- Rare Disease Day 2013
- European Neuromuscular Centre (ENMC) workshop
- GSK announces support for AllTrials campaign for clinical data transparency
- Drisapersen (skips exon 51) US phase II clinical trial
- SKIP-NMD Bi-annual Meeting
- 18th International World Muscle Society Congress, 1-5th October 2013, California USA
- Members of the Skip-NMD consortium met in Rome on 2nd-3rd June 2014 for the scheduled 6 monthly meeting.
- World Duchenne Awareness Day
- SKIP-NMD Clinical Trial Applications submitted
- SKIP-NMD Webinar for DMD families on 8th September
- SKIP-NMD Webinar now available
- Information regarding the SKIP-NMD clinical trial of Sarepta’s drug candidate SRP-4053 is now available on www.clinicaltrials.gov.
- Sarepta Therapeutics Announces First Patient Dosed in European Phase I/II Study of SRP-4053 in Duchenne Muscular Dystrophy Patients
- SKIP-NMD clinical trial now progressing to part 2
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:
- 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
- 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.