8th International Wolfram Symposium Presentation Dr Christophe Orssaud – Functional Unit of Ophthalmology, Paris, France
8th International Wolfram Symposium Presentation Dr Christophe Orssaud – Functional Unit of Ophthalmology, Paris, France
Audiowolf : Description, contribution and interest of this protocol
Alongside the Treatwolfram protocol initiated by Pr T Barrett, a second new protocol has been initiated in France, called Audiowolf. These two protocols are similar in terms of their duration (3 years), the patients for whom they are intended (patients with Wolfram syndrome) and the molecule used (sodium valproate). They nevertheless differ by several points which make them complementary.
These differences relate first to the primary judgment criterion. This primary judgment criterion is auditory in Audiowolf and not visual. It is also a non-randomized protocol without double blinding. All the patients included receive the treatment at a dose adapted to their weight. Finally, it was initially designed as being monocentric (Paris), although it is currently undergoing extension to Spain (Almeria).
However, this Audiowolf protocol is not a competitor but rather complementary to Treatwolfram. Indeed, it makes it possible to include patients who did not meet the inclusion criteria of the Treatwolfram protocol due to too low visual acuity. On the other hand, the auditory, visual, neuro-radiological and biological tests are identical. Thus, after a separate processing of both protocols, these different data can be combined for common analyses. Finally, this protocol will make it possible to better analyze the auditory impairment of Wolfram syndrome. On the other hand, all patients receiving sodium valproate, an ancillary study was initiated to understand how this molecule may affect insulin regulation.
In practice, the inclusion criteria require, among other things, a hearing impairment of at least 5 dB at 8000 Hz. And this protocol can be delivered “turnkey” to teams that would like to propose it to their patients.
Points noted:
• Patients enrolled in this study must have some level of hearing loss (those with hearing implants can be included).
• Study first started in Nov 2021 – after TreatWolfram (to avoid patients needing to choose which study to participate in).
• Secondary objective of the study is to evaluate C-peptide to try to understand how sodium valproate acts on insulin secretion.
• A rare side effect of sodium valproate is hearing loss, which is typically bilateral and reversible. This will need to be considered should any patients experience severe hearing loss during the study (patient data likely to be excluded, at least initially).