A Phase I/II clinical trial with SENS-501 in children suffering from severe to profound hearing loss due to Otoferlin (OTOF) mutations
- Conditions
- Bilateral severe to profound hearing loss due to Otoferlin deficiency.MedDRA version: 20.0Level: LLTClassification code: 10057844Term: Hearing loss bilateral Class: 10013993Therapeutic area: Diseases [C] - Otorhinolaryngologic Diseases [C09]
- Registration Number
- CTIS2023-504466-28-00
- Lead Sponsor
- Sensorion
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 12
Children (male or female) = 6 to = 31 months at the time of inclusion., Severe to profound hearing loss assessed by ABR, Biallelic mutation in the Otoferlin gene, Presence of OAEs, Documented normal cochlea and internal auditory canals, Patients with intact vestibular function
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 (in accordance with the definition in Art. 2.2(1) of REGULATION (EU) No 536/2014)., 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method