AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutation
This study, called AUDIOWOLF, is investigating a medication called "DEPAKINE CHRONO" (sodium valproate) and its potential benefits for people living with Wolfram syndrome. Specifically, the main goal is to see if taking this medicine daily can help preserve hearing. Researchers will measure hearing very carefully over three years, looking for any changes. They will focus on hearing at 8 kHz, which is a high frequency sound. The hope is that the medication will prevent hearing from declining significantly. Beyond hearing, the study will also look at other aspects of Wolfram syndrome, such as how the medication might affect diabetes, vision, balance, and sleep. Safety is also a key part of what will be monitored throughout the study.
At a glance
What is this study about?
Wolfram syndrome is a rare genetic condition that can affect many parts of the body, including hearing. This study, called AUDIOWOLF, aims to find out if a daily medication, DEPAKINE CHRONO (which is a form of sodium valproate), can help protect the hearing of people with Wolfram syndrome.
The main focus of the study is to see if this medication can stop hearing from getting worse over a three-year period. Researchers will use special hearing tests, similar to ones you might have at an audiologist, to measure changes very precisely. They are particularly interested in how well people can hear high-pitched sounds. If the medication can limit how much hearing is lost, it could be a really important step forward for people with Wolfram syndrome.
Besides hearing, the research team will also be looking at other effects of the medication. This includes checking if it helps with other common issues in Wolfram syndrome, such as diabetes (both types), vision, how well people can balance, and their sleep patterns. Throughout the study, the team will also be very careful to monitor for any side effects from the medication to make sure it is safe.
Key takeaways
- The study tests if DEPAKINE CHRONO (sodium valproate) can protect hearing in Wolfram syndrome.
- It focuses on preserving hearing, especially high-frequency sounds, over three years.
- Beyond hearing, researchers will also check impacts on diabetes, vision, balance, and sleep.
- Participants will take daily medication and attend regular clinic visits for tests.
- Safety of the medication will be closely observed throughout the study.
- It's an adult-only study for individuals with specific hearing loss related to Wolfram syndrome.
Who may be eligible?
This study is looking for adults who have Wolfram syndrome caused by a specific genetic change. Participants must have already experienced some hearing loss in their high-frequency hearing at 8 kHz. You'll need to be aged 18 or older to take part.
There might be other health conditions or medications that would prevent you from joining, as the researchers need to ensure the study is safe and that results are clear. The research team will review all your health information to see if you are a good fit for the study.
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.
- Are you 18 years old or older?
- Have you been diagnosed with Wolfram syndrome due to a genetic change?
- Do you have some hearing loss in the higher frequencies (specifically at 8 kHz)?
- Are you able to commit to daily medication and regular hospital visits for three years?
- Are you willing to have blood tests, hearing tests, and other health assessments?
What does participation involve?
If you decide to take part, you would be taking the study medication, DEPAKINE CHRONO, every day for three years. You would have regular visits to the study clinic to check your hearing, vision, balance, and overall health. These visits would include specific hearing tests, eye checks, and possibly MRI scans to look at your brain. You would also have blood tests to make sure the medication level in your body is right and to check for any side effects. Some visits might involve filling out questionnaires about your sleep. The study will carefully monitor your health and how the medication affects you over the full three-year period.
Potential risks and benefits
Locations (2)
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Common questions
What is Wolfram syndrome?
Wolfram syndrome is a rare genetic condition that can affect many parts of the body, including hearing, vision, and how the body handles sugar (diabetes).
What is DEPAKINE CHRONO?
DEPAKINE CHRONO is a medication that contains sodium valproate. It's used in this study to see if it can help people with Wolfram syndrome.
How long will the study last if I join?
If you join this study, you would be taking the medication and having regular check-ups for a total of three years.
Will I know if the medication is helping me during the study?
The study team will be monitoring your health and test results, but typically for studies like this, individual results are not shared until the study is complete and analysed.
Can I stop taking part in the study at any time?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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