Date: 2014-09-24
Type of information: Initiation of the trial
phase:
Announcement: initiation of the trial
Company: Alnylam Pharmaceuticals (USA - MA)
Product:
Action mechanism:
Disease: cardiac amyloidosis
Therapeutic area: Rare diseases - Genetic diseases - Cardiovascular diseases
Country:
Trial
details: DISCOVERY is a prospective, multi-center screening study designed to enroll up to 1,000 patients suspected of having cardiac amyloidosis. The study\'s primary objective is to characterize the frequency of TTR mutations. The study\'s secondary objective is to further characterize disease features in patients found to have a TTR mutation.
Latest
news: * On September 24,2014, Alnylam Pharmaceuticals announced that it has initiated its DISCOVERY trial, a screening study examining the prevalence of transthyretin (TTR) mutations in patients suspected of having cardiac amyloidosis. With this study, Alnylam aims to identify and facilitate the diagnosis of familial amyloidotic cardiomyopathy (FAC). Alnylam is advancing ALN-TTRsc, an investigational candidate targeting TTR for the treatment of patients with TTR cardiac amyloidosis, including FAC - which is caused by mutations in TTR - and senile systemic amyloidosis (SSA) - which is caused by idiopathic amyloid deposition of wild-type TTR in the heart. DISCOVERY is a prospective, multi-center screening study designed to enroll up to 1,000 patients suspected of having cardiac amyloidosis. Results from DISCOVERY are expected to determine the frequency of TTR mutations in patients with clinical and/or radiological findings of cardiac amyloidosis and identify FAC patients who may be eligible for clinical trials of novel investigational therapeutics such as ALN-TTRsc. Alnylam is currently conducting a Phase 2 study of ALN-TTRsc aimed at evaluating the safety, tolerability, pharmacodynamic and preliminary clinical activity of ALN-TTRsc in patients with FAC and SSA. The Phase 2 trial is an open-label, multi-dose study of ALN-TTRsc, designed to enroll approximately 25 TTR cardiac amyloidosis patients with FAC or SSA. The primary objective of the study is to evaluate the safety and tolerability of ALN-TTRsc. The secondary objectives are to assess the pharmacodynamic effect of ALN-TTRsc on serum levels of TTR and characterize the pharmacokinetics of ALN-TTRsc. In addition, a number of exploratory clinical endpoint data are being collected. Alnylam expects to present initial results from the Phase 2 study at a meeting to be held during the American Heart Association meeting in November. Patients completing the Phase 2 trial may be eligible to participate in an open-label extension (OLE) study for further assessment of general tolerability and clinical activity with long-term dosing; the ALN-TTRsc Phase 2 OLE study is on track to be initiated in the coming weeks. Alnylam expects to begin a Phase 3 trial in TTR cardiac amyloidosis patients by the end of 2014.
