Essence

Study of SRP-4045 and SRP-4053 in DMD Patients

Lay summary/DUK summary

This study is a stage 3 trial of Sareptas exon 45 and exon 53 skipping drugs. Exon skipping drugs use a small piece of genetic material to skip over a the part of the dystrophin gene with a mutation. The part of the dystrophin gene with a mutation varies between patients. This is why exon skipping trials are mutation specific.

The main objective of this study is to determine the efficacy of the drugs compared to a placebo in DMD patients. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers.

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Lorem ipsum dolor sit amet, consectetur adipisicing elit. Doloremque assumenda minus rerum dolores, quos animi laboriosam nesciunt? Numquam nemo, accusantium! Earum, placeat itaque assumenda qui voluptatum consectetur libero amet officia?

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Trial Status
Recruiting
Age
7-17 years
UK Locations
Leeds, Liverpool, GOSH, Newcastle
Trial Sponsor
Sarepta
Mutation Specific?
Yes, must be amenable to exon 45 and 53 skipping
Muscle Biopsy?
Yes
MRI
Yes
Phase
3
Length Of Participation
Up to 96 weeks
Recruitment Target
99
Ambulatory
Yes 6MWT required
Therapeutic Category
Exon skipping