Santhera completes enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone)
Today, Santhera Pharmaceuticals announced full recruitment of its Phase 3 SIDEROS study with idebenone in Duchenne muscular dystrophy (DMD). The sample-size and variability re-assessment performed according to study protocol demonstrated that with the currently enrolled patients the study has a very high power. Santhera is now assessing the potential of conducting an interim analysis to test for overwhelming efficacy with a view of completing the trial early.
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Santhera hosts a teleconferences dedicated to answer questions regarding the Sideros study. Those interested can select the dates and times that fit them here by completing this Doodle.
About the study
SIDEROS, the largest currently ongoing clinical trial in DMD, is a double-blind randomized placebo-controlled Phase 3 study evaluating the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD. Patients on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status were randomized to receive oral idebenone (900 mg/day three times a day) or placebo for 18 months. The primary endpoint of the trial estimates the treatment difference in FVC%p (forced vital capacity % predicted). Patients completing the trial are offered the opportunity to enroll in an open label extension study where all patients receive idebenone. The study is currently conducted in 62 sites in the United States, Europe and Israel. Further information is available at ClinicalTrials.gov NCT#02814019.
About Idebenone
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS) and supplementing cellular energy levels.
DELOS is a Phase 3, double-blind, placebo-controlled 52-week study which randomized 64 patients, not taking concomitant glucocorticoids, to receive either idebenone (900 mg/day) or matching placebo. The study met its primary endpoint, the change from baseline in peak expiratory flow (PEF) expressed as percent of predicted, which demonstrated that idebenone can slow the loss of respiratory function and reduces the risk of bronchopulmonary adverse events. Supportive data for idebenone were shown in the Phase 2 double-blind, placebo-controlled DELPHI study and its 2-year open-label extension study (DELPHI-E).
SYROS is a prospectively planned, retrospective collection of long-term respiratory function data from 18 patients who completed the DELOS study and subsequently received idebenone (900 mg/day) under Expanded Access Programs (EAPs). The SYROS study showed that the previously observed beneficial effect of idebenone in reducing the rate of respiratory function decline was maintained for up to six years during treatment.
