The UK Medicines and Healthcare products Regulatory Agency (MHRA) has authorised Decibel Therapeutics’ clinical trial application (CTA) for initiating a Phase I/II clinical trial of DB-OTO in paediatric patients.
The dose escalation clinical trial will assess DB-OTO’s tolerability, safety, and efficacy in paediatric patients with congenital hearing loss due to an otoferlin deficiency.
In the trial, the auditory brainstem response (ABR), an objective, clinically accepted, physiologic measure of hearing sensitivity, will be used as an efficacy endpoint, in addition to safety and tolerability endpoints.
In animal models, the ABR was used for characterising the dose-response of the therapy following intra-cochlear delivery in translational studies.
An update on the trial design is expected during the middle of this year.
Decibel Therapeutics CEO Laurence Reid said: “This CTA marks the second regulatory clearance for DB-OTO, and what we believe to be the first in Europe for any gene therapy targeting otoferlin.
“This is an important milestone as we execute our international clinical development strategy, which addresses paediatric patients of diverse ages, including infants two years of age and younger.
“We believe DB-OTO has transformative potential for individuals with otoferlin-related hearing loss, and we intend to initiate the clinical trial in the first half of 2023. We expect to share initial data from the first cohort of patients in the first quarter of 2024.”
The authorisation provides approval for Decibel to expand its planned Phase I/II trial to the UK in patients aged two years and younger.
DB-OTO, the company’s lead gene therapy product candidate, is being developed in partnership with Regeneron Pharmaceuticals.
The cell-selective, adeno-associated virus gene therapy has been designed to offer durable hearing in individuals with congenital hearing loss which is caused by otoferlin gene mutations.
The UK CTA is part of a global regulatory strategy for the therapy’s clinical development.
Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.
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