In short
SENS-601 is an investigational gene therapy being studied for hearing loss caused by changes in the GJB2 gene. One clinical trial is currently authorised in the EU, a Phase 1/2 study planning to enrol 18 paediatric participants. The trial will test the safety of injecting SENS-601 directly into the cochlea using a device called the SPHYNX administration system, and will also look at whether it improves hearing.
Key points
- SENS-601 is an investigational gene therapy for hearing loss caused by GJB2 gene changes.
- The only registered trial is a Phase 1/2 study with a planned enrolment of 18 participants.
- The trial is currently authorised in the EU Clinical Trials Information System.
- The study population is paediatric patients with hearing loss related to biallelic pathogenic GJB2 variations.
- SENS-601 is given by intracochlear injection using the SPHYNX administration system.
- Part 1 measures safety and tolerability; Part 2 measures hearing outcomes including auditory brainstem response and pure-tone audiometry.
What is SENS-601?
SENS-601 is an investigational gene therapy. It is not yet an authorised medicine. The substance is being developed for the treatment of hearing loss caused by changes in the GJB2 gene. In the EU Clinical Trials Information System (CTIS), SENS-601 is recorded as a drug product for intracochlear injection, meaning it is injected directly into the inner ear structure called the cochlea. The trial record lists the substance under the name SENS-601 with no additional trade names or development codes.
How does SENS-601 work?
The trial registry record does not describe the mechanism of action of SENS-601 in detail. The study targets hearing loss related to biallelic pathogenic variations of the GJB2 gene. The GJB2 gene provides instructions for making a protein called connexin 26, which is important for the normal function of cells in the inner ear. Pathogenic variations in both copies of this gene are a known cause of inherited hearing loss. SENS-601 is administered directly into the cochlea, the spiral-shaped organ of the inner ear responsible for hearing, suggesting the aim is to act locally within the inner ear.
Which conditions is SENS-601 studied for?
- Hearing loss related to biallelic pathogenic variations of the GJB2 gene — the trial is limited to this specific form of inherited hearing loss in the paediatric population.
How is SENS-601 given?
SENS-601 is given by intracochlear injection, meaning it is injected directly into the cochlea of the inner ear. The injection is performed using a device called the SPHYNX administration system. In Part 1 of the trial, participants receive a unilateral injection, meaning the injection is given into one ear only. In Part 2, participants receive a bilateral injection, meaning both ears are injected.
What do the clinical trials show?
One clinical trial of SENS-601 is registered in the EU Clinical Trials Information System. The trial is a Phase 1/2 study, which means it combines an early phase focused on safety with an early look at whether the treatment has an effect. The study is currently authorised, meaning it has received regulatory approval to proceed. The planned enrolment is 18 participants, and the study population is paediatric patients with hearing loss related to biallelic pathogenic variations of the GJB2 gene.
The trial is divided into two parts. Part 1 assesses the safety and tolerability of SENS-601 and the SPHYNX administration system following unilateral injection of SENS-601 into the cochlea. The primary outcomes for Part 1 are the incidence and severity of treatment-emergent adverse events, adverse device events, serious adverse device events, and device deficiencies. Part 2 assesses the efficacy of SENS-601 following bilateral intracochlear injection. The primary outcome for Part 2 is the change in auditory brainstem response and pure-tone audiometry from baseline. Auditory brainstem response is a test that measures how well sound signals travel from the ear to the brainstem, and pure-tone audiometry is a standard hearing test that measures the quietest sounds a person can hear at different pitches.