News Publication

SKIP-NMD clinical trial now progressing to part 2

22 July 2015

 The SKIP-NMD team successfully completed the first part of the clinical trial, a dose escalation phase with no significant problem for the 12 children recruited so far, and recently obtained the go-ahead from the data safety monitoring board to initiate the second, maintenance  phase of the study.

We are now looking to recruit 12 additional DMD boys for the Part 2, maintenance phase, of the study, at 4 clinical sites in London, Paris, Newcastle and Rome.

This clinical trial has two arms for which we are recruiting:

  1. SRP-4053 treated group: Have been diagnosed with Duchenne muscular dystrophy and have a deletion amenable to exon 53 skipping (e.g., deletions of exons such as 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, or 54-58); and
  2. Untreated control group: Have an established clinical diagnosis of DMD with confirmed genomic deletion of exon(s) not amenable to exon 53 skipping. Boys in the untreated control group will not receive SRP-4053 but will participate in the same study assessments as the treated group, on a reduced schedule. Taking part in the untreated control group will not preclude participation in future Sarepta trials involving active treatment.

Generally, although other requirements may apply, boys who meet the following criteria may participate in Part 2 of this clinical trial in the SRPT-4053 treated group or the Untreated control group:

  • Are between six to 15 years old.
  • Can walk at least 250m in six minutes.
  • Have stable respiratory and heart function.
  • Have been on a stable dose of corticosteroids for at least 6 months.

Please contact the team for further information.

Information regarding this clinical trial is also available on www.clinicaltrials.gov.