A Phase 2 open-label trial to investigate the long term safety of VO659 administered intrathecally in participants with Huntington’s disease and spinocerebellar ataxia type 1 and 3
This Phase 2 clinical trial is testing a new experimental medicine named VO659. It is designed for adults living with Huntington’s disease or spinocerebellar ataxia (types 1 or 3). Both of these are inherited conditions that affect the nervous system, leading to changes in movement, balance, and thinking. The main goal of the trial is to see how safe VO659 is when taken over a longer period. The medicine is given through an injection into the lower back, directly into the fluid surrounding the spinal cord. This is known as an intrathecal injection. Doctors will carefully check for side effects and monitor each participant’s general health, brain function, and well-being. They will also look at whether the medicine helps to slow down the symptoms of these conditions.
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¿De qué trata este estudio?
Huntington’s disease and spinocerebellar ataxia (types 1 and 3) are rare genetic conditions that damage nerve cells in the brain over time. Over months and years, this damage can cause problems with walking, balance, speech, and thinking skills. Currently, there are treatments to help manage some of the symptoms, but there are no cures that stop the underlying damage.
This trial is testing an experimental treatment called VO659. The medicine is designed to target the faulty processes inside nerve cells that cause these diseases. Because the brain is protected by a natural barrier, ordinary pills often cannot reach it easily. For this reason, VO659 is given directly into the fluid around the spinal cord so it can travel straight to the brain.
The study is 'open-label', which means both you and the research team will know that you are receiving the real study drug, rather than a dummy treatment (placebo). By studying participants over time, doctors hope to understand whether VO659 is safe, what doses work best, and whether it could become a helpful future treatment.
Puntos clave
- Tests the safety of an experimental treatment called VO659 over a longer period.
- Open to adults aged 18 and over with Huntington’s disease or SCA types 1 and 3.
- The medicine is given by an injection into the fluid around the spine (lumbar puncture).
- There is no placebo; every participant in this study receives the active medicine.
- Participation is voluntary, and you are closely monitored throughout by medical specialists.
¿Quién puede ser elegible?
This trial is open to men and women aged 18 and older who have a confirmed diagnosis of either Huntington’s disease or spinocerebellar ataxia type 1 or 3. Because it is a specialised study, participants need to be well enough to travel to the study clinic for regular appointments and procedures.
You may not be able to join if you have other serious medical issues that could make taking part unsafe, such as severe heart, kidney, or liver disease. People who cannot safely have an MRI scan or a spinal injection might also be unable to take part.
A doctor at the trial clinic will review your full medical history, run safety tests, and talk with you in detail to see if the trial is a good and safe match for you.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- I am aged 18 or older.
- I have been diagnosed with Huntington’s disease, SCA1, or SCA3.
- I am able to attend regular hospital appointments for tests and scans.
- I am willing and able to receive injections into my lower back (lumbar punctures).
- I can safely undergo an MRI scan (for example, I do not have incompatible metal implants).
¿En qué consiste la participación?
If you take part, you will visit the hospital clinic regularly for check-ups and treatments. The study medicine is given using a lumbar puncture, where a thin needle is placed into your lower back to deliver the medicine into your spinal fluid. During clinic visits, you will have regular physical and neurological checks, blood and urine tests, and heart tracings (ECGs). You will also have MRI brain scans to check your brain health, as well as memory and movement assessments. Doctors will ask you to fill out questionnaires about your mood, quality of life, and daily activities. The study team will follow your progress closely over many months to monitor your health and safety.
Riesgos y beneficios potenciales
Ubicaciones (4)
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- —No verificadoAlemania
Preguntas comunes
Will I definitely receive the real medicine?
Yes. This is an open-label trial, which means there is no dummy treatment (placebo) and all participants receive VO659.
How is the medicine given?
It is given as an injection into the lower back, into the fluid that cushions your spinal cord and brain. This is called an intrathecal injection or lumbar puncture.
Do I have to pay to take part?
No. The trial medicine and all study-related tests are provided free of charge, and help with travel expenses is often available.
¿Puedo abandonar el estudio si cambio de opinión?
Yes. You can leave the trial at any point, for any reason. Your regular doctors will continue to look after your health as normal.
¿Cuánto tiempo durará el estudio?
This is a long-term safety study, meaning it will run over many months. The study team will give you a clear schedule before you decide to join.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.