STandardizing Operating Procedures and end-Points in Myotonic Dystrophy type 1 (STOPP-DM1) – getting ready for clinical trials

There are a number of potential drugs coming into clinical research in the very near future for patients living with Myotonic Dystrophy. The success of these drugs is not just related to the safety and efficacy of the drugs themselves, but it depends largely on the possibility to monitor the effects of the drugs in the best possible way and in the largest cohorts available. The effect of any intervention can only be understood and quantified if there is information on what would happen to that symptom or organ without the intervention. This is the natural history information which however is still lacking in Myotonic Dystrophy type 1 (DM1) especially when we think of symptoms affecting organs beyond muscle. This study will allow to collect information on the different organs involved over time, so that any intervention affecting muscle or other organs will have a comparison set of data obtained without the intervention/drug to verify the effects of the treatment option. The other critical factor for the upcoming clinical trials with new potential drugs is that there are a limited number of spaces and resources available at the Clinical Research Trial Units and a limited number of patients who can therefore have access to potential drugs in a clinical research trial setting. Often patients are obliged to travel long distances, to sleep overnight outside their homes increasing the burden of fatigue, time and resources for patients and families to reach the Research sites. This study will allow to implement the clinical research framework at local sites throughout the country facilitating access to clinical trials for a greater number of patients.