality, behavior, and language. It is associated with mutations in the progranulin gene, which result in reduced levels of the progranulin protein. The disease typically begins with subtle changes in behavior and personality, such as apathy, disinhibition, or compulsive behavior. As it progresses, individuals may experience difficulties with language, including speaking and understanding words. Over time, the disease can lead to more severe cognitive impairments, affecting memory and executive functions. The progression of symptoms can vary widely among individuals, with some experiencing rapid decline while others have a more gradual progression.