REGAIN V 1.0

• Research type

  Research Study

• Full title

  A phase I/II multiple ascending dose open-label safety and efficacy study of the Notch Inhibitor LY3056480 in patients with mild to moderate sensorineural hearing loss.

• IRAS ID

  224214

• Contact name

  Anne Schilder

• Contact email

  a.schilder@ucl.ac.uk

• Sponsor organisation

  Audion Therapeutics BV

• Eudract number

  2016-004544-10

• Duration of Study in the UK

  1 years, 6 months, 1 days

• Research summary

  Hearing loss affects 1 in 6 people in the UK, and it is estimated that over 328 million adults are affected worldwide. Sensorineural hearing loss (SNHL), also known as nerve deafness, is the most common cause of hearing loss. This can be caused by damage to the hair cells lining the inner ear. Up until now, this damage has been considered irreversible, but recent discoveries indicate that it is possible to regenerate these hair cells and therefore potentially restore hearing loss using a drug.

  The aim of this trial is to assess the safety and tolerability as well as the effect of the Notch Inhibitor LY3056480, a compound thought to help regenerate hearing in patients suffering SNHL. The compound will be administered directly into the middle ear targetting the inner ear in patients from 18 to 80 years old with mild to moderate SNHL.
  The study will be divided in two parts:

  - Part A: will be conducted in a signle centre located in the UK, where the safety and tolerability of the drug will be evaluated in a minimum of 12 patients.
  - Part B: will be conducted in centres in Germany and Greece. The efficacy of the drug will be evaluated in 50 patients.

  The duration of the study is expected to be 18 months in total and 14 weeks for each participant.

  The REGAIN trial was completed in 2022. Its results have been published in 2024:
  Schilder, A.G.M., Wolpert, S., Saeed, S. et al. A phase I/IIa safety and efficacy trial of intratympanic gamma-secretase inhibitor as a regenerative drug treatment for sensorineural hearing loss. Nat Commun 15, 1896 (2024). https://doi.org/10.1038/s41467-024-45784-0

• REC name

  London - Central Research Ethics Committee

• REC reference

  17/LO/0632

• Date of REC Opinion

  12 Jun 2017

• REC opinion

  Further Information Favourable Opinion