BridgeMD
A Phase I/II clinical trial with SENS-501 in children suffering from severe to profound hearing loss due to Otoferlin (OTOF) mutations
EU CTIS ID: 2023-504466-28-00
What this study is testing
Part 1: To assess safety, tolerability of SENS-501 following unilateral injection into the cochlea. Part 2: To assess the efficacy following unilateral injection SENS-501 into the cochlea.
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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
You likely can't join if
- 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.
See the full eligibility criteria
- 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
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.