Rare Disease Report

Duchenne Patient Conference Update: Eli Lily's Tadalafil Registration Trial

JUNE 30, 2014
RDR Staff

David Cox PhD, of Eli Lily recently presented an update on their phase 3 clinical trial examining the efficacy and safety of tadalafil in boys with Duchenne muscular dystrophy at PPMD’s Connect 20th Annual Conference.
As many of you are aware, tadalafil increases blood flow and is most notably used to help men with erectile dysfunction.  The ability of tadalafil to increase blood flow – in skeletal muscles – may also help boys with Duchenne muscular dystrophy.

Phase 3 Clinical Trial

The phase 3 registration trial began in September 2013. The study is a 48-week placebo controlled trial in which ambulant boys (N=306) with Duchenne muscular dystrophy between the ages of 7 and 14 are randomized to receive placebo or tadalafil (0.3 or 0.6 mg/kg). The study is being conducted on over 60 centers in 15 countries. For details of the inclusion/exclusion criteria, as well as the list of study locations, visit clinicaltrials.gov
Primary endpoint is the 6-minute walk test (6MWT) as well as numerous secondary endpoints including North Star Ambulatory Assessment (NSAA) global score, numerous timed function tests, time to persistent 10% worsening of the 6MST, and time to persistent 10% worsening of the timed function tests.  Pharmacokinetics and safety outcomes will also be assessed.
For more information about the study, visit clinicaltrials.gov


At the present time, no data is available but Dr Cox  stated they expect to complete the double blind phase of the study by end of 2015 and initial results should be available in early 2016.  A 48-week extension study is also planned for participants wanting to continue the trial.

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