The First in Human trial of Rincell-1

Hearing aids and cochlear implants are globally recommended treatment options for sensorineural hearing loss, but they cannot restore normal hearing and do not treat the neural component of sensorineural hearing loss. Hearing aids work by making sounds lounder and they rely on good hair cell and auditory neuron survival in the cochlea to work best. Cochlear implants (CI) can bypass damaged hair cells, but they rely on healthy auditory neurons to work well. There are currently no treatments for damage to auditory neurons. As a result, people with neural hearing loss may not receive full benefit from the existing hearing devices they are offered.

Find out more information on hearing loss caused by damaged auditory neurons here.

Rincell-1 is a cell therapy made of specialised auditory neuron cells grown in the laboratory. These cells have the potential to grow into new auditory neurons. Laboratory and animal studies have shown that Rincell-1 is safe and able to improve hearing.

In our first in human clinical trial, Rincell-1 will be given to participants in combination with a cochlear implant. The main purpose of the trial is to check the safety of Rincell-1 in humans, and if it can improve neural health.

The first in human clinical trial of Rincell-1 will be delivered in partnership with NHS cochlear implant programs in the UK.

Our first in human trial is not yet recruiting participants. We are currently setting up our research sites in the UK.

To stay informed about the trial’s progress, including when recruitment opens, please check our news or the ClinicalTrials.gov website.

The Rincell-1 trial will be available to adults who have been approved to receive a cochlear implant through the NHS in the United Kingdom at one of the participating NHS hospital sites.  After having been approved to receive an implant, people will be considered for the Rincell-1 trial if they also fit into one of the below categories:

-Have age related hearing loss and are between 60 and 75 years old

Or

– Have auditory neuropathy due to auditory neuron damage or loss and are between 18 and 75 years old

People will also need to meet other eligibility criteria to take part in the trial and suitability to participate will be assessed by trained healthcare professionals at the participating NHS hospitals. More information on the eligibility criteria for the study can be found on the ClinicalTrials.gov website

Before deciding to take part, all participants will have the trial fully explained to them by a member of the trial team at a participating NHS hospital.

Participants in the trial will be randomly assigned to either receive a cochlear implant alone (standard of care) or a cochlear implant with an injection of Rincell-1 (at the same time as cochlear implant surgery).

Regardless of which group someone is assigned to, all participants will need to attend regular appointments at their hospital for health and hearing checks. Participants will also be given an electronic tablet so they can take daily measurements from their cochlear implant at home. Each participant will be followed for 13 months after their cochlear implant surgery.

People who may be eligible will be invited to take part through NHS cochlear implant centres involved in the trial.

If you’re interested in taking part, we recommend speaking to your audiology or cochlear implant team. You can also check for updates and eligibility details on the ClinicalTrials.gov website

When we say the trial is randomised, it means that participants are put into different treatment groups by chance. Some participants receive the current standard treatment, and others receive a new treatment. Participants are randomly allocated to a group by a computer program. No one – not the researchers, and not the participants – chooses who goes into which group.

We do this to make sure the groups are as similar as possible at the start. That way, if we see any differences in the results, we can be more confident they’re due to the treatment being tested – not other factors. Information from the control group allows the researchers to see whether the new treatment(s) are more or less effective than the current standard treatment.

In this trial, we want to see whether having the Rincell-1 therapy alongside a cochlear implant is just as safe as having the cochlear implant on its own, and whether it works as well – or even better.

Randomising is a key part of making sure the results are fair, unbiased, and can be trusted.

Travel expenses for the participant and a companion will be covered.

People with hearing loss, their loved ones and members of the public with patient and public involvement experience have advised us on the design of this trial. They have shared their ideas on how to support people who join this trial and their families. They have also helped us write the Participant Information Sheet and other participant materials to make sure that they are easy to understand.

This trial has been reviewed and authorised by the Medicines and Healthcare products Regulatory Agency (MHRA). The MHRA also inspects the sites where a trial might be taking place to ensure compliance with good clinical practice. Visit the GOV.UK website to read about the MHRA

The trial has also been reviewed by the Health Research Authority (HRA) and an independent research ethics committee (REC). The HRA works to protect and promote patient and public interests in health research. It oversees research ethics committees across the UK. The REC approves any medical research involving people, whether conducted by the NHS or the private sector and ensures the rights and interests of the trial participants are protected. Find out more on the HRA website

Resources for patients, families and caregivers living with hearing loss

Research Study of Objective Measurements of Cochlear Health Using a Cochlear Implant

Using the electrode of a cochlear implant, we can record signals from the auditory neurons in response to electrical stimulation. This allows us to measure how well the neurons are working and monitor their health in the cochlea. These measurements will be very important in our future trials to help us demonstrate if the Rincell-1 cells are working.

Some of these measurements are used already as part of normal care in cochlear implant clinics. There are other tests that may be an even better measure of how well auditory neurons are working, but these are mainly only used in research. To decide whether these tests should be used in our trials for Rincell-1 we need to make sure they are reliable. In this study we have performed these measurements with people who already have a cochlear implant.

The results of this study will help us choose the best set of tests for our future trials and make recommendations for how the measurements could be improved. Find out more about the first in human clinical trial of Rincell-1

Recruitment for this study has finished. The preliminary findings show that these tests are reliable, sensitive to changes, and can be carried out by trained audiologists.

Frequently Asked Questions

Rincell-1

A cell therapy is a medical treatment that uses living cells to repair, replace, or regenerate damaged tissues or organs in the body. These cells can be taken from the patient, a donor, or grown in a lab, and are designed to help restore normal function or treat a disease.

Cell therapy involves introducing new or modified cells into the body to restore or improve function. It can be used to treat diseases where cells are damaged or lost, even if the exact cause isn’t known.

Gene therapy involves changing or adding genetic material within a person’s cells to treat or prevent a disease. It typically requires knowing which specific cells are affected and what genetic changes need to be made.

Rincell-1 is a cell-based therapy grown in the laboratory. Rincell-1 is made up of cells known as otic neural progenitors.  Otic neural progenitors are one developmental stage away from becoming fully mature auditory neurons. Once implanted into the inner ear, these progenitors mature into auditory neurons and have the potential to re-establish cochlear connections and hearing.

The cells that make up Rincell-1 were generated from stem cells donated by a single human donor and are stored in a stem cell bank. In the laboratory these cells are programmed to become auditory neurons and are then multiplied. After that they are kept in a medical freezer until they are ready to be given to a patient.

The Rincell-1 cell therapy has gone through rigorous screening and years of laboratory research, as well as early-stage testing to confirm safety before progressing to human trials.

Early testing showed that Rincell-1 didn’t cause any side effects.

The otic neural progenitors used in Rincell-1 only turned into auditory neurons. They didn’t change into any other kind of cell. They also stayed where they were placed and didn’t move to other parts of the brain or body. Even when they were placed in other areas on purpose, they didn’t cause any side effects.

In preclinical testing, the disease models that received Rincell-1 showed improved hearing thresholds. Those that didn’t receive the treatment didn’t show any improvement. This suggests that Rincell-1 may help improve hearing ability.

We are targeting hearing loss caused by the damage or loss of auditory neurons. There are many different factors that can lead to this type of damage, including genetic mutations, environmental and lifestyle factors, aging, and others.

For example, as we age, we naturally begin to lose auditory neurons, which can affect our ability to hear—particularly in challenging listening environments, such as understanding speech in background noise or enjoying music. People with age-related hearing loss often show significant auditory neuron loss.

Additionally, certain genetic mutations can directly affect the health or function of auditory neurons, also leading to a type of hearing loss called auditory neuropathy.

Hearing aids and cochlear implants can significantly improve hearing in individuals with damage to hair cells in the inner ear. However, their effectiveness depends on the presence of functional auditory neurons to transmit sound signals to the brain.

Neural hearing loss—specifically, damage or degeneration of auditory neurons—is a key underlying factor in many forms of sensorineural hearing loss. In particular, while age-related hearing loss (ARHL) involves multiple components, including hair cell degeneration and metabolic changes, all individuals with ARHL will experience some degree of auditory neuron loss.

This neuron loss severely limits the benefit of hearing aids and cochlear implants, especially in complex listening environments such as noisy settings, where understanding speech becomes extremely difficult. Our treatment is specifically designed to address this critical gap by targeting and restoring auditory neurons—something traditional devices cannot do. By focusing on the neural component, we aim to provide a foundational solution that enhances the effectiveness of existing hearing technologies or, in some cases, offers benefits where they cannot.

Clinical Research?

Clinical research refers to projects which aim to find out more about an illness, condition, treatment, therapy, or care by directly involving people to improve health and care.

The people taking part choose to follow a plan and to provide information about themselves. The information collected is used to make decisions to improve treatments, therapies and care for people more generally.

Participants in clinical research can be patients or healthy volunteers and while it frequently takes place in hospitals, universities or GP settings, it can also occur in community settings or in people’s homes.

Clinical research can be a clinical trial to test a treatment or care package, or a clinical study to learn more about a condition, therapy, experience, or setting. It may involve participants trying a new treatment, using a device, receiving a specific type of care, or providing information about their usual care.

A clinical trial is a type of clinical research study that compares the effects of different treatments and may involve patients, healthy individuals, or both. Trials help doctors determine if treatments work and what types of conditions they work best at treating. Participating in a trial may or may not directly benefit the person taking part, but it can be of benefit to others in the future.

All clinical trials for new medicines go through several phases to assess if they are safe and if they work. These medicines are usually compared to a control treatment, which can be either a placebo (dummy) or an existing standard treatment.

Participation in a clinical trial might give you access to a new treatment before it’s widely available, though the new treatment could be less effective than the standard treatments. Doctors and specialists follow participants closely in trials to monitor their ongoing safety and to ensure patients receive the care they need if the trial treatments do not perform as well as hoped.

Before you agree to participate in clinical trial, it is really important that you understand all aspects of the trial and have had an opportunity to discuss any questions you may have with your doctor and the research team.

Comprehensive details about the trial will be included in the Participant Information Sheet (PIS). A research team member will review this information with you and provide a copy for you to take home. This document covers everything you need to know about the trial.

You can refer to the PIS when deciding whether to participate in the trial. You are encouraged to discuss it with friends, family, your GP, specialist, or nurse. The research team will give you ample time to think it over and ask any questions before making your decision.

Before joining the trial, you must sign a document confirming that you understand what participation entails. This is known as informed consent.

You cannot participate in the trial without providing this written agreement. You will have the opportunity to ask questions if anything is unclear. You might also bring someone with you to ensure all your concerns are addressed. Participation is entirely voluntary, and you can decline if you choose.

Informed consent is an ongoing process throughout the trial, allowing you to continuously ask questions and receive answers while participating.

It is also very important that before agreeing to consent to a trial you understand that doctors are conducting the research as they genuinely do not know if a new treatment works. If the trial is randomised, meaning you could be randomly assigned to a treatment group, there is no evidence yet to suggest that being in one group will be better than the other.

Clinical trials are closely regulated in most countries to protect the safety, rights, and well-being of participants, and to ensure the results are reliable. While the specific rules can vary by region, they generally include review by independent ethics committees, oversight by government authorities, and compliance with international standards such as Good Clinical Practice (GCP).