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

NCTID	NCT06370351 (View at clinicaltrials.gov)
Description	This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene. (Show More)
Indication	OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders, Ear Diseases, Otorhinolaryngologic Diseases, Deafness, Hearing Loss, Sensorineural
Compound Name	SENS-501
Sponsor	Sensorion
Funder Type	Industry
Status	Recruiting
Enrollment Count	12

Target Gene/Variant	OTOF
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracochlear
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV
Editor Type	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed high dose
Dose 3	Undisclosed expansion dose
Dose 4
Dose 5

No.of Trial Sites	2
Locations	Australia,France

Has US IND	False
Recent Updates	First patient dosed Q3 2024