Date: 2015-01-14
Type of information: Treatment of the first patient
phase: 1-2
Announcement: treatment of the first patient
Company: Sarepta Therapeutics (USA - MA) Institut de Myologie (France) Consultants for Research in Imaging and Spectroscopy, Great Ormond Street Hospital for Children (UK) NHS Foundation Trust (UK) Catholic University of the Sacred Heart (UK) Royal Holloway University (UK) SYSNAV, University College, London (UK) University of Newcastle Upon-Tyne (UK)
Product: SRP-4053
Action
mechanism: antisense oligonucleotide.
Disease: Duchenne Muscular Dystrophy
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country: France, Italy, UK
Trial
details: This first-in-human, multiple-dose 2-part study will assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping. This first part will be followed by an open-label efficacy and safety evaluation. (NCT02310906)
Latest
news: * On January 14, 2015, Sarepta Therapeutics announced that it has initiated dosing of SRP-4053 in its first human trial, a Phase I/II study in Duchenne muscular dystrophy (DMD). This multiple-dose study will assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in DMD patients with genotypes amenable to exon-53 skipping. The study will be conducted at four sites in Europe under a consortium agreement between Sarepta and various European hospitals, institutions, and scientists established to conduct the study and which is funded in part by the European Union’s Seventh Programme for research, technological development and demonstration under grant agreement No. 305370. The SKIP-NMD is an EU FP7 funded collaborative grant involving 10 partners from Europe and the U.S., whose aim is to restore dystrophin production in a subset of DMD boys. This will be achieved by developing a drug which ‘skips’ the mutations causing DMD, so as to restore dystrophin protein expression. The SKIP-NMD partners include University College London, Great Ormond Street Hospital for Children, Royal Holloway-University of London, Charles River Laboratories, Università Cattolica del Sacro Cuore, Institute of Myology, Sysnav, Consultants for Research in Imaging and Spectroscopy, Newcastle University, and Sarepta Therapeutics.
