Date: 2014-11-18
Type of
information: Initiation of the trial
phase: 3
Announcement: initiation of the trial
Company: Sarepta Therapeutics (USA - MA)
Product: eteplirsen
Action
mechanism: Eteplirsen is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Eteplirsen uses Sarepta\'s novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene\'s ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.
Disease: Duchenne Muscular Dystrophy
Therapeutic
area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country: USA
Trial
details:
- The main objective of this study is to provide confirmatory evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety and biomarkers. This open-label, multi-center, 48-week study will evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen. Patients in the untreated group will not receive treatment. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
- The sponsor is working to initiate approximately 39 sites across the United States. Sites will vary in the following functions: Local Site (N=39) - Enrolls patients and is the primary contact point for their patients. Sites will perform all protocol activities (including dosing and laboratory assessments), except for functional assessments and biopsies. Hub Site (N=14) - Performs functional (physical) assessments at specified times per protocol. Surgical Site (N=2) - Performs muscle biopsies for the Treated group. (NCT02255552)
Latest
news:
- • On November 18, 2014, Sarepta Therapeutics announced that it has initiated dosing in a confirmatory study of eteplirsen in ambulatory patients who meet specific criteria on their baseline 6-minute walk test score.
This open-label study, 4658-301 (PROMOVI), designed to evaluate the efficacy and safety of eteplirsen in DMD patients over 48 weeks of dosing, will be conducted at approximately 39 sites across the United States and include up to 160 patients. The study will enroll 60-80 boys, aged 7-16 years with genotypes amenable to exon 51 skipping who will be treated with eteplirsen, while an additional 60-80 patients with genotypes not amenable to exon 51 skipping will serve as a concurrent control group. Patients enrolled in the treatment group of the study will receive once weekly intravenous infusions of 30 mg/kg of eteplirsen. Data will be collected across a number of efficacy and safety parameters, including dystrophin assessments and will be powered to show a benefit on the 6-minute walk test compared with the untreated DMD control group. Sites are currently being initiated into the study. Initiation of approximately 39 planned sites in the United States is expected to be completed by January 2015.
Is
general: Yes
