A note from Debra

Update on Phase 3 tadalafil DMD study: enrollment completed

Posted on January 13, 2015 8:48 pm

CureDuchenne is pleased to share the following update to the Duchenne muscular dystrophy community from Lilly regarding the ongoing Phase 3 trial of tadalafil in boys with Duchenne.

Eli Lilly and Company is pleased to announce the completion of enrollment of the Phase 3 trial of tadalafil for patients with Duchenne muscular dystrophy (DMD).

The study, entitled A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy (ClinicalTrials.gov #NCT01865084), is a 48-week clinical trial designed to determine the efficacy and safety of once-daily tadalafil treatment in ambulatory boys with DMD.  The primary endpoint is the change in walking distance as measured by the six-minute walk test.  Enrollment in the trial has ended with 331 patients randomized to placebo or one of 2 doses of tadalafil at 63 study sites globally. Top-line results from the trial are expected in the first half of 2016.

We would like to sincerely thank the many boys and their families who gave of their time to be screened for the trial, and for the continuing commitment of those patients who currently are participating in the trial.  We also greatly appreciate the hard work and dedication of the site investigators and staff that made completion of enrollment possible.

 

Keeping the Promise for our Children with Duchenne

Posted on January 12, 20158:25 pm

Today, CureDuchenne announced a $1 million investment in Capricor Therapeutics for a clinical trial to test its CDC (adult) stem cells as a cardiac therapy for Duchenne patients. CureDuchenne’s mission is our name…to cure Duchenne.  Duchenne affects so many parts of the body and cardiac failure is the primary cause of death in this disease.  Read more »

Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy

Posted on 5:03 pm

Sarepta Therapeutics released the 168 week data for their exon skipping drug, eteplirsen, this morning.  During a three year period, 7-13 year old boys with Duchenne (the age range of the boys who began the 201 study) would be expected to slow down considerably and many expected to lose ambulation entirely.  After more than three Read more »