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Gene Therapy
Gene Therapy for Congenital Hearing Loss (CHORD Trial)
Phase 1 & 2
Recruiting
Research Sponsored by Regeneron Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Profound sensorineural hearing loss (SNHL; ≥ 90 dB HL) based on behavioral and physiologic measurements (ABR) of inner ear function for infants ≤24 months of age
Presence of otoacoustic emissions (OAE) in the ear(s) to receive DB-OTO for infants ≤24 months of age in UK & Spain
Must not have
Prior or current history of meningitis
Prior or current history of malignancies
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Summary
This trial tests a gene therapy to help kids with a genetic disorder. It assesses safety, tolerability & efficacy of the therapy.
Who is the study for?
This trial is for children under 18 with profound sensorineural hearing loss due to OTOF gene mutations, who meet cochlear implant criteria and have not benefited from ear amplification. They must not have had previous gene therapy or cochlear implants in the affected ear(s), nor other untreatable hearing conditions.
What is being tested?
DB-OTO, an AAV based gene therapy, is being tested on pediatric patients with biallelic OTOF mutations. The study has two parts: Part A tests increasing doses in patients, while Part B expands to more participants receiving the treatment bilaterally.
What are the potential side effects?
As this is a first-in-human trial for DB-OTO, specific side effects are unknown but may include typical gene therapy-related reactions such as immune responses to the viral vector or inflammation at injection sites.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My child has severe hearing loss diagnosed by tests.
Select...
My child's ears can produce sounds in response to a test, and they are 24 months old or younger.
Select...
My child's ear function for hearing has been confirmed to be normal.
Select...
My child under 2 years doesn't respond to certain sounds and is in the UK or Spain.
Select...
My child has severe hearing loss and struggles to understand words.
Select...
My child does not respond to certain sounds and is between 2 and 18 years old.
Select...
I am under 18 and fit the age group needed for this study.
Select...
I have genetic mutations in both copies of my OTOF gene.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have had meningitis before.
Select...
I have had cancer before or have it now.
Select...
My ear structure allows for the planned surgery based on my scans.
Select...
I have permanent hearing loss not related to treatment.
Select...
I have previously undergone gene therapy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: DB-OTO - Dose ExpansionExperimental Treatment1 Intervention
Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from the Dose Escalation phase (Part A).
Group II: DB-OTO - Dose EscalationExperimental Treatment1 Intervention
Unilateral intracochlear dosing
Find a Location
Who is running the clinical trial?
Regeneron PharmaceuticalsLead Sponsor
671 Previous Clinical Trials
385,821 Total Patients Enrolled
Decibel TherapeuticsLead Sponsor
2 Previous Clinical Trials
42 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
284 Previous Clinical Trials
254,895 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had meningitis before.I have had cancer before or have it now.My child's inner ear function is confirmed normal for DB-OTO treatment.My child has severe hearing loss diagnosed by tests.My child's ears can produce sounds in response to a test, and they are 24 months old or younger.My child's ear function for hearing has been confirmed to be normal.My child has detectable inner ear responses and is between 2 and 18 years old.Your recent lab tests don't show any important health problems.My ear structure allows for the planned surgery based on my scans.My child under 2 years doesn't respond to certain sounds and is in the UK or Spain.My child has severe hearing loss and struggles to understand words.My child does not respond to certain sounds and is between 2 and 18 years old.You have had a cochlear implant in the ear where the study drug will be injected.I am under 18 and fit the age group needed for this study.I have permanent hearing loss not related to treatment.Your hearing loss is not affected by changes in body temperature.I agree to use effective birth control and not to conceive or father a child.I have previously undergone gene therapy.I have risk factors for hearing issues not due to genetic causes, such as being born early or with low weight.The doctor decides that the patient is a good candidate for cochlear implants in both ears based on the recommended guidelines for cochlear implants.I have genetic mutations in both copies of my OTOF gene.The doctor decides that using a hearing aid won't be very helpful for you.
Research Study Groups:
This trial has the following groups:- Group 1: DB-OTO - Dose Escalation
- Group 2: DB-OTO - Dose Expansion
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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