ATALUREN - A Phase 3 Trial in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

A phase 3 randomized, double-blind, placebo-controlled efficacy and safety study of ataluren in patients with nonsense Glossary Link mutation Duchenne Muscular Dystrophy (nmDMD), and Open-label Extension

 

Frequently Asked Questions (FAQs):

What stage is this research?

  • This trial is actively recruiting participants.

 

What is the goal or purpose of this study?

  • The goal is to study the long-term safety and efficacy effects of ataluren in patients with nmDMD.

 

Who is funding this research?

  • This study is funded by PTC Therapeutics.

 

Who is eligible to participate in this study?

  • To participate in this study you must be:
    • Male with Duchenne
    • Age ≥5 years
    • Nonsense point mutation in the dystrophin Glossary Link gene
    • Use of systemic corticosteroids (prednisone/prednisolone or deflazacort) for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
    • Ability to perform 6MWD ≥150 meters
    • Ability to perform timed function tests within 30 seconds
    • Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. 

 

What do I have to do if I decide to participate in this study?

  • This study involves clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period.

 

How long will this study last, and will I have access to the drug/treatment once the study has ended?

  • The anticipated length of the study is 72-week of double-blind, placebo controlled and 72-week open-label period.

 

Where does this study take place?

  • This study will be conducted internationally with sites around the world.
    • How many visits to the study site are necessary?
      • Approximately 11 visits over the 144 week duration of the study.
    • Can any visits be done locally?
      • No, visits to the study site will be required to ensure consistency.
    • Is there any funding to help pay for travel?
      • Reimbursement will be determined on a case-by-case basis.

 

Will I get paid for participating in this study?

  • No there is no payment for study participants.

 

Why should I consider participating in this study?

  • While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new research treatments before they are widely available and having access to medical specialists that are normally not available to you or your child, and helping others by contributing to the better understanding of Duchenne.

 

Where can I learn more about this study?

 

(Updated 9/25/2017)