Date: 2016-09-28
Type of information: Treatment of the first patient
phase: 3
Announcement: treatment of the first patient
Company: Sarepta Therapeutics (USA - MA)
Product: SRP-4045 and SRP-4053
Action
mechanism: antisense oligonucleotide. SRP-4045 and SRP-4053 are designed to address the underlying cause of DMD by restoring the messenger RNA (mRNA) reading frame, thus enabling the production of a shorter form of the dystrophin protein. SRP-4045 and SRP-4053 use Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exons 45 and 53, respectively, of the dystrophin gene. Promoting the synthesis of a shorter dystrophin protein is intended to slow the decline of ambulation and mobility seen in DMD patients.
Disease: Duchenne muscular dystrophy
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country: Belgium, Canada, France, Germany, Italy, Netherlands, Sweden, UK, USA
Trial
details: The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53 respectively. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers. Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group, 66 patients) or placebo (33 patients) for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open label extension period in which all patients will receive open-label active treatment for up to 96 weeks. (NCT02500381)
Latest
news: * On September 28, 2016, Sarepta Therapeutics announced the first patient dosed in the phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.
The Phase III study, ESSENCE, is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group, 66 patients) or placebo (33 patients) for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open label extension period in which all patients will receive open-label active treatment for up to 96 weeks.
The study will enroll approximately 99 patients aged 7 to 13 years, inclusive, with a minimum target of 45 patients amenable to exon 45 skipping and 45 patients amenable to exon 53 skipping. Twice as many patients will receive active treatment as will receive placebo. Approximately 66 patients will be randomized to receive active treatment with either SRP-4045 or SRP-4053 (depending on deletion mutation), and 33 patients will be randomized to receive placebo.
