A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1
Type 1 Gaucher disease is a genetic condition where the body cannot make enough of an essential enzyme. Without it, fatty substances build up in organs like the liver, spleen, and bone marrow, causing tiredness, easy bruising, and enlarged organs. Most people currently need lifelong, regular treatment through a drip. This phase 3 trial is investigating a new gene therapy called FLT201. The treatment aims to deliver working instructions to your cells so your body can make the missing enzyme itself. Participants receive FLT201 alongside temporary medicines that calm the immune system to help the therapy work safely. Doctors will monitor participants closely over 52 weeks using blood tests, body scans, and health questionnaires to check whether blood counts, liver and spleen size, and bone health stay stable. The goal is to see if FLT201 is safe and effective as a long-term treatment.
At a glance
What is this study about?
Gaucher disease type 1 is a rare inherited condition. People with this condition have low levels of an enzyme responsible for clearing certain fatty chemicals from the body. When these fats build up, they collect in the spleen, liver, and bones. This often causes low red blood cell counts (anaemia), low platelets that lead to easy bruising or bleeding, enlarged organs, bone pain, and deep fatigue.
Current standard care usually involves enzyme replacement therapy, which requires lifelong infusions into a vein every two weeks. This trial is studying a gene therapy called FLT201. Instead of replacing the enzyme every few weeks, gene therapy provides your liver cells with the correct genetic code so that they can continuously produce the enzyme on their own.
This study is a phase 3 trial, which is the final major stage of clinical research before a medicine can be approved for wider use. Researchers want to confirm whether a single dose of FLT201 safely keeps haemoglobin and platelet levels stable, prevents organs from swelling, and protects bone health over at least 52 weeks. If successful, it could offer people with type 1 Gaucher disease more independence from frequent hospital infusions.
Key takeaways
- Tests a one-off gene therapy (FLT201) designed to help your body produce its own missing enzyme.
- Aims to keep red blood cells, platelets, and organ sizes stable without frequent drips.
- Open to adults aged 18 and over with type 1 Gaucher disease.
- Involves temporary medicines to calm the immune system around the time of the infusion.
- Follows participants closely with regular clinic visits and tests for at least 52 weeks.
Who may be eligible?
This trial is open to adults aged 18 and older who have a confirmed diagnosis of type 1 Gaucher disease. Both men and women are welcome to take part.
Because gene therapy is a specialised treatment, the study team will carry out detailed medical checks to ensure it is safe for you. You may not be able to join if you have certain other long-term medical conditions, severe liver disease, or if your body produces antibodies that could block the gene therapy from working.
Participants must also be able to take short-term medicines that temporarily lower the immune system. Women who are pregnant or breastfeeding, and people planning a pregnancy in the near future, cannot take part for safety reasons.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have been diagnosed with type 1 Gaucher disease.
- I am willing and able to attend regular hospital check-ups and scans for at least a year.
- I can safely take temporary medicines that calm the immune system.
- I am not currently pregnant, breastfeeding, or planning a pregnancy during the trial.
What does participation involve?
If you take part, you will start with screening visits to check your general health, blood counts, liver and spleen size, and bone density. If the trial is right for you, you will receive the FLT201 gene therapy as a single infusion through a drip into a vein.
Around the time of your infusion, you will also take oral or injectable medicines (such as prednisolone or tacrolimus) to calm your immune system and stop it from fighting off the new gene therapy. You will attend regular clinic visits over 52 weeks (one year). During these visits, doctors will perform blood tests, physical checks, heart tests (ECGs), and imaging scans. You will also complete short questionnaires about your energy levels and quality of life. The study team will track your health carefully throughout the trial.
Potential risks and benefits
Locations (1)
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Common questions
What is gene therapy?
Gene therapy is a treatment that gives your cells a working copy of a gene, allowing your body to produce a protein or enzyme that it was previously unable to make properly.
How is FLT201 given?
FLT201 is given as a single infusion into a vein through a drip, usually in a hospital clinic setting under close observation.
Why do I need immune-calming medicines?
Your body's immune system might naturally try to fight off the harmless virus shell used to deliver the new gene. Short-term medicines help protect the gene therapy so it can reach your liver cells safely.
Will I have to stop my current Gaucher treatments?
The trial team will explain whether and when you can pause your current enzyme replacement therapy. Your health and blood counts will be checked continuously to make sure you stay safe.
Do I have to pay to take part in this trial?
No, clinical trials in the UK do not charge participants for trial medicines or tests, and travel costs to hospital visits are usually reimbursed.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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