I think that this progress report exemplifies the trials and tribulations that all researchers go through in putting together a human clinical study. A lot of regulatory bodies, each with their own set of paperwork.
In the last six months, we have been working on the regulatory hurdles for the immunosuppression trial.
We submitted an Investigational New Drug (IND) Application to the Federal Drug Administration under section 505(i) of the Federal Food, Drug, and Cosmetic Act for the use of tacrolimus and prednisone in facioscapulohumeral dystrophy and have received the permission to proceed.
We are in the process of obtaining UW IRB approval and are working on revisions.
We are in the process of completing negotiations with the following UW services:
- UW lab services to establish serum drug levels and safety monitoring measures
- DISC research radiological services for research MRI and chest x-ray pricing
- UWMC Pharmacy Services IDS for research drug monitoring and dispensing.
We are in the process of establishing case report forms (CRFs) for the study.
We have discussed the study in broad terms to subjects in the Wellstone project to inform them of the study. This will be part of our recruitment strategy.
Unfortunately, without IRB approval, we have not been able to access funds and have been using alternate funds to start the process. We are hopeful to get IRB approval, to then be able to access funds, to get all the services in place, to get a Data Safety Monitoring Board in place, and to get the study started.
It has been a lot of work but we are slowly making progress. We believe that this study remains important and are grateful for the support of Friends of FSH Research in this endeavor.
Leo H. Wang, MD PhD
Department of Neurology
University of Washington
See grant Clinical Study using MRI to measure immune response.