This is an update on the progress of the REGAIN trial (from the original blog post from July 2017)
Currently the only option for people with inner ear hearing loss is to wear a hearing aid, or, for those with severe to profound hearing loss, a cochlear implant. These devices help people to communicate, but do not treat the underlying cause of their hearing loss. With one in six people in the UK having hearing loss, there is an urgent need for new treatments. The researchers of the REGAIN project (REgeneration of inner ear hair cells with GAmma-secretase Inhibitors) have worked for years to develop a new drug that could regenerate inner ear sensory hair cells, and they are now at the stage of testing if the drug is safe to use in people and if it affects their ability to hear.
Testing new treatments
New drugs that could potentially treat hearing loss must first be tested in clinical trials. To test a drug in people for the first time, a ‘first-in-man’, or phase I, study is conducted. The aim is to test how safe the drug is in a small group of people. Once this has been proven, the next step is to test the drug in a larger group of people, to see if the drug works and to continue monitoring its safety. This is a phase II study.
The development of new treatments and testing in clinical trials is carefully watched over by the health regulatory authorities and requires their approval to proceed. This is in line with UK and EU law and best practice guidance that governs how clinical trials are designed and conducted.
The REGAIN study
In 2015, the European Union awarded a Horizon2020 research grant to a group of leading scientists and clinicians from across Europe, to develop and test a new drug with the potential to regenerate inner ear (cochlear) sensory hair cells and improve hearing. The project is called REGAIN, and the drug is a Gamma Secretase Inhibitor (GSI). It has been shown in animal studies that this class of drugs is able to turn on a chemical ‘switch’ to produce new sensory hair cells from other cells in the inner ear, called ‘supporting cells’, and improve hearing of these animals. The REGAIN researchers have selected a drug from this class and after completing the preclinical safety testing according to UK and EU regulations, are now at the stage of testing this drug for the first time in people with hearing loss. This is called a phase I trial.
First-in-Man Clinical Trial (Phase I)
The first-in-man REGAIN clinical trial is designed to assess how safe the drug is in up to 24 people with hearing loss. The study team at the UCL Ear Institute and Royal National Throat Nose and Ear Hospital (UCLH NHS Foundation Trust) in the UK, led by ENT surgeons Professor Anne Schilder and Professor Shakeel Saeed, are looking for people with hearing loss who are interested in taking part in this trial.
Who can participate?
The team are looking for people aged 18-80 years with mild to moderate sensorineural hearing loss, who are either using or have been previously offered a hearing aid. People are eligible to take part in the study if they have had hearing loss for less than 20 years. Participants must be willing to refrain from wearing their hearing aid in the ear to be treated for three months, the duration of treatment.
People who suffer from tinnitus, and consider this to be more of a problem than their hearing loss, are not eligible for the study.
All potential participants will be provided with detailed information about the study, including the potential risks and benefits, so they can make an informed choice about whether to take part.
Can people with congenital hearing loss take part?
It is not possible for people who are born with hearing loss (congenital) to take part in the REGAIN study. This is because the study drug is not designed to treat this type of hearing loss.
Can people outside the UK take part?
At this Phase I stage, only people living in the UK can take part in the REGAIN study. However, we are approaching the end of Phase I and will be moving on to Phase II. We anticipate that Phase II will commence by the end of 2018 and expand to Greece and Germany.
Why can only people with mild to moderate sensorineural hearing loss take part? What about people with severe hearing loss?
To be eligible for the REGAIN study, people must have a mild to moderate hearing loss. This is for two reasons. We are in the early stages of testing this new drug for people with hearing loss; the study is testing the safety of this drug that aims to regenerate inner ear sensory hair cells; it is thought that the capacity of the inner ear to do so potentially decreases with the level of hearing loss. In addition, detecting any potential negative effects of the drug on hearing, can be done more reliably in people who have mild to moderate hearing loss than in those with severe or profound hearing loss.
How is the drug administered?
The drug will be injected through the ear drum into the middle ear, using a local anaesthetic to numb the ear drum. This technique is regularly practiced to deliver other drugs inside the ear, such as corticosteroids for the treatment of Ménière's disease.
When and where is it taking place?
The Phase I stage opened to recruitment of people with hearing loss in November 2017 and is taking place at the Royal National Throat Nose and Ear Hospital (UCLH NHS Foundation Trust). This is the largest ear, nose and throat hospital in the UK, and hosts the European Centre for Audiological Medicine and Research. Each participant will be asked to attend the hospital for nine visits over 14 weeks; these visits include:
- a screening visit with a range of hearing and balance tests to assess if they are eligible to take part
- three visits, one week apart, for administration of the drug and a range of tests to assess safety of the drug (including hearing and balance tests)
- follow up visits to continue assessing the safety of the drug.
Participating in the REGAIN study will require considerable time and commitment over 14 weeks. The study is designed to rigorously test the safety of the drug, which means receiving the drug three times, and attending regular appointments for tests at set times.
By the end of 2018, the results from Phase I will inform the next trial stage, Phase II. This will focus on how the drug affects hearing and will continue to monitor safety in a larger group of people. Phase II will take place in the UK, Greece and Germany.
If you are or know a family or friend who would be interested in taking part in the study and would like to receive further information, please contact the REGAIN research team:
By telephone: 020 3108 9344
By email: firstname.lastname@example.org
Or visit our website.