Active, not recruitingPhase 1/2
A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
Key facts
- Who runs it
- Sensorion
- Where
- Australia, France, Germany, and Italy4 sites in 4 countriesChoose a country to see its sites
- Trial code
- Status
- Active, not recruiting
- Phase
- Phase 1/2
- People taking part
- 12 people, planned
- Started
- 21 Jun 2024
- Expected to end
- Jul 2031
- Type of study
- Interventional, 3 groups
- Treatments tested
- SENS-501 administration
- Record updated
- 8 July 2026
Who can take part
- Age
- 6 months to 31 months
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 13 criteriaShow less
Who can take part
- 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
Who cannot take part
- 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)
- 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
- 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
- Participation in any other interventional clinical trial
- Anticipated noncompliance with the protocol requirements
This trial has no sites in United States.
It has sites in 4 countries. Choose a country to see its sites.
About the trial
What the trial wants to find out
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.
The 3 groups in the trial
- Dose escalation - Low dose groupTreatment being tested
Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system
Treatment: SENS-501 administration
- Dose escalation - High dose groupTreatment being tested
Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system
Treatment: SENS-501 administration
- Dose expansion groupTreatment being tested
Intracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase
Treatment: SENS-501 administration
Data and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- A Phase I/II, Open-ended, Adaptative, Open Label Dose Escalation and Expansion Clinical Trial to Evaluate the Efficacy and Safety of Unilateral Intracochlear Injection of SENS-501 Using an Injection System in Children With Severe to Profound Hearing Loss Due to Otoferlin Gene Mutations
- Record updated
- 8 July 2026