NATURAL HISTORY STUDY FOR FUTURE TRIALS
A detailed mapping out of the natural history of the functioning of eyesight and hearing with the different types of Usher Syndrome is essential for being able to determine the effectiveness of future therapy. Only after studies have demonstrated the effectiveness and safety of certain therapy types, these will be made available to patients on a large scale after a long process of market implementation.
NATURAL HISTORY STUDY FOR USH 1C AND USH 1F READY TO START
By now, Dr. Lentz and her team in Louisiana have started a natural historystudy with patients having mutations in the USH 1C gene. Both the eyesight and the loss of balance are accurately recorded.
The Usher 1F Collaborative in the USA received a large subsidy of $ 450.000 from Facebook top executive Mark Zuckerberg early in the year 2020. A part of the subsidy will be used to start a natural development study, ensuring accurate evaluation of future trials for effectiveness.
The CRUSH – Characterizing Rate of progression of USHer syndrome – study has been aligned to RUSH2A. This means that the research questions and the study measurements are largely similar, allowing the results of the CRUSH study to be compared with those of the RUSH2a study. This comparison is of high scientific value.
By now, both the RUSH2A and the CRUSH studies have been started in the Usher Syndrome Expertise Centre in Radboudumc in Nijmegen, the Netherlands.
Read more about the differences between de CRUSH en the RUSH2A.
RUSH 2A STUDY
In the international RUSH2A study of Jacque Duncan, MD, of the University of California, San Francisco, 70 patient spread over 9 different clinics are monitored for 4 years. In this study only syndromic and non-syndromic patients with mutations in the USH 2A gene are included.
The RUSH study – Rate of progression of USHer syndrome – was started late 2017 and takes place at about 20 clinical locations throughout the world, including the Radboudumc in Nijmegen, the Netherlands.
RUSH 2A researchers make use of various technologies to follow the changes and deterioration in eyesight and the retina.
This study is financed by the Foundation Fighting Blindness.
In the summer of 2018, a natural development study for USH 1B was started in Oogziekenhuis Rotterdam with 10 patients. Apart from Oogziekenhuis Rotterdam institutes in Naples and Madrid are involved in this study as well. The study is a preparation of the start of the clinical trial USHTher, a gene therapy using double AAV vectors.