Tuesday, August 25, 2015

Updates on Clinical Trials

There are numerous clinical trials and research projects happening all over the globe for Stargardt's in different scientific and technological fields giving us great hope for the future. Here are the current clinical trials taking place:

Drug Trial of ALK-001
Alkeus Pharamceuticals are conducting a phase 2 trial of an oral drug in the United States. A phase 2 trial looks at the safety of the drug, what side effects it may have, how long it stays in a person's body and the dosing required. This study will also look at whether it affects the progression of Stargardt's. The trial will follow participants over two years assessing these key factors. For more information click here. If you are located in the United States and are interested in finding out more about participating in the trial visit their website here.

Phase 1 Trial using Gene Therapy in the US and France
This is a gene therapy trial currently recruiting in the US and France. It is a phase 1 trial thus the main focus is on looking at the dose of the sub-retinal injection and the effects it may have. If you are interested in participating, the two centres involved are the Casey Eye Institute in the US and the Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts in France. For more information on the specifics of the trial click here.

Stem Cell Trials
The stem cell trial using human derived embryonic stem cells (hESC) I have previously mentioned is still in progress. This trial is run by Ocata over three sites in the US (California, Florida and Pennsylvania). The trial involves an injection of the stem cells into the retina. The researchers will be observing the participants over a 15 year period to monitor the progression of the stem cells, whether they become retinal cells, how this affects vision and if there are any long term complications. More information can be found here and here.

Investigative Research
There are a couple of research projects taking place looking at different aspects of Stargardt's Disease such as its progression. It is important that researchers continue to look at the how, what, where and why of SD to better understand the disease as we don't have a lot of information about it. This information can then be used for targeted treatments and even prevention of disease progression. These projects are always good to participate in because they involve little risk (unlike treatment trials) as they simply gather information, test results and images. The more participants these types of studies get the more information can be gathered and learnt about a condition. 
The National Institute for Health Clinical Centre in Maryland, USA, is currently recruiting for a natural history study of SD. If you are interested in participating check out their website.
The other project is the Inherited Retinal Degenerative Disease Register which is not only for SD but other inherited retinal diseases. This is run by the Foundation Fighting Blindness and more information can be found here.





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