A Gaucher Disease Gene Therapy Trial With FLT201
Gaucher disease type 1 is a rare genetic condition where the body cannot make enough of a vital enzyme, leading to tiredness, bone problems, and swelling in the liver or spleen. Most people manage this by taking lifelong medicines or regular hospital drips. This clinical trial is testing a new gene therapy called FLT201. Gene therapy aims to give your liver cells the genetic instructions to make this missing enzyme on their own. Researchers want to see if a single dose of FLT201 is safe and effective enough to allow adults to stop their usual ongoing treatments. The study is open to adults who have been on stable treatment for at least two years. It will closely track their health and blood counts over time to see how well the new treatment works.
Em resumo
Sobre o que é este estudo?
Type 1 Gaucher disease is a lifelong condition caused by a faulty gene. Normally, an enzyme called glucocerebrosidase clears out certain fatty substances in your cells. Without enough of it, these fats build up in places like the liver, spleen, and bone marrow. Current treatments, like regular enzyme infusions or daily tablets, do a great job of keeping symptoms under control. However, they must be taken for life and can take up a lot of time.
This clinical trial is looking at whether a one-off gene therapy called FLT201 can offer a longer-lasting solution. FLT201 works by delivering a working copy of the gene directly to your liver cells using a harmless, modified delivery vehicle. If successful, your liver can begin to produce the needed enzyme continuously, which could remove the need for regular drips or tablets.
By taking part, participants help doctors find out if this treatment is safe and truly effective. Finding a treatment that works long term from a single dose could greatly improve freedom and quality of life for people living with Gaucher disease.
Pontos principais
- Tests a one-off gene therapy (FLT201) for adults with type 1 Gaucher disease.
- Aims to replace regular, lifelong treatments like enzyme infusions or tablets.
- Open to adults aged 18+ who have been stable on treatment for at least two years.
- Participants must pause their standard therapy under close medical care.
- Requires long-term follow-up visits to check safety and blood counts.
Quem pode ser elegível?
This trial is for adults aged 18 and older who have a confirmed diagnosis of type 1 Gaucher disease. To take part, you must have been taking regular enzyme therapy or substrate reduction therapy without breaks for at least two years, and your blood counts must currently be steady.
You cannot join if you have type 2 or type 3 Gaucher disease, or if you have had your spleen completely removed. People who are pregnant, breastfeeding, or who have had a stem cell, bone marrow, or organ transplant in the past are also unable to join. Because of how the therapy is given, you cannot take part if your body already has natural antibodies against the harmless carrier used to deliver the gene.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- I am aged 18 or older.
- I have been diagnosed with type 1 Gaucher disease (not type 2 or 3).
- I have been taking enzyme therapy or substrate reduction therapy regularly for at least two years.
- I still have my spleen (have not had a total splenectomy).
- I have never had an organ or bone marrow transplant.
- I am not pregnant or breastfeeding.
O que envolve a participação?
If you join the study, you will first undergo a screening check, including blood tests and health scans, to make sure the trial is safe for you. You will then receive the FLT201 gene therapy as a one-off drip into a vein. Afterward, you will be guided by doctors to stop your regular Gaucher medication so researchers can see if the gene therapy is working on its own. You will need to attend regular clinic visits for blood tests, health checks, and scans. Because gene therapy effects are long-lasting, the trial will follow your health closely over several years to ensure your safety.
Riscos e benefícios potenciais
Locais (32)
- Kaiser Permanente - CACódigo postal verificadoLos Angeles, Estados Unidos· Recrutamento
- UCLA HealthCódigo postal verificadoLos Angeles, Estados Unidos· Recrutamento
- University of California, San Francisco (UCSF)Código postal verificadoSão Francisco, Estados Unidos· Recrutamento
- University of Colorado AnschutzCódigo postal verificadoAurora, Estados Unidos· Recrutamento
- Emory HealthcareCódigo postal verificadoAtlanta, Estados Unidos· Recrutamento
- University of Iowa Health CareCódigo postal verificadoIowa City, Estados Unidos· Ainda não recrutando
- Universidade de MinnesotaCódigo postal verificadoMinneapolis, Estados Unidos· Recrutamento
- Hackensack University Medical CenterCódigo postal verificadoHackensack, Estados Unidos· Recrutamento
- NYU Langone HealthCódigo postal verificadoNova Iorque, Estados Unidos· Recrutamento
- Duke University Medical CenterCódigo postal verificadoDurham, Estados Unidos· Recrutamento
- Cincinnati Children's Hospital Medical CenterCódigo postal verificadoCincinnati, Estados Unidos· Ainda não recrutando
- Hospital Infantil da FiladélfiaCódigo postal verificadoFiladélfia, Estados Unidos· Recrutamento
Perguntas frequentes
O que é terapia genética?
Gene therapy is a medical approach that introduces a healthy, working copy of a gene into your body to help treat a genetic condition.
How is FLT201 given?
FLT201 is given as a single infusion into a vein, usually at a specialised hospital clinic.
Will I have to stop my current Gaucher medication?
Yes, the study involves stopping your current regular treatment after receiving FLT201, under strict medical supervision.
Can I go back to my old treatment if the trial therapy does not work?
Yes, doctors will monitor your health closely and restart standard treatment if your body needs it.
Does it cost anything to take part?
No, the trial treatment is provided free of charge, and travel costs to hospital visits are usually covered.
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