Résumé :
|
Background: A 12-month double-blind, randomized, placebo-controlled phase II study in 21 patients with DMD (DELPHI study) has indicated that idebenone (Catena®, 450 mg/d) may improve cardiac and respiratory function in 8-16 year old patients with DMD. Patients completing the DELPHI study were eligible to enroll into the open label DELPHI study (DELPHI-E) of 24 months duration to further investigate the long-term safety and efficacy of idebenone in this disease.Methods: Nineteen patients were enrolled into DELPHI-E conducted at the University Hospitals Leuven. All patients received Catena®at 450 mg/d (patients _ 45 kg body weight) or 900 mg/d (patients > 45 kg body weight). Safety and tolerability was assessed by adverse event recording, vital signs, ECG and laboratory evaluations. Efficacy was assessed as change from baseline to month 6, 12, 18 and 24 for respiratory function tests (e.g. peak expiratory flow (PEF), forced vital capacity (FVC), maximum mouth pressures). Cardiac function tests were conducted by echocardiography and Tissue Doppler Myocardial Imaging, particularly by assessment of the peak systolic radial strain of the left ventricular inferolateral wall, the region of the heart that is the earliest and most severely affected in DMD.Results: Nineteen patients at age 15.1 ± 2.6 years (mean ± SD) have been enrolled and completed the 24 months study period of DELPHI-E. Eleven patients were taking concomitant glucocorticoids. Catena®was safe and generally well tolerated. Baseline respiratory function data for the entire study cohort were: FVC: 2.20 ± 0.85 l, FVC percent predicted: 68.8 ± 25.6%, PEF: 269.4 ± 93.6 l/min, PEF percent predicted: 66.1 ± 22.3 %. The change over time in respiratory as well as cardiac function data will be presented. Conclusion: Data from this long-term open label study will inform on the usefulness of different respiratory and cardiac efficacy outcome measures for intervention trials in DMD. The study also will provide additional data to assess the safety and therapeutic potential of idebenone (Catena®) in patients with DMD. Study sponsored by Santhera Pharmaceuticals.
|