Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene study: DB-OTO
Official title A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
ClinicalTrials.gov ID: NCT05788536
What this study is testing
What is DB-OTO?
DB-OTO is an investigational medicine, being studied as a potential treatment for congenital hearing loss secondary to biallelic mutations of the otoferlin gene (otof).
Also referred to as Otarmeni™, Otoferlin.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to check if the treatment is safe and well-tolerated. It also looks at how well the therapy works.
- Who it's for
- This study is for people of all ages with severe to profound hearing loss caused by changes in the otoferlin gene.
- What's involved
- Participants will receive an experimental gene therapy. The study will monitor their health and hearing to see how they respond.
- Time commitment
- The source text does not specify the total duration or the number of visits required.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join if
- Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide...
- Willingness to consent to genetic testing for the participant (by at least one parent/legal guardian for pediatric participants, and with participant...
- Willingness to consent to vaccinations for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to...
- Participant able to perform all necessary assessments to qualify for enrollment and dosing in the corresponding cohort at the time the participant or...
- Presence of biallelic, likely pathogenic or pathogenic OTOF variants
You likely can't join if
- History of prior treatment with gene therapy
- Surgical anatomy that would preclude or meaningfully impact the planned surgical approach as indicated by medical imaging (eg, Computed Tomography...
- History or presence of other permanent or untreatable hearing loss conditions
- Prior or current history of malignancies
- Prior or current history of meningitis
- History or presence of cochlear implants in the ear(s) to be infused with DB-OTO
See the full eligibility criteria
- Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) and willingness to...
- Willingness to consent to genetic testing for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in...
- Willingness to consent to vaccinations for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in...
- Participant able to perform all necessary assessments to qualify for enrollment and dosing in the corresponding cohort at the time the participant or parent/legal guardian signing the informed consent form (and...
- Presence of biallelic, likely pathogenic or pathogenic OTOF variants
- No clinically significant laboratory findings on clinical laboratory tests at time of Screening as described in the protocol
- Investigator diagnoses the participant with profound sensorineural hearing loss (SNHL; \>90 dB HL) based on behavioral and physiologic measurements (ABR) of inner ear function
- Outer hair cell presence is confirmed via presence of otoacoustic emissions (≥6 dBSNR) at ≥3 frequencies from 1 to 8 kHz in the ear(s) to be infused with DB-OTO.
- No evidence from measures of hearing loss that show a dependence on body temperature
- From study start and for the duration of the short-term follow-up period (48 weeks): Female participants of childbearing potential and fertile males, must agree to use highly effective contraception.
- History of prior treatment with gene therapy
- Surgical anatomy that would preclude or meaningfully impact the planned surgical approach as indicated by medical imaging (eg, Computed Tomography [CT] or Magnetic Resonance Imaging [MRI]) in the ear(s) to be infused...
- History or presence of other permanent or untreatable hearing loss conditions
- Prior or current history of malignancies
- Prior or current history of meningitis
- History or presence of cochlear implants in the ear(s) to be infused with DB-OTO
- History of risk factor(s) for auditory neuropathy not caused by OTOF pathogenic variants including but not limited to: prematurity, low birth weight, hyperbilirubinemia, Neonatal Intensive Care Unit (NICU) admission...
The study team makes the final eligibility decision.
Where it is enrolling
- Los Angeles, California, United States
- San Diego, California, United States
- Jacksonville, Florida, United States
- Orlando, Florida, United States
- Boston, Massachusetts, United States
- New York, New York, United States
- Cincinnati, Ohio, United States
- Seattle, Washington, United States
- Milwaukee, Wisconsin, United States
- Tübingen, Germany
- Matsumoto-shi, Nagano, Japan
- Pamplona, Navarre, Spain
- Barcelona, Spain
- Madrid, Spain
- Cambridge, United Kingdom
- London, United Kingdom
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, but 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, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Los Angeles, California, United States; San Diego, California, United States; Jacksonville, Florida, United States; Orlando, Florida, United States; Boston, Massachusetts, United States; New York, New York, United States and 10 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.