Résumé :
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Respiratory complications cause early morbidity and mortality in patients with Duchenne muscular dystrophy (DMD). The use of glucocorticoids slows the decline in respiratory function, but their long-term use is hampered by significant side effects. Idebenone, a small molecule ATP production modulator and antioxidant, has shown potential efficacy on early cardiac and respiratory parameters in a Phase IIa randomized controlled study (DELPHI study). Patients receiving Catena®at 450 mg/d for 12 months improved in peak expiratory flow (PEF), whilst patients receiving placebo declined on this early marker of respiratory involvement in DMD. Results of the DELPHI study were the basis for the planning of a large international confirmatory phase III trial.The DELOS study is a double-blind, randomized, placebo-controlled Phase III study investigating the efficacy and safety of 12 month's treatment with idebenone (Catena®, 900 mg/d) compared to placebo in patients with DMD. The international multi-center study follows a group sequential design where 40 patients not using concomitant glucocorticoids are enrolled first (cohort I; ongoing), followed by up to 200 patients allowed of using concomitant steroids (cohort II). Patients between 10 and 18 years of age, independent of their ambulatory status are eligible for enrolment. The primary objective of the study is to assess the efficacy of idebenone compared to placebo in improving respiratory function or delaying the loss of respiratory function in DMD patients. The primary endpoint of the study is the change from baseline to week 52 in PEF percent predicted (PEF %), supported by secondary respiratory endpoints (including FVC and peak cough flow). Respiratory function data will be collected by gold-standard spirometry at baseline and at 3 month intervals during hospital visits. In addition, PEF data will be collected on a weekly basis (home-setting) with a child-compatible hand-held respiratory function device (ASMA-1 from Vitalograph).The update on the DELOS study includes recruitment progress, baseline demographic and respiratory function data summarized for patients enrolled into cohort I of the DELOS study. Study sponsored by Santhera Pharmaceuticals
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