A Phase I/II, open-label, dose escalation study to evaluate the safety of 2 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (limb-girdle muscular dystrophy LGMDR5, formerly LGMD2C).
This research study is investigating a new gene therapy called ATA-200 for people with limb-girdle muscular dystrophy type R5 (LGMD-R5). This condition causes muscles to weaken over time. The main goal of this study is to find out if ATA-200 is safe and well-tolerated. It's also designed to determine the best dose of the treatment. Beyond safety, researchers will look at whether the therapy can improve muscle strength and daily activities. This is an early-stage study, meaning it's one of the first times this treatment is being given to people. Participants will be monitored closely for any side effects and changes in their muscle health.
Em resumo
Sobre o que é este estudo?
This study is about an investigational new treatment for a specific type of muscular dystrophy called limb-girdle muscular dystrophy type R5 (LGMD-R5). This condition, previously known as LGMD2C, leads to muscle weakness, particularly in the shoulders, hips, and thighs. Currently, there isn't a cure for LGMD-R5, so researchers are looking for new ways to help.
The new treatment being tested, called ATA-200, is a type of gene therapy. This means it aims to deliver a healthy version of a specific gene (called gamma-sarcoglycan) into the body. People with LGMD-R5 have a faulty version of this gene, which stops their muscles from working properly. By introducing a healthy gene, the hope is that muscle cells can start producing the correct protein, which could help strengthen muscles and improve symptoms.
This is an early-stage study, combining what are called Phase I and Phase II trials. Phase I studies primarily focus on safety and finding the right dose of a new medication. Phase II studies then look at whether the treatment shows any early signs of helping the condition. Participants will receive the treatment through a drip into their vein, and the study will carefully monitor their health, looking for any side effects and changes in their muscle function.
Pontos principais
- This study is testing a new gene therapy (ATA-200) for LGMD-R5.
- The main goals are to check treatment safety, find the right dose, and see if it helps muscle function.
- It's an early-stage study, so the treatment is experimental.
- Participants will receive the treatment through a drip and other medications.
- Regular clinic visits, tests, and monitoring are required.
- Você pode parar de participar a qualquer momento.
Quem pode ser elegível?
To be able to take part in this study, you would need to have been diagnosed with limb-girdle muscular dystrophy type R5, which used to be called LGMD2C. The study is open to both males and females of all ages.
Before joining, doctors will check your overall health to make sure the study treatment is suitable and safe for you. They will also look at your current medications and medical history to ensure there are no reasons why you shouldn't take part.
More detailed checks would be done by the study team, including blood tests and other examinations, to confirm if this study is right for you. It's important to discuss your full medical history with the study doctors.
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.
- Do I have a confirmed diagnosis of limb-girdle muscular dystrophy type R5 (LGMD-R5)?
- Am I willing and able to attend all required clinic visits and tests?
- Am I comfortable with receiving an experimental treatment?
- Have I discussed this study with my current doctor?
- Am I able to receive treatments given through an IV drip?
- Am I able to take other medications, such as steroids?
O que envolve a participação?
If you decide to take part in this study, you would receive the investigational treatment, ATA-200, through an intravenous (IV) drip, meaning it goes directly into your bloodstream. You would also receive other medications like Prednisolone or Methylprednisolone, which are steroids, and possibly Rapamune, to help your body accept the new gene therapy and reduce potential side effects. These would be given either by mouth or intravenously as directed by the study team.
Throughout the study, you would have regular visits to the clinic for various checks. These include physical examinations, vital signs (like blood pressure and heart rate), heart checks, and extensive blood tests. You’ll also do specific tests to measure your muscle strength and movement, such as walking tests, stair climbing, and tests focusing on your arm and hand function. Imaging scans, like MRI, will be used to look at your muscles, and lung function tests will check your breathing.
Some participants may also have a muscle biopsy, which involves taking a small sample of muscle tissue to see how the treatment is working at a cellular level. You will also be asked to fill out questionnaires about your daily activities and how you feel, to understand the treatment's impact on your quality of life. The study involves many visits and detailed assessments, and its total duration would be explained by the study team. There will be multiple follow-up appointments after the initial treatment period.
Riscos e benefícios potenciais
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Perguntas frequentes
O que é terapia genética?
Gene therapy is a new type of treatment that tries to fix diseases by changing genes inside your body's cells. In this study, it's designed to give muscle cells a working version of a gene that's faulty in LGMD-R5.
What is LGMD-R5?
LGMD-R5, also known as limb-girdle muscular dystrophy type R5, is a rare genetic condition that causes muscles to weaken, especially around the hips and shoulders. It used to be called LGMD2C.
Will I have to stay in the hospital?
While the treatment is given through a drip, it's likely you would need to spend time at a clinic or hospital for the treatment and close monitoring, but not necessarily for a long stay. The full details of clinic visits would be explained by the study team.
What are 'Phase I' and 'Phase II' studies?
Phase I studies check if a new treatment is safe and help find the right dose. Phase II studies then look for early signs that the treatment might be effective and continue to monitor safety. This study combines both stages.
What information will the study collect about me?
The study will collect information about your general health, muscle strength, how you move, your breathing, and how you feel in your daily life. They will also take blood samples and possibly muscle samples.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
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