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Study on the Safety of LY3884961 for Patients with Type 1 Gaucher Disease

Verified siteRegistered drugNo 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 Type 1 Gaucher Disease, which is a condition that affects certain parts of the body but not the brain. The study will test a new treatment called LY3884961, which is given as a single dose through an intravenous infusion, meaning it is delivered directly into the bloodstream. The main goal of the study is to see how safe and tolerable this treatment is for patients with this type of Gaucher Disease.

During the study, participants will receive a single dose of LY3884961 and will be monitored for any side effects or changes in their health. This includes regular checks of vital signs, blood tests, and imaging tests like MRIs to look at the abdomen and bones. The study will also track changes in certain proteins and antibodies in the blood, which can help understand how the body is responding to the treatment.

The study will take place over several years, with participants being followed up regularly to monitor their health and any long-term effects of the treatment. The researchers will also look at how the treatment affects the size of the spleen, the number of platelets in the blood, and the activity of certain enzymes related to Gaucher Disease. This information will help determine if LY3884961 could be a beneficial treatment option for people with Type 1 Gaucher Disease.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, genetic mutations, and current treatment stability.

    Participants must have been on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years and on a stable dose for at least three months.

  2. Step 2

    Consent and preparation

    Participants provide informed consent, agreeing to comply with study requirements and restrictions.

    Participants must agree to use effective contraception and abstain from blood donations for the first year.

  3. Step 3

    Medication administration

    A single intravenous dose of LY3884961 is administered to evaluate safety and tolerability.

    This medication is delivered through an intravenous infusion.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for any adverse effects, including changes in vital signs, laboratory parameters, and physical examinations.

    Regular assessments include 12-lead ECGs, abdominal and bone MRIs, and measurements of waist circumference and weight.

  5. Step 5

    Evaluation of treatment effects

    The study evaluates changes in complement proteins and the presence of specific antibodies.

    Changes in spleen volume, platelet count, and enzyme activity are also assessed.

  6. Step 6

    Long-term follow-up

    Participants are followed up to determine the time from LY3884961 administration to discontinuation of ERT/SRT.

    The study also monitors the time from discontinuation to potential re-initiation of ERT/SRT.

Who can join the trial?

6 criteria

  • Must be between the ages of 18 and 65 at the time of giving consent to participate.
  • Must have two copies of a specific gene change called GBA1 mutations, which will be confirmed by the study team.
  • Must have been receiving treatment called ERT (Enzyme Replacement Therapy) or SRT (Substrate Reduction Therapy) for at least 2 years and be on a stable dose for at least 3 months before the study starts.
  • Must be able to understand and sign a document that explains the study and agree to follow the study rules.
  • Both females and males can participate. Those who can have children must use a very effective method to prevent pregnancy during the study and follow-up period.
  • Must agree not to donate blood for at least the first year of the study.

Who cannot join the trial?

8 criteria

  • Patients with any other serious health condition that might interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients who have a history of allergic reactions to similar treatments.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients with a history of drug or alcohol abuse.
  • Patients who have received certain medications that might affect the study results.
  • Patients with any condition that the study doctors believe would make participation unsafe.
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Investigated drugs

LY3884961 is an investigational medication being studied for its potential to treat Gaucher Disease, which is a condition that affects various parts of the body. This medication is given as a single dose through an intravenous infusion, meaning it is administered directly into the bloodstream. The main goal of the study is to assess how safe and tolerable this medication is for patients who have symptoms of Gaucher Disease affecting areas outside the central nervous system.

What is already known about the treatment

LY3884961 – This medication is administered as a single intravenous dose and is currently being studied in a Phase 1/2 clinical trial. It is being evaluated for its safety and tolerability in individuals with Type 1 Gaucher Disease, specifically targeting peripheral or non-neuronopathic manifestations. The main therapeutic indication is to address symptoms associated with this form of Gaucher Disease. At the molecular level, LY3884961 is designed to interact with specific biological pathways to alleviate disease symptoms, although detailed mechanisms are still under investigation. It falls under the pharmacological classification of investigational drugs, as it is not yet approved for general medical use.

Investigated diseases

Type 1 Gaucher Disease – This is a genetic disorder caused by a deficiency in the enzyme glucocerebrosidase, leading to the accumulation of fatty substances in certain organs, particularly the spleen and liver. It is characterized by symptoms such as enlarged liver and spleen, low blood platelet counts, and bone abnormalities. The disease does not affect the nervous system, distinguishing it from other types of Gaucher disease. Over time, individuals may experience fatigue, bleeding, and bone pain or fractures. The progression of the disease can vary widely among individuals, with some experiencing mild symptoms and others more severe complications.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2022-500281-10-02Protocol codeJ3Z-MC-OJAEEstimated enrolment15 patientsSponsorPrevail Therapeutics Inc.

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