A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations

Launched by SENSORION · Apr 12, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called SENS-501 for young children who have severe to profound hearing loss caused by a specific genetic change in the Otoferlin gene. The trial aims to determine whether SENS-501 is safe and effective for children between 6 to 31 months old who have this type of hearing loss. The researchers are looking for children who meet certain criteria, such as having a confirmed Otoferlin gene mutation and showing some signs of hearing capability, like otoacoustic emissions (sounds produced by the inner ear).

If your child is eligible and decides to participate, they will undergo assessments to ensure their safety and monitor their response to the treatment. The study is currently recruiting participants, and it’s important to note that children with certain health conditions or previous treatments that may interfere with the study cannot take part. Overall, the trial is an important step in finding new ways to help children with this type of hearing loss improve their hearing and overall quality of life.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Children (male or female) ≥ 6 to ≤ 31 months at the time of inclusion
• • Severe to profound hearing loss assessed by auditory brainstem response (ABR)
• • Biallelic mutation in the Otoferlin gene
• • Presence of Otoacoustic emissions (OAEs)
• • Documented normal cochlea and internal auditory canals
• • Patients with intact vestibular function
• Exclusion Criteria:
• • History of chronic, acute, or major disease, or unspecified reasons, that in the opinion of the Investigator, makes the participant unsuitable for participation in the study or constitutes an unacceptable risk.
• • Have been dosed in a previous gene therapy clinical trial
• • Patients with a prior or current cochlear implant
• • Any contraindication to the surgery determined by the surgeon or anesthesia determined by the anesthesiologist, or designee, or history of therapy known as ototoxic (e.g., cisplatin, high dose and long treatment with aminoglycosides, etc.) for an extended period (more than 2 weeks).
• • Participation in any other interventional clinical trial
• • Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or might interfere with the evaluation of study treatment
• • Anticipated noncompliance with the protocol requirements