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Study on the Long-term Safety of FLT201 for Patients with Gaucher Disease Type 1

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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 Gaucher Disease Type 1, a genetic disorder that affects the body's ability to break down a certain type of fat, leading to its buildup in various organs. The study is investigating a treatment called FLT201, which is a special type of therapy using an adeno-associated viral vector. This vector is designed to deliver a modified gene that helps produce an enzyme called beta-glucocerebrosidase, which is deficient in people with Gaucher Disease. The treatment is given as a solution for infusion, meaning it is administered directly into the bloodstream.

The purpose of this study is to assess the long-term safety of FLT201 in individuals who have Gaucher Disease Type 1. Participants in the study will be monitored over an extended period to observe how their bodies respond to the treatment. This includes checking for any side effects and measuring changes in various health indicators, such as enzyme activity levels and organ sizes, using methods like MRI scans. The study will also track the presence of the treatment in the body over time.

Participants in this study are those who have previously received the FLT201 treatment. They will be required to attend regular follow-up visits to ensure their safety and to gather important data on the treatment's effects. The study aims to provide valuable information on the safety and effectiveness of this innovative therapy for managing Gaucher Disease Type 1.

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

    Joining the study

    Participation begins after receiving the FLT201 treatment in a previous study. This study focuses on long-term follow-up.

    Participants must provide informed consent and agree to comply with all study requirements.

  2. Step 2

    Treatment monitoring

    The study involves monitoring the long-term safety of FLT201, a solution for infusion used in treating Gaucher Disease Type 1.

    Participants will be observed for any treatment-emergent adverse events (TEAEs), which are any side effects that occur after the treatment.

  3. Step 3

    Health assessments

    Regular health assessments will be conducted, including checks on vital signs, heart activity through a 12-lead ECG, physical examinations, and laboratory tests.

    Changes in enzyme activity, specifically glucocerebrosidase (GCase), will be measured in blood samples over time.

  4. Step 4

    Biomarker monitoring

    The study will track changes in specific biomarkers, such as lyso-Gb1 concentration, hemoglobin levels, platelet count, and organ volumes (spleen and liver) using MRI scans.

    GCase antibody levels will also be monitored to assess the body's immune response to the treatment.

  5. Step 5

    Genetic material clearance

    The study will determine the time it takes for the vector genomes, which are part of the treatment, to be cleared from the blood and semen. This involves testing until three consecutive negative results are obtained.

  6. Step 6

    Study duration

    The study is expected to continue until March 31, 2029, providing a comprehensive long-term follow-up for participants.

Who can join the trial?

6 criteria

  • Participants must have **Gaucher Disease Type 1**. This is a specific condition that affects certain cells in the body.
  • Participants must have previously received **FLT201**. This includes those who may have needed to restart or start **ERT**/**SRT**. **ERT** stands for Enzyme Replacement Therapy, and **SRT** stands for Substrate Reduction Therapy. These are treatments for Gaucher Disease.
  • Participants must be able to give full **informed consent**. This means they understand the study and agree to take part.
  • Participants must be able to follow all the requirements of the trial.
  • Both **male** and **female** participants are eligible.
  • The study includes participants from a **vulnerable population**. This means people who might need extra protection or care.
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Investigated drugs

FLT201 is an experimental therapy being studied for its potential to treat Gaucher disease type 1. It uses an adeno-associated viral vector to deliver genetic material into the body. The goal of this therapy is to provide a long-term solution by addressing the underlying cause of the disease, potentially improving symptoms and quality of life for patients. This study aims to evaluate the safety and effectiveness of this approach over an extended period.

What is already known about the treatment

FLT201 – FLT201 is administered as an adeno-associated viral vector, typically delivered through intravenous infusion. It is currently under investigation in clinical trials for its safety and effectiveness in treating Gaucher Disease Type 1. The main therapeutic indication for FLT201 is to address the underlying enzyme deficiency in Gaucher Disease Type 1. At the molecular level, FLT201 works by delivering a functional copy of the gene responsible for producing the enzyme glucocerebrosidase, which is deficient in individuals with this condition. This medication falls under the pharmacological classification of gene therapy.

Investigated diseases

Gaucher Disease Type 1 – Gaucher Disease Type 1 is a genetic disorder caused by a deficiency in the enzyme glucocerebrosidase. This enzyme deficiency leads to the accumulation of fatty substances in certain organs, particularly the spleen and liver, causing them to enlarge. As the disease progresses, individuals may experience symptoms such as fatigue, anemia, and a tendency to bruise easily due to low platelet counts. Bone pain and fractures can also occur as the disease affects bone tissue. Unlike other types of Gaucher disease, Type 1 does not typically involve the nervous system. The severity and progression of symptoms can vary widely among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-511172-33-00Protocol codeFLT201-02Estimated enrolment5 patientsSponsorSpur Therapeutics Limited

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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