Summary

Eligibility
for males (full criteria)
Location
at UC Davis UCLA UCSF
Dates
study started
completion around
Principal Investigator
by Jonathan Strober (ucsf)
Headshot of Jonathan Strober
Jonathan Strober

Description

Summary

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).

The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

Official Title

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Keywords

Duchenne Muscular Dystrophy, Becker muscular dystrophy, Nonsense mutation, Premature stop codon, DMD, BMD, nmDBMD, DBMD, Ataluren, PTC124, Muscular Dystrophies

Eligibility

Locations

Lead Scientist at University of California Health

  • Jonathan Strober (ucsf)
    Professor, Neurology, School of Medicine. Authored (or co-authored) 63 research publications

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
PTC Therapeutics
Links
PTC Therapeutics, Inc website Protocol V7.0
ID
NCT01247207
Phase
Phase 3 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 270 study participants
Last Updated