Funding transnational collaborative research through joint transnational calls is one of the major objectives of E-Rare. This is the most important and effective joint activity to enhance the cooperation between European scientists working on rare diseases and thus reducing the fragmentation of research in this field. E-Rare launches calls on a yearly basis. The topic and eligibility criteria are specified every year and therefore may vary from one call to the other.

Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial

Project Coordinator

University of Basel - Universitätskinderspital beider Basel


Vincent Laugel Hautepierre University Hospital Strasbourg, France
Helge Amthor Raymond Poincaré University Hospital Garches, France
Sebahattin Cirak University of Cologne Cologne, Germany
Arpad von Moers DRK Klinik Berlin | Westend Berlin, Germany
Haluk Topaloglu Hacettepe Children’s Hospital Ankara, Turkey

Duchenne muscular dystrophy (DMD) is a rare disease that affects about 1 in 3500 to 1 in 6000 boys. Patients suffer from progressive muscle wasting, respiratory and cardiac impairments and premature death. Currently, only symptomatic treatment with glucocorticoids is available; these have limited efficacy but many adverse effects. Using the mouse DMD model, our partners in Geneva, Dorchies et al., have shown that tamoxifen (TAM), given orally for periods of 2 or 15 month at doses as low as 0.3mg/kg/day, resulted in almost full recovery of force and structure of muscles. Our aim is to investigate whether TAM treatment, compared to placebo, reduces the disease progression in DMD patients. We plan a 48-week placebo controlled randomised clinical trial with 65 ambulant (6.5-10 year old) DMD patients (under stable standard treatment of care with glucocorticoids). Patients will receive 10mg (0.3mg/kg) of TAM or of a placebo daily.

E-Rare 2012 - Created by Toussaint Biger