BRAVE: Phase 2 Study of MNK-1411 in people with Duchenne

A Study to Assess the Efficacy and Safety of MNK-1411 in Boys with Duchenne

Questions and Answers

What stage is this research?

This trial is actively recruiting participants.

What is the goal or purpose of this study?

The goal of this research is to demonstrate that MNK1411 is safe and effective as a treatment for Duchenne.

Who is funding this study?

This study is funded by Mallinckrodt ARD Inc.

Who is eligible to participate in this study?

To participate in this study you must…

  • Be male between 4 and 8 years of age with a documented diagnosis of Duchenne
  • Be able to tolerate subcutaneous injections twice weekly
  • Have serum potassium within the reference range at screening or upon retesting within the screening period
  • Have no symptomatic cardiomyopathy (in the opinion of the investigator)

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

This study involves 11 clinic visits over a period of up to 56 weeks. An open Label Extension is offered to all participants until either the compound is marketed or in case MNK would stop development of MNK-1411. Every participant will receive two subcutaneous injections weekly. This trial does have a placebo arm. The primary outcome measure is the 10 meter walk/run test.

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

The study includes 4 weeks screening, 24 weeks of double blind treatment and an optional open label treatment. An open Label Extension is offered to all participants until either the compound is marketed or in case MNK would stop development of MNK-1411.

Where does this study take place?

This study takes place at up to 55 study sites in 15 countries including the USA, EU, and Middle East.

How many visits to the study site are necessary?

11 clinic visits (after the first 56 weeks: visits during the extended Open Label period will take place every three months.)

Can any visits be done locally?

No, visits must be done at a participating investigator site.

Is there any funding to help pay for travel?

Yes, all study visit related travel expenses will be provided to study participants and their caregivers.

Will I get paid for participating in this study?

No, but all travel expenses are paid by the study.

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.

Participation this study will also help to determine whether MNK1411 is a safe and effective therapy for boys with Duchenne.

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