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 (cochlear) hair cells, and they are now at the stage of testing if the drug is safe to use and if it affects the ability to hear in people.
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 (REgeneration of inner ear hair cells with GAmma-secretase Inhibitors)
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) hair cells and improve hearing. The project is called REGAIN, and the drug is a Gamma Secretase Inhibitor (GSI). The REGAIN group has shown in animal studies that this class of drugs is able to turn on a chemical ‘switch’ to produce new hair cells from other cells in the inner ear, called ‘supporting cells’, and improve hearing of these animals. After completing the preclinical safety testing according to UK and EU regulations, the group are now at the stage of testing if the drug is safe to use in people and if it affects their ability to hear.
First-in-Man Clinical Trial (Phase I)
The first-in-man clinical trial will test a drug of this class in people for the first time. It 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), 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 10 years. Participants must be willing to refrain from wearing their hearing aid in the ear to be treated for three weeks, 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.
Where and when is it taking place?
The study will start in August 2017 and take 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.
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.
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.
The results of this Phase I trial will inform the next trial stage – the Phase II trial - which will focus on how the drug affects hearing. This study will take place in the UK, and at partner sites in Greece and Germany, from the end of 2017.
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.
If you are 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.
To find out more information about clinical trials, please visit the UK Clinical Trials Gateway.