Decibel Therapeutics has secured approval from the Spanish Agency of Medicines and Medical Devices (AEMPS) for its clinical trial application (CTA) to commence the Phase I/II CHORD study of DB-OTO to treat paediatric patients with congenital hearing loss due to an otoferlin deficiency.
The CTA marks a global regulatory strategy for the clinical development of DB-OTO, which is a cell-selective, adeno-associated virus (AAV) gene therapy product candidate.
Decibel, in partnership with Regeneron Pharmaceuticals, has been developing the product.
In October last year, DB-OTO obtained regulatory approval for an investigational new drug (IND) application in the US.
The product also secured a CTA approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) in January this year.
The dose escalation CHORD trial will examine the safety, tolerability and efficacy of DB-OTO in the targeted patients.
It will also use the auditory brainstem response (ABR), a clinically accepted, physiologic measure of hearing sensitivity, as an efficacy endpoint.
The trial plans to include infants aged two years and under in the UK and Spain.
Initial safety and tolerability data as well as preliminary efficacy results, as measured by ABR, from the first patients of the trial are expected to be revealed in the first quarter of next year.
Decibel Therapeutics CEO Laurence Reid said, “Spain is an important country within our DB-OTO clinical development strategy, and our trial will leverage the collaborations and natural history work that we have pursued there over the past several years.
“Our team believes that DB-OTO could be a transformative treatment for individuals with otoferlin-related hearing loss, and this approval broadens the opportunity to evaluate its potential in infants two years of age and younger.”
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