Since 2011, Action Duchenne has invested in two programs of the Pilot Trials Now initiative, which ended at the end of 2013.

The first trial testing sildenafil (Revatio or Viagra), originally developed by Pfizer as a heart medication, has been shown to delay and even prevent heart failure in the DMD animal model.  The prinicipal Investigator Kathryn Wagner, Kennedy Krieger Institute of Johns Hopkins University led the 12-month, double-blind, placebo-controlled REVERSE DBMD pilot trial to test if Phosphodiesterase  5 inhibition with sildenafil improved cardiac function in adults and adolescents living with Duchenne.

In August 2012, the Food and Drug Administration authority notified healthcare professionals that “use of Revatio, particularly chronic use, in children is an off-label indication, not approved by FDA and is not recommended.” The trial consequently excluded those enrolled under the age of 18.

The full data analysis of the secondary endpoints is still ongoing and is awaiting publication.  This was a small study due to Data Safety Monitoring Board recommended closure; therefore this is a cautious interpretation. Overall, Sildenafil was well tolerated in DMD men, but is unlikely to provide benefit to cardiac or skeletal muscle in adult DMD. Sildenafil at 20 mg three times a day for 6 to 12 months may have adverse effects on cardiac function.  The study authors concluded that the monitoring of cardiac function in future clinical trials modulating this pathway will be necessary.

The second trial from the Pilot Trials Now scheme was recombinant human insulin-like growth factor-I (IGF-I) therapy in Duchenne. IGF-I was seen as offering potential as a therapeutic agent by improving or preserve muscle function, and countering the effects of glucocorticoid steroid use in some boys, of growth failure and insulin resistance.

In this first study of IGF-I therapy in DMD boys, 6 months of once-daily IGF-I significantly increased height velocity and compared to controls, but there was no difference in motor functional outcomes.

These results were presented at the World Muscle Congress in October 2013 and are currently awaiting acceptance in a leading peer-reviewed scientific journal the European Society of Paediatric Endocrinology.